Bioprocessing Summit Europe Highlights Innovations in Gene Therapy CMC and Manufacturing – Yahoo Finance

Spark Therapeutics, Ultragenyx and Others to Headline March 24-26 Meeting in Barcelona

With the global market for gene therapy expected to hit $13 billion (USD) by 2024 (Research And Markets), Bioprocessing Summit Europe is returning March 24-26, 2020 with its popular Gene Therapy CMC and Manufacturing track.

Expanding to a new venue to accommodate increased registrations, the meeting will take place at The Crowne Plaza Barcelona (Spain)Fira Center.

From the Plenary Keynote by Diane Blumenthal, PhD, Head, Technical Development, Spark Therapeutics, the first company to obtain approval of a gene-therapy product in the EU and US, to insights from Biogen, Sanofi, Vivet Therapeutics, Takeda and Ultragenyx, content will focus on accelerating therapies into the market.

"There is a real thirst for knowledge about how to rapidly deploy biologics while ensuring quality and cost-efficiency. This conference reveals the critical challenges facing the manufacture, analysis and control of these exciting new therapies," said Dan Barry, Senior Conference Director, CHI.

Among highlights:

The event will also include:

See bioprocessingeurope.com.

About Cambridge Healthtech InstituteCambridge Healthtech Institute (CHI), a division of Cambridge Innovation Institute, is the preeminent life science network for leading researchers and business experts from top pharmaceutical, biotech, CROs, academia, and niche service providers. CHI is renowned for its vast conference portfolio held worldwide including PepTalk, Molecular Medicine Tri-Conference, SCOPE Summit, Bio-IT World Conference & Expo, PEGS Summit, Drug Discovery Chemistry, Biomarker World Congress, World Pharma Week, The Bioprocessing Summit, Next Generation Dx Summit, Immuno-Oncology Summit, and Discovery on Target. CHI's portfolio of products include Cambridge Healthtech Institute Conferences, Barnett International, Insight Pharma Reports, Bio-IT World, Clinical Research News and Diagnostics World.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200211006146/en/

Contacts

Dawn Ringel+1-781-449-8456, dawn@ringelpr.com

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Bioprocessing Summit Europe Highlights Innovations in Gene Therapy CMC and Manufacturing - Yahoo Finance

University of Minnesota Medical School Ranks 27th in 2019 NIH Rankings – Yahoo Finance

The U of M increases three spots in Blue Ridge Institute of Medical Research rankings

Minneapolis, MN, Feb. 12, 2020 (GLOBE NEWSWIRE) -- The Blue Ridge Institute for Medical Research recognizes the University of Minnesota Medical School as the 27th most funded institution by the National Institutes of Health (NIH) in the 2019 NIH rankings.

The University of Minnesota Medical School is ranked 27th nationally in this years rankings, improving from last years spot as 30th (and 33rd in 2017). In addition, five of the Medical Schools departments Biochemistry, Molecular Biology and Biophysics; Neuroscience; Family Medicine and Community Health; Pediatrics; and Rehabilitation Medicine ranked in the top 10 for department-specific rankings. Several other departments also improved rankings in 2019.

The continued progress of our medical school's climb in the NIH rankings reflects the incredible work taking place every day on our campus and in our healthcare facilities by our faculty, researchers and students, said University of Minnesota President Joan T.A. Gabel. We look forward to building on this sustained success as we seek to solve the most pressing healthcare issues here in Minnesota and around the world."

The NIH is the largest federal provider of basic research money to universities. Each year, the Blue Ridge Institute evaluates NIH data tables and ranks universities based on their annual NIH grant support. Improving the Medical School rankings is a top priority of the Board of Regents as a measure for advancing the University.

Rankings are good indicators of how we are building momentum at the Medical School, said Jakub Tolar, MD, PhD, Dean of the University of Minnesota Medical School and Vice President for Clinical Affairs. They help us recruit and retain our faculty, physicians, staff, and learners, generate community support, and most importantly build trust with our patients. We are grateful for the support of President Gabel and our Board of Regents, as well as the strong partnership we have with the State of Minnesota in advancing our mission.

About the University of Minnesota Medical School

The University of Minnesota Medical School is at the forefront of learning and discovery, transforming medical care and educating the next generation of physicians. Our graduates and faculty produce high-impact biomedical research and advance the practice of medicine. Visit med.umn.edu to learn how the University of Minnesota is innovating all aspects of medicine.

Naomi McDonaldUniversity of Minnesota Medical School6123019525naomim@umn.edu

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University of Minnesota Medical School Ranks 27th in 2019 NIH Rankings - Yahoo Finance

Study Shows That Caris Life Sciences’ ADAPT Biotargeting System Has Discovered Protein Expression Pattern Differences Between Two Prostate Cancer…

First time findings published in Nucleic Acids Research demonstrate the ability to differentiate exosomes from cancer cell subtypes from the same tumor type offering broad potential applications in biomarker discovery

IRVING, Texas, Feb. 12, 2020 /PRNewswire/ -- Caris Life Sciences, a leading innovator in molecular science focused on fulfilling the promise of precision medicine, today announced the publication of new data in Nucleic Acids Research,illustrating that use of the Company's proprietary ADAPT Biotargeting System can lead to the identification of differences in protein expression patterns between exosomes from two related prostate cancer cell lines, vertebral cancer of the prostate (VCaP) and lymph node cancer of the prostate (LNCaP).

Caris Life Sciences Logo (PRNewsfoto/Caris Life Sciences)

The paper, "ADAPT identifies an ESCRT complex composition that discriminates VCaP from LNCaP prostate cancer cell exosomes," also demonstrates that the ADAPT platform can be a powerful method that allows for the enrichment of polyligands that can distinguish even between different subpopulations of the same disease.

The results show that the ADAPT Biotargeting System has the resolution and sensitivity to discover differences in protein complexes using exosomes secreted by cancer cells from the same tumor type.

"The results of this research are highly significant in that they show that the ADAPT system can be deployed against multiple cancer types in various biological matrices and offers broad potential applications in biomarker discovery," said David Spetzler, M.S., Ph.D., M.B.A., President and Chief Scientific Officer of Caris Life Sciences, and an author of the study. "Further, we were able to show that in prostate cancer, ADAPT not only discriminated between cancer types but between subtypes of a specific lineage. We anticipate that this could potentially help inform treatment decisions based on the patient's specific molecular profile in prostate cancer and across a range of tumor types."

"The differences in the composition of the Endosomal Sorting Complex Required For Transport (ESCRT) pathway and associated complexes between exosomes derived from VCaP and LNCaP cells could point to them as novel biomarkers for these different prostate cancers," said Michael Famulok, Ph.D., University of Bonn (Germany), Life & Medical Sciences Institute (LIMES), Max Planck Fellow and co-author of the study. "We look forward to further investigating this potential and how the ADAPT system can be used to gain a greater understanding of the molecular composition of cells across tumor types."

The ADAPT Biotargeting System is Caris' proprietary unbiased profiling platform that uses a broad library of synthetically-manufactured molecules (aptamers) that bind to a wide range of biological targets and characterize complex biological systems in their native state, enabling them to profile biological samples at a systems-wide scale.

Thepaperwaspublishedonline on January 28 inNucleic Acids Research,andisavailableonlinehere and DOI:https://doi.org/10.1093/nar/gkaa034.

About Caris Life Sciences

Caris Life Sciences is a leading innovator in molecular science focused on fulfilling the promise of precision medicine through quality and innovation. The company's suite of market-leading molecular profiling offerings assesses DNA, RNA and proteins to reveal a molecular blueprint that helps physicians and cancer patients make more precise and personalized treatment decisions.

Story continues

Caris is also advancing precision medicine with Next Generation Profiling that combines its innovative service offerings, Caris Molecular Intelligence and ADAPT Biotargeting System, with its proprietary artificial intelligence analytics engine, DEAN, to analyze the whole exome, whole transcriptome and complete cancer proteome. This information, coupled with mature clinical outcomes on thousands of patients, provides unmatched molecular solutions for patients, physicians, payers and biopharmaceutical organizations.

Whole transcriptome sequencing with MI Transcriptome provides the most comprehensive and unique RNA analysis available on the market and covers all 22,000 genes, with an average of 60 million reads per patient, to deliver extremely broad coverage and high resolution into the dynamic nature of the transcriptome. Assessing the whole transcriptome allows us to dig deeper into the RNA universe to uncover and detect fusions, splice variants, and expression changes that provide oncologists with more insight and actionable information when determining treatment plans for patients.

Caris Pharmatech, a pioneer of the original Just-In-Time research system with the largest research-ready oncology network is changing the paradigm from the traditional physician outreach model to a real-time approach where patient identification is completed at the lab and the physician is informed so that the patient can be enrolled days earlier, and remain in the local physician's care, without having to travel to a large central trial site. This fundamentally redefines how pharmaceutical and biotechnology companies identify and rapidly enroll patients in precision oncology trials by combining Caris' highest quality industry leading large-scale molecular profiling services with Pharmatech's on-demand site activation and patient enrollment system.

Headquartered in Irving, Texas, Caris Life Sciences offers services throughout the U.S., Europe, Asia and other international markets. To learn more, please visitwww.CarisLifeSciences.comor follow us on Twitter (@CarisLS).

Media Contact:Lindsey BailysGCI Healthlindsey.bailys@gcihealth.com +1-212-798-9884

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It’s all in the delivery nanoparticle platform could transform medical treatments – Princeton University

Optimeos Life Sciences, a startup founded by two Princeton University faculty members, has reached agreements with six pharmaceutical companies to develop therapeutics using a Princeton-developed drug delivery technology. The collaborations have the potential to improve the effectiveness of medications for the treatment of diseases, ranging from cancer to diabetes.

Optimeos, founded in 2016 by Robert Prudhomme, professor of chemical and biological engineering, and Shahram Hejazi, a faculty memberinthe Keller Center for Innovation in Engineering Educationandelectrical engineering,focuses on bringing technology developed over 15 years in Prudhommes lab to market. The technology, called flash nanoprecipitation, enables the encapsulation of drugs into nanoscale particles that improve delivery and effectiveness.

The startupOptimeos,founded by two Princeton faculty members, has agreements with pharmaceutical companies to bring a nanoscale drug delivery system to market. Developed in Robert Prudhommes lab, the system is a vehicle for delivering medications to precise locations in the body or the interior of cells to treat myriad diseases, including cancer and diabetes. Shown is an artists rendering of Optimeos nanoparticle system.

Illustration by Rachel Davidowitz

The new venture extends the impact of the technology, which already is being used in a project with the Bill and Melinda Gates Foundation. The foundation awarded Prud'homme'slab a $1.2 million grant in 2016 to apply their technology to increase the effectiveness of drugs used in global health. Solutions for global health problems have to be low cost and robust, and the flash nanoprecipitation process is both. The method has been applied to three drugs sponsored by the Gates Foundation. The first was for a drug to treat diarrhea in infants caused by drinking polluted water, the second a tuberculosis drug, and the third a single-dose treatment for malaria.

Optimeos new agreements involve creating improved delivery methods for six different medicines. The names of the six biopharmaceutical companies are currently undisclosed due to the proprietary nature of the ventures. The targeted indications for the various projects are immuno-oncology, autoimmune diseases, diabetes, diseases of the central nervous system and ocular diseases.

These new projects address an inverse set of constraints to that of the Gates Foundation. With Gates, the goal is to take drugs that normally dissolve poorly in the body, because they are water-resistant, and use the delivery system to increase absorption. These projects also need the formulations to be inexpensive, as well as impervious to high humidity and other extreme storage conditions. In contrast, the drug companies working with Optimeos need methods for delivering highly soluble biologics a class of therapeutics including proteins, peptides and nucleic acids.The structural complexity of biologics imparts higher potency and greater specificity, which causes fewer side effects. However, biologics require delivery through frequent injections, and their activity is restricted to targets outside the walls of individual cells, unless sophisticated pharmaceutical formulations are used.

The future of therapeutics are potent biological drugs, many of which have delivery challenges with respect to how much drug needs to be delivered to exactly where in the body, while minimizing side effects, Hejazi said.

Optimeos is overcoming the limitations of biologics by encapsulating them into carefully designed nanoparticles that are 10-times smaller than a red blood cell, or into larger microparticles about the width of a human hair. Such particles have been difficult to manufacture in a reproducible and scalable way, until now.

To make these particles, Optimeos first creates primary nanoparticles in which the drug-filled core is covered by a skin of specially designed polymers. These primary nanoparticles can then be coated with additional polymers that are engineered to interact with specific tissues or cells in the body. These coated nanoparticles could deliver drugs to more precise locations in the body or into the interior of cells. Alternatively, the primary nanoparticles can be assembled into larger composites, much like a cluster of grapes. These microparticles slowly release the encapsulated therapeutic, over a period of weeks to months.

One use of these slow-releasing particles is in the treatment and management of diabetes. In 2019, Optimeos received funding through a National Science Foundation grant to develop a once-monthly injection of liraglutide, a non-insulin medicine used to treat type II diabetes and obesity. Liraglutide is currently administered by a daily injection. The Optimeos formulation aims to reduce the total amount of drug needed, reduce side effects and reduce the frequency of injections. These attributes enhance patient comfort, adherence to treatment regimens, quality of life, cost of care, as well as the medical outcomes, said Robert Pagels, director of R&D for the company and former graduate student of Prudhomme.(As a student in 2017, Pagels' pitch of the technology won first place at the Keller Center's annual Innovation Forum.)

What makes Optimeos approach novel is that the particles can be easily scaled to accommodate mass production needed for the marketplace. I would say none of the technologies out there that are being published on or worked on can be, in fact, scaled up," said Hejazi.

As the onetime head of the Kodak Molecular Imaging Group, now Carestream, Hejazi knew that a persistent problem in the marketplace is that many innovations in nanotechnology cannot be reproduced consistently at industrial scales. Flash nanoprecipitation and its corollary, called inverse flash nanoprecipitation, solve that problem, Hejazi said.

Most methods of creating nanoparticles require combining active ingredients in precise proportion to each other to create consistently sized nanoparticles. This can often be done effectively in small volumes in the laboratory, but cannot be translated to large-scale production. To solve this challenge, flash nanoprecipitation uses multiple, continuous, high-velocity streams containing the active ingredients that constantly combine at the correct proportions within specially engineered mixing chambers. To create more nanoparticles, the process can be left to run over longer time periods. To increase speed of production, the thickness of the streams can be increased, as long as they are in correct proportion to each other. In effect, it is an assembly line approach to mixing the agents.

Pictured at Princeton Innovation Center BioLabs, team members from left: Shahram Hejazi, Bumjun Kim, Robert Pagels, Robert Prud'homme, Chester Markwalter and Madeleine Armstrong.

I have always been passionate about solving health care problems, said Hejazi. Through his industrial contacts, he identified problems the technology could potentially solve. Once the researchers demonstrated that the technology could meet industry demands, the team filed a patent for inverse flash nanoprecipitation in 2016. This patent, written by Pagels and Prudhomme, details how the flash nanoprecipitation process can be used to encapsulate water-soluble drugs such as biologics. This was a major innovation first developed by Pagels in his doctoral research. He flipped the flash nanoprecipitation process (hence the term inverse) to encapsulate soluble drugs, rather than water-insoluble drugs. This made the technology applicable to biologics, and opened up this fast-growing market as a potential target.

Hejazis experience in industry and venture capital provided the vision that drove the founding of the company. Hejazi helped to raise capital, and the team formed a sponsored research agreement with Princeton to continue developing the technology at Princeton. Optimeos also hired recent graduates from Prudhommes lab to work in the company.

Prudhomme sees Optimeos bringing his scientific goals to fruition. My academic research has focused on understanding the fundamental principles behind polymer assembly and processing to enable us to make elegant nanoparticles, said Prudhomme.However, as an engineer, I also want to do something that can make an impact on human health, as opposed to just trying to do a one-shot thing that is beautiful and advances science.

Optimeos story is part of a wider effort at Princeton to move discoveries from University labs to the market and to benefit society more broadly.

The Office of Technology and Licensing at Princeton educates researchers in the steps needed to commercialize technologies and offers entrepreneurial resources, said Anthony J. Williams, new ventures associate in the office.

In recent years, the technology licensing office at Princeton has doubled the creation of startups growing out of faculty research.

Princeton and other universities have been commercializing research since the 1980s when the Bayh-Dole Act allowed institutions to market research that was funded through federal grants. Since then, some of the biggest entrepreneurial success stories at Princeton have been the drug Alimta, a highly effective drug for certain lung cancers, and a breakthrough that tripled the effectiveness of organic LEDs (OLEDS), a display technology now widely used in flat-screen televisions and smartphones.

Princeton isnt an ivory tower," said Williams. Princeton is developing a lot of innovative technologies and a lot of great inventions that can do good in the world.

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It's all in the delivery nanoparticle platform could transform medical treatments - Princeton University

Myriad Genetics to Present at the SVB Leerink Global Healthcare Conference – Yahoo Finance

SALT LAKE CITY, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN), a leader in molecular diagnostics and personalized medicine, announced that R. Bryan Riggsbee, president and CEO, is scheduled to present at the SVB Leerink Global Healthcare Conference at 10:30 a.m. EST on February 25, 2020 in New York City.

The presentation will be available to interested parties through a live audio webcast accessible through a link in the investor information section of Myriads website at http://www.myriad.com.

About Myriad GeneticsMyriad Genetics, Inc., is a leading precision medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on five critical success factors: building upon a solid hereditary cancer foundation, growing new product volume, expanding reimbursement coverage for new products, increasing RNA kit revenue internationally and improving profitability with Elevate 2020. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, EndoPredict, Vectra, GeneSight, riskScore Prolaris, ForeSight and Prequel are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

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Myriad Genetics to Present at the SVB Leerink Global Healthcare Conference - Yahoo Finance

No. 474: On molecular diagnostics, robo-busts and T Rex and happy birthday to the author of your youth – Innovate Long Island

Back stretch: Welcome to Wednesday, dear readers, and the clubhouse turn of our latest socioeconomic sprint.

Have you seen me: National Lost Penny Day on Lincolns birthday? Makes cents.

Its Feb. 12 out there, replete with offbeat observations: choose from National Lost Penny Day, National Plum Pudding Day and Valentines Hug Day, a cuddly precursor to the big lovers day.

They do: Speaking of lovers, its also a notable date for same-sex unions National Freedom to Marry Day, which was actually started by a law firm but earns a shoutout here.

Georgia on our mind: To our many readers in The Peach State, a joyous Georgia Day, recalling the Feb. 12, 1733, establishment of the then-Colonial province.

Sparta something big: Happy anniversary also to Michigan State University, home of the mighty Spartans, founded on this date in 1855.

Face it: Turning to sports innovations, to the delight of receivers and the chagrin of dentists, the catchers mask was patented by inventor Frederick Thayer on Feb. 12, 1878.

And it was this date in 1879 when North Americas first artificial ice-skating rink opened inside Madison Square Garden.

But whos counting: Thomas Edison racked up nine total patents on Feb. 12 four in 1884, two in 1889 and three in 1895, including his incandescent electric lamp.

Other notable innovators earning patent protections on this date include automotive and electronics icon James Packard, who landed the very first of his 43 patents this covering an Igniting Device for Hydrocarbon Engines on Feb. 12, 1901.

Use it or lose it: Fiji was the first nation to ratify the historic Paris Agreement on climate change.

A change is gonna come: And it was this date in 2016 when Fiji became the first nation to ratify the Paris Agreement, an environmental accord under the United Nations Framework Convention on Climate Change.

One-hundred-ninety-four other countries including the United States, albeit temporarily would follow.

Honestly: Happy birthday, Abe an imperfect man with an unerring sense of justice, a leader during a time of unparalleled crisis and an enduring tribute to the solemn weight and responsibility of the U.S. presidency, Abraham Lincoln would be 211 years old today.

Also born on Feb. 12 were the king of origin stories, English naturalist Charles Darwin (1809-1882); American paleontologist Barnum Brown (1873-1963), who discovered the first Tyrannosaurus Rex fossils and evidence of many other dinosaur species; American physicist and Nobel laureate Julian Schwinger (1918-1994), who helped square quantum mechanics with Einsteins theory of relativity; and Swedish actress Maud Adams (born 1945), still the only performer to play two different Bond girls in the long-running spy series.

Its me, Judy: Blume has beaten back critics with numerous awards and 82 million copies sold.

Full Blume: And take a bow, Judy Blume the beloved (and sometimes controversial) author of honest, timeless and influential adolescent fiction turns 82 today.

Give your first favorite writer, your fathers 1974 fantasy woman and all the other Feb. 12 innovators your best at editor@innovateli.com give us the story tips and calendar items, please and thank you.

About our sponsor:Farrell Fritz, a full-service law firm with 15 practice groups, advises startups on entity formation, founder and shareholder agreements, funding, executive compensation and benefits, licensing and technology transfer, mergers and acquisitions and other strategic transactions.The firms blog,New York Venture Hub, discusses legal and business issues facing entrepreneurs and investors.

BUT FIRST, THIS

Park place: Albanys investments in New Yorks state parks appear to be paying off, with Gov. Andrew Cuomo trumpeting a banner year in 2019 for statewide park attendance including turnout at several recently refurbished Long Island sites.

New Yorks state parks, historic sites, campgrounds and officially sanctioned hiking trails welcomed a record-breaking 77.1 million visitors in 2019, an estimated 4 percent increase (nearly 3 million visitors) from 2018 numbers, according to the governors office, which also counts a roughly 33 percent jump (about 19 million annual visits) since Cuomo took office in 2011.

Cuomo credited efforts like the $90 million NY Parks 2020 plan, which has brought facility upgrades to Bethpage State Park (where annual attendance grew by 18 percent in 2019), Sunken Meadow State Park (annual attendance up 13 percent) and a host of other state properties on and off Long Island. The NY Parks 2020 initiative has revitalized our state parks and historic sites, Cuomo said Tuesday. Tourism is booming in New York and these beautiful sites are drawing visitors to all corners of the state.

Jeff Boyd: Molecule man.

Atta Boyd: A renowned expert in cancer genetics and clinical molecular diagnostics has joined the Northwell Health Cancer Institute.

Boasting three decades-plus of cancer-research experience, Jeff Boyd has been named vice president and chief scientific officer of the Cancer Institute and director of its Center for Genomic Medicine. Boyd, who most recently served as associate deputy director of the Miami Cancer Institute at Baptist Health South Florida, also will become a professor at Cold Spring Harbor Laboratory and member of the CSHL Cancer Center.

His primary function at the Northwell Health Cancer Institute will be heading up a new, state-of-the-art Molecular Diagnostics Laboratory the perfect challenge for the veteran researcher, according to Northwell Health Senior VP and Cancer Institute Director Richard Barakat. Genomics has become an important piece to helping us understand the genetic roots of the various forms of cancer, Barakat said Tuesday. We are extremely fortunate to have an internationally known genomics expert such as Dr. Boyd join the Cancer Institutes leadership team.

TOP OF THE SITE

Made for walking: And thats just what this multifaceted boot camp will do, not only teaching learners to code but putting them on the path to 21st century employment.

Robo cop: If Long Island-based autodialer assassin Nomorobo was any closer to a new federal crackdown on illegal robocalls, it would need a badge.

Growing Hain: Discontinued operations are still a drag, but the bottom line is slowly brightening for Lake Success-based global distributor Hain Celestial.

VOICES

Going to extremes: Students on the educational extremes special-ed students needing the most help, high-achieving aces needing more challenges have it roughest in distant rural districts. Voices columnist and K-12 education ace Harry Aurora wants to bring the solution to them.

STUFF WERE READING

Start your engines: Forbes takes a spin on the future of automotive innovation.

Water works: Engadget dives into rainwater as a renewable energy source.

Weather (or not): Popular Mechanics reviews 150 years of forecasting innovations.

RECENT FUNDINGS

+ Outset Medical, a California-based medtech developing innovative hemodialysis systems, raised $125 million in Series E equity financing led by D1 Capital Partners, with participation from Fidelity Management and Research Company, Partner Fund Management, Perceptive Advisor and funds advised by T. Rowe Price Associates.

+ Accion Systems, a Massachusetts-based in-space propulsion system provider, raised $11 million in Series B funding co-led by Boeing HorizonX Ventures and Shasta Ventures.

+ Sonoma Biotherapeutics, a California- and Washington State-based company developing regulatory T cell therapies for autoimmune and degenerative diseases, raised $40 million in Series A financing. Backers included Lyell Immunopharma, ARCH Venture Partners, Milky Way Ventures and 8VC.

+ Houwzer, a Pennsylvania-based real estate brokerage, raised $9.5 million in Series A funding led by Edison Partners, with participation from Admiral Capital Group and real estate investor Ira Lubert, and new investors GO Philly Fund and Chestnut Street Ventures.

+ Lumos Diagnostics, a Florida-based healthcare company providing complete point-of-care diagnostic test solutions, closed a $15 million Series A funding round. Australian commercialization company Planet Innovation made the investment.

+ Suzy, a New York City-based consumer-insight platform, closed a $12 million Series C funding round led by Bertelsmann Digital Media Investments, with participation from Foundry Group and Triangle Peak Partners, and existing investors Tribeca Venture Partners and 35 Ventures.

BELOW THE FOLD

Rocks solid: Behold, the new whiskey rebellion.

Eat: The Mars menu may convince astronauts to stay home.

Drink: What whiskey can teach us about innovation.

Be merry: Does innovation create happiness, or vice versa? You decide.

For tomorrow we fly: With the help of the Regulatory & Government Relations Practice Group and all the other topflight resources waiting at Farrell Fritz, one of the amazing firms that support Innovate LI. Check them out.

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No. 474: On molecular diagnostics, robo-busts and T Rex and happy birthday to the author of your youth - Innovate Long Island

$3.75m awarded to transform the way ovarian cancer is treated – News – The University of Sydney

Cancer Council has awarded its annual $3.75 million Translational Program Grant to a research team led by Professor Anna deFazio from the University of Sydney, exploring how to better personalise ovarian cancer treatment for Australian women.

Professor Anna deFazio and her team will spend the next five years creating a process by which ovarian cancer patients can have their cancer comprehensively analysed to determine its molecular profile. If standard treatments are not effective, patients can be matched with an appropriate clinical trial, based on the individual characteristics of their tumour.

This will improve treatment outcomes by ensuring that treatment approaches offered to an ovarian cancer patient are those with the highest likelihood of being successful for their specific subtype of the disease.

Professor deFazio, who is the Sydney West Chair in Translational Cancer Research and co-director of the Centre for Cancer Research at the Westmead Institute for Medical Research (WIMR), is hopeful that this research will transform the way women with ovarian cancer are treated.

Many people dont know that ovarian cancer has the lowest survival rate of any womens cancer in Australia, said Professor deFazio, from the University of Sydney School of Medicine in the Faculty of Medicine and Health.

Currently, only around 45 percent of ovarian cancer patients will survive for five years. Unlike many other cancers, these survival odds have only slightly improved in the last two decades one of the main reasons for this is that each ovarian cancer differs significantly in its genetic and molecular make-up, which results in widely varied treatment outcomes.

Encouragingly, the last few years have seen a rapid expansion in the number and variety of targeted cancer treatment options."

We will work to provide the missing link in this treatment path, generating the data and processes to match patients with the ideal treatment for their cancer type, said Professor deFazio.

The University of Sydney-led team has assembled an expert team of researchers and clinicians to make this happen, including from WIMR, The Crown Princess Mary Cancer Centre, Westmead Hospital, the Prince of Wales Hospital, Royal Hospital for Women, Royal North Shore Hospital, Royal Prince Alfred Hospital, Chris OBrien Lifehouse, the University of Technology Sydney, the Childrens Medical Research Institute, and from interstate, the Peter MacCallum Cancer Centre, and overseas, from the National Cancer Institute in the United States.

To reach their goal, Professor deFazios team will analyse the molecular profile of more than 300 ovarian cancer patients in NSW to better understand the subsets of ovarian cancer and how each type responds to specific treatments. The team will also look at ways of more simply communicating complex molecular test results to the treating clinical team, so they are easier to interpret.

Finally, Professor deFazios team will use patient samples to print 3D models of ovarian tumours to test treatment approaches and help design new early phase clinical trials.

Professor Karen Canfell, Director of Research at Cancer Council NSW, is proud the organisation was able to fund such innovative research: We only award our Translational Program Grant to cancer research that will rapidly translate research discoveries into clinical practice and policy, she said.

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$3.75m awarded to transform the way ovarian cancer is treated - News - The University of Sydney

Discovery raises Prospect of Universal Cancer Therapy – Labmate Online

The use of T-cell therapies for cancer - where immune cells are removed, modified and returned to the patients blood to seek and destroy cancer cells includes the use of CAR-T treatments which are personalised to each patient; however, this is said to target only a few types of cancers and has not been successful for solid tumours, which make up the vast majority of cancers.

Researchers at Cardiff University have now discovered T-cells equipped with a new type of T-cell receptor (TCR) which identifies and kills most human cancer types.

Conventional T-cells can recognise small parts of cellular proteins bound to cell-surface molecules called human leukocyte antigen (HLA), allowing killer T-cells to see whats occurring inside the cells by scanning their surface. HLA varies widely between individuals which has created barriers to creation of a T-cell-based treatment that can target most cancers in all people.

Now the findings of the Cardiff study(1) describe a unique TCR that can recognise many types of cancer via a single HLA-like molecule called MR1. Unlike HLA, MR1 does not vary in the human population - meaning it is a hugely attractive new target for immunotherapies.

T-cells equipped with the new TCR were shown, in the lab, to kill lung, skin, blood, colon, breast, bone, prostate, ovarian, kidney and cervical cancer cells, while ignoring healthy cells.

Professor Andrew Sewell, lead author of the study and an expert in T-cells from Cardiff Universitys School of Medicine, said: Cancer-targeting via MR1-restricted T-cells is an exciting new frontier - it raises the prospect of a one-size-fits-all cancer treatment; a single type of T-cell that could be capable of destroying many different types of cancers across the population. Previously nobody believed this could be possible.

Experiments are under way to determine the precise molecular mechanism by which the new TCR distinguishes between healthy cells and cancer. The researchers believe it may work by sensing changes in cellular metabolism which causes different metabolic intermediates to be presented at the cancer cell surface by MR1.

The Cardiff group hope to trial this new approach in patients towards the end of this year following further safety testing. Professor Sewell said a vital aspect of this ongoing safety testing was to further ensure killer T-cells modified with the new TCR recognise cancer cells only.

"There are plenty of hurdles to overcome; however if this testing is successful, then I would hope this new treatment could be in use in patients in a few years time, he said.

Further information at http://www.cardiff.ac.uk

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Discovery raises Prospect of Universal Cancer Therapy - Labmate Online

Scientists Study Sweat, the Small Stuff – UANews

Imagine if you could know the status of any molecule in your body without needing to get your blood drawn. Science fiction? Almost but researchers at the University of Arizona are working on ways to do this by measuring molecules in sweat.

When physicians take blood samples from patients, they send the samples to labs to be analyzed for biomarkers. These biological clues indicate everything from cholesterol levels to disease risks, and they can be used to monitor patient health or make diagnostic decisions. The same biomarkers also are found in sweat.

Using $519,000 in funding from the SEMI Nano-Bio Materials Consortium, or SEMI-NBMC, Erin Ratcliff, a materials science and engineering professor and head of the UArizona Laboratory for Interface Science of Printable Electronic Materials, is leading a project to develop new ways of collecting and analyzing the clues sweat has to offer. Ultimately, this work could allow physicians to use patient sweat samples in the same way they currently use blood samples, for a less invasive and more informative approach to establishing and monitoring patient health.

Whats unique about this is that we are combining biology and engineering expertise to develop a wearable device that will detect molecules in sweat, so you dont have to get your blood drawn to know the health status of your immune system, your nervous system, indeed, any system in the body, said co-investigator and sweat biomarker pioneer Esther Sternberg, M.D. The goal, eventually, is to create a device that will provide physicians and health care providers the ability to monitor your health status continuously and in real-time without needing to draw blood.

We are pleased to sponsor and eager to complete this project with the University of Arizonas impressive team bridging the disciplines of engineering and life sciences, said Melissa Grupen-Shemansky, chief technology officer and executive director of SEMI-NBMC. A concerted interdisciplinary approach at the early stages of R & D is relatively new, and there is much learning on both sides. The University of Arizona team brings unique strengths in both areas, and we are excited to be partnering and collaborating with them.

Ratcliffs co-investigators are J. Ray Runyon, a research assistant professor in the Department of Environmental Science, and Sternberg, research director for the Andrew Weil Center for Integrative Medicine; director of the Institute on Place, Wellbeing, and Performance; and the Andrew Weil Inaugural Chair for Research in Integrative Medicine. All three researchers are members of the BIO5 Institute.

Standardized Sample Collection

In order to study sweat, researchers need to collect samples of it, and there are a number of ways to do so.

The obvious idea would be to make a patch that gets information from many pores at once, but the problem is that this creates a space between the patch and your skin, and you have to wait for it to fill up with sweat, Ratcliff said. We hypothesize that while youre waiting, these molecules the very molecules youre trying to detect and analyze are changing chemically.

The teamsfirst task is to develop new, continuous and hands-free collection devices that deliver high-quality, standardized sweat samples. This will allow health care professionals to gain a more holistic picture of a patient's bodily systems over an extended period, rather than the snapshot a blood draw can provide of a particular moment.

Currently, sweat labs across the world are using different methods to collect samples, which limits researchers ability to compare data. Standardizing the collection method could provide researchers, including medical device developers, with a new degree of confidence in sweat sample data.

High-quality data, with respect to different target molecular biomarkers in sweat, requires that a high-quality sample be collected, Runyon said. This will be the first hands-free method that will truly take into account the interplay of the chemistry of sweat, the target biomarker and the device material.

Low-level Detection

The team is also developing methods for researchers to detect and analyze neuropeptides in the collected samples. Used by neurons to communicate with each other, these small molecules are involved in biological functions, including metabolism, reproduction and memory.

Commercial wearable devices monitor metrics like heart rate, and some use sweat sensors to monitor dehydration level. Measuring neuropeptides, however, will allow researchers to zoom in millions of times closer to investigate stress and relaxation responses at the molecular level.

The idea is that your sweat is reflecting your nervous system all of the neurotransmitters your body uses to signal between the brain and the rest of the body, Ratcliff said. Monitoring this biochemical response continually, over a 24-hour cycle, can inform us about the health of the wearer and also act as a diagnostic tool.

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Nuclear Medicine Imaging Market To Reach USD 5.13 Billion By 2027 | Reports And Data – Yahoo Finance

Nuclear Medicine Imaging Market Size USD 3.36 billion in 2018, CAGR of 4.7%, Nuclear Medicine Imaging Industry Trends Rising occurrences of cancer

New York, Feb. 11, 2020 (GLOBE NEWSWIRE) -- The global nuclear medicine imaging market is expected to reach USD 5.13 Billion by 2027, according to a new report by Reports and Data. The growth of the nuclear medicine imaging market is primarily owing to the increasing prevalence of target diseases.

Cancer has a significant impact on the society in the U.S. and across the globe. It has been estimated that 1,735,350 new cancer cases were diagnosed in the U.S. and 609,640 people suffered death from the disease. The number of new incidences of cancer is 439.2 per 100,000 men and women annually and the number of cancer-related morbidity is 163.5 per 100,000 men and women annually. Moreover, it has been estimated that in 2017, 15,270 children and adolescents in the age range of 0-19 years were diagnosed with cancer out of which 1,790 suffered death owing to the disease. In the year 2017, an estimated USD 147.30 billion was spent on cancer care in the U.S.

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There is an increasing demand for detecting subclinical disease that require the development of imaging procedures that can precisely locate diseased tissue whereby it can be treated with a minimally invasive and image-guided method. The molecular imaging, which is a type of medical imaging that offers detailed pictures of happenings inside the body at the molecular and cellular level, is one of the most lucrative areas in the field of imaging technology. Nuclear medicine imaging finds application in the field of molecular targeting, which in turn, is likely to propel the market growth in the future.

Further key findings from the report suggest:

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For the purpose of this report, Reports and Data have segmented the global nuclear medicine imaging market on the basis of type, application, end-users, and region:

Type Cause Outlook (Revenue, USD Million; 2016-2027)

ApplicationOutlook (Revenue, USD Million; 2016-2027)

End-UsersOutlook (Revenue, USD Million; 2016-2027)

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Market segment by Region/Country include:

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Precision Medicine Leaps Ahead with Data Science: Bio-IT World 2020 to Highlight Data-Driven Approaches to Discovery – BioSpace

More than 3,200 such experts from pharmaceutical, biotech, healthcare and technology organizations, government and academia will converge for the 19th annual event, taking place April 21-23, 2020 in Boston.

Data science is foundational to life science companieschanging the competencies required to compete. The opportunities to push our industry to the edge of discovery are thrilling for those organizations that embrace a collaborative, data-driven approach, said Allison Proffitt, Editorial Director, Bio-IT World.

From a plenary keynote presentation on the National Institutes of Healths (NIH) Strategic Vision for Data Science, to a new Data Science and Analytics Technologies track with Bristol-Myers Squibb, Takeda and more, the conference will highlight effective strategies, analytics and tools.

Altogether, 16 conference tracks will cover AI for Drug Discovery, Bioinformatics, Data Storage and Transport, Pharmaceutical R&D Informatics, Cancer Informatics, Genome Informatics, Clinical Research and Translational Informatics, Data and Metadata Management, Data Visualization Tools, Emerging AI Technologies, AI: Business Value Outcomes, Software Applications and Services, Data Security and Compliance, Cloud Computing, and Open Access and Collaborations, in addition to Data Science and Analytics Technologies.

Other highlights include 250+ presentations, 160+ exhibitors, Plenary Keynotes, pre-conference workshops, a Hackathon, awards, poster sessions and networking. See details at bio-itworldexpo.com.

About Cambridge Healthtech Institute Cambridge Healthtech Institute (CHI), a division of Cambridge Innovation Institute, is the preeminent life science network for leading researchers and business experts from top pharmaceutical, biotech, CROs, academia, and niche service providers. CHI is renowned for its vast conference portfolio held worldwide including PepTalk, Molecular Medicine Tri-Conference, SCOPE Summit, Bio-IT World Conference & Expo, PEGS Summit, Drug Discovery Chemistry, Biomarker World Congress, World Pharma Week, The Bioprocessing Summit, Next Generation Dx Summit, Immuno-Oncology Summit, and Discovery on Target. CHI's portfolio of products include Cambridge Healthtech Institute Conferences, Barnett International, Insight Pharma Reports, Bio-IT World, Clinical Research News and Diagnostics World.

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Precision Medicine Leaps Ahead with Data Science: Bio-IT World 2020 to Highlight Data-Driven Approaches to Discovery - BioSpace

There could be a link between marijuana use and heart health – Medical News Today

New research found that 2.3% of reported marijuana users also have some form of cardiovascular disease.

The study appeared in the Journal of the American College of Cardiology.

The paper also looked at the possible interactions marijuana may have with drugs typically prescribed for cardiovascular issues, and the way marijuana chemicals may affect the heart at a molecular level.

According to the Centres for Disease Control and Prevention (CDC), heart disease a type of cardiovascular disease (CVD) is the leading cause of death in the United States.

In 2015, heart disease caused 23.4 % of all deaths in the U.S.

According to the American Heart Association (AHA), nearly half of all U.S. adults have some form of CVD. They cite an article in the journal Circulation that sets this figure at 121.5 million U.S. adults, which is approximately 48% of the population.

In contrast, the reported 2.3% of reported marijuana users who also have CVD is relatively low compared to the broader U.S. population.

This does not mean that marijuana use and CVD have no links. However, there is insufficient evidence to prove any connection, and what is available is of poor quality.

In the U.S., there are significant restrictions on researching marijuana. This means that scientists cannot carry out randomized clinical trials to understand its effects on health.

Researchers typically focus their research on observational studies, and the authors of the study claim that these show a possible link between marijuana use and cardiovascular events.

However, according to an article in the journal Annals of Internal Medicine, observational studies that have taken place are either inconclusive or have problems with their methodologies. This means that it is difficult for scientists to draw any conclusions from them.

The authors of the article in the Journal of the American College of Cardiology looked at data from the National Health and Nutrition Examination Survey (NHANES) to estimate how many marijuana users have CVD.

They found that 2 million of the 89.6 million U.S. adults who reported using marijuana also had CVD. This equates to 2.3% of reported marijuana users.

Dr. Ersilia M. DeFilippis, a former internal medicine resident at Brigham and Womens Hospital, Boston, MA, is the lead author. She is now a cardiology fellow at Columbia University Irving Medical Center.

She says, Marijuana use, both recreational and medical, is increasing nationally, yet many of its cardiovascular effects remain poorly understood. In our NHANES query, we estimated that 2 million adults with marijuana use had CVD in 20152016.

Since that time, additional states have passed legislature related to marijuana, so its use may have increased even further.

Notably, many of our cardiology patients are on medications that can interact with marijuana in unpredictable ways, depending on the formulation. This highlights that we need more data so that we can better counsel providers as well as patients.

Given the increase in marijuana use in the U.S., the authors of the study urge doctors to ask patients whether they use marijuana and to review their medication if necessary.

Current evidence is limited, and what little there is does not suggest a clear link between marijuana use and cardiovascular events. Researchers need to do more high quality research to determine precisely how and why marijuana affects cardiovascular health if it does at all.

According to the corresponding author Dr. Muthiah Vaduganathan, who is a cardiologist at Brigham, In the clinic, patients often ask us about the safety of marijuana use, and were pressed to offer the best scientific evidence.

Our current approach is that patients who are at high risk of cardiovascular events should be counseled to avoid or at least minimize marijuana use and that rigorous scientific research should be conducted to further inform recommendations for patient care.

Dr. Muthiah Vaduganathan

Before this can happen, however, authorities will need to lift the restrictions on marijuana research.

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Urgent Care, Telemedicine – Ways Going to the Doctor Is Changing – Men’s Health

Any doctor will tell you that every guy needs a primary-care providera trusted physician who knows you, knows that your dad had a heart attack at age 40, helps you figure out how to tweak your life according to your health historyand heck, knows your name without looking at your chart first.

Except that for most of us, thats not how it goes. First, the doctor never sees us, because the yearly visit has been called off. Theres no reason for healthy men under 40, or anyone for that matter, to get annual checkups, says Eric Topol, M.D., a professor of molecular medicine at Scripps Research in La Jolla, California, echoing the view of the Society of General Internal Medicine. Having an annual exam does not reduce your risk of death or serious disease, according to a review of 14 studies. When it comes to yearly checkups, all the data shows the lack of value, and they should be strongly discouraged, Dr. Topol says.

Second, we move around. If you take a 21-year-old male in 2020 and ask, Where are you going to get care today versus five, ten, or 20 years from now? hes not going to know, because people change employers, health plans, and even doctors relatively frequently, says Ateev Mehrotra, M.D., an associate professor of public health-care policy and medicine at Harvard Medical School. So the question is: Is that relationship even possible?

Add to that issues with cost and convenience and its no wonder nearly half of adults in the U.S. under age 30 dont have a primary-care physician.

Andtwist you didnt see coming!maybe thats okay.

There are now many cheaper, more convenient ways to get medical help than waiting for a traditional doctors office to fit you in and potentially having to travel to get there. And if youre basically healthy, you might do fine with these.

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But with choice and convenience comes great responsibility. Most millennials use urgent care, go online, talk to their friends, and think thats health care, says Allan H. Gorroll, M.D., a professor of medicine at Harvard Medical School. Its not. A catch-as-catch-can health plan leads to a lot of issues not getting caught. With this approach, some things may be overlooked or prescribed inappropriately, he says.

So youve got to have a plan. Not all of these answers to medical-system frustration are equal. Some are welcome, some dodgy, some outright dangerous, and some ingenious.

Look, if you have a doctor you love, stick with them. And if you have a chronic illness like diabetes or high blood sugar thats about to turn into diabetes, or if youre at high risk for anything else (like heart disease), then yes, youre going to need a main doctor.

But today, the old way is only one way to get what you need. If youre going to bypass a PCP, then let us show you how to navigate whats out there so that you find the best route to the best care.

Heres the lowdown on which new ways to see the doctor spare you time, money, and scheduling hassles without endangering your health.

You get to talk to or videoconference with a real doctor without making an appointment or even leaving your living room/car/office/campsite. Some big insurance companies have partnered with big telehealth start-ups, including Teladoc, Doctor On Demand, and American Well, so these options may be part of your plan. In 2018, an estimated 7 million people visited a doc this way.

You can find and use a telehealth site or app yourself, but youll have to do some homework on your choices before dialing.

The pros: Speed. Ease. Since youre calling or video chatting about a specific problem, such as a sore throat, youll likely be spared the you need to lose weight speech. And research has shown that people may be more honest in revealing symptoms or undesirable behaviors like alcohol consumption when disclosing to a computer.

The cons: Not all telehealth providers are equalsome have been scammy. So when choosing an app, look for one partnered with a known health-care entity, like Blue Cross Blue Shield or a hospital that you trust. Then see who the doctors aresome apps list providers names and contact information, so you can check them out online.

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More than half of all millennials seek help from urgent care (like Concentra, CityMD, and MedExpress) or retail health clinics, found in drugstores, supermarkets, or big box stores. Retail clinics are typically manned by a nurse practitioner or physicians assistant; theyre good at managing minor scrapes, colds, and fevers, and they can give you a flu shot. But they cant X-ray you or stitch you up. Freestanding urgent-care centers are for pressing but non-life-threatening medical issues (so bypass them for an ER if you have chest pain) and are generally staffed by a licensed physician.

The pros: Theyre cheaper and faster than the ER, they can usually send a report to you to keep on file (and to a doctor, if you have one), and theyre everywhere.

The cons: They may overprescribe meds. One recent study discovered that nearly half of people who dropped into urgent care with an ear infection, flu, allergies, asthma, bronchitis, or another viral upper-respiratory infectionnone of which respond to antibioticsgot a prescription. Retail clinics had the best track recordantibiotics were inappropriately prescribed only 14 percent of the time there (and 17 percent of the time at a doctors office).

If you need a prescription for an issue like ED, genital herpes, performance anxiety, or hair loss, a few finger taps can get you one from a digital health clinic such as Hims.

The pros: No embarrassing in-person doctor visit needed. Fill out an online formyoull answer a lot of questions about your issue and your health history (youve got to be honest)to determine if youre eligible for the prescription youre after. If a doc has questions or concerns, they may call you. Your medication arrives at your door a few days later.

The cons: The idea of getting meds with little human contact ruffles the feathers of practically every careful human prescriber we know. But it might not be as sketchy as all that: Dr. Mehrotra sent college students undercover to test online prescription apps and sites. My initial skepticism was tempered, he says. Thats still not a ringing endorsement, by the way.

These fancy doctors offices/wellness centers are about as far as you can get from low-contact telehealth apps. Packages vary by company, but monthly fees can get you a round of initial diagnostics and a certain number of appointments with those physicians every year. The promise is that these docs have extra time to spend with you not only to solve your health problem but also to help determine lifestyle changes to keep it in check and prevent other issues. At a company called Forward, for instance, members receive genetic testing, body scans, and a personalized health plan. It considers itself a supplement to your insurance plan, not a substitute for it.

The pros: By virtue of the fact that theyre not bound to a 15-minute reimbursable visit, these doctors can take the time to understand patterns and concerns that arent just todays problem. They also help with stress reduction and recommend healthy eating and exercise programs.

The cons: These physicians can feel like partners in your care, but theyre pricey. Forward runs about $149 a month, and Parsley Health costs about $150 to $250 a month, depending on how many visits a year you want. And the testing and blood work may not be included in the membership fees.

Different wearables can track your heart rate, heart rhythms, blood pressure, and sleep patterns. They also have a hint of Orwellian, Black Mirror creepiness, as some life-insurance companies have started using them to assess an individuals activity level and grant discounts on premiums based on lifestyle. So make sure youre aware of whos watching your watch.

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The pros: Your wearable may alert you to a heart problem (the Apple Watch Heart Rate app). And the Weill Cornell Alzheimers Prevention Clinic uses the Whoops sleep-activity data to track whether people experience a decrease in REM sleep, a potential risk factor for Alzheimers.

The cons: Wearables can be helpful for monitoring areas of concern, like not getting adequate sleep, but they cant do a doctors work yet. Plus, watch-based EKGs may deliver endless data outputs that send guys in for hours of fruitless worry and testing.

By the start of 2019, more than 26 million Americans had spat into a tube and sent a DNA sample off to a lab to check genetic health risks and ancestry.

The pros: Knowing that your DNA indicates youre at increased risk for certain diseases, including breast cancer (yes, even men can get it), age-related macular degeneration, and blood-clotting disorders, can help you step up efforts to prevent them.

The cons: It can be jarring to receive the news that youre at risk for a disease via a report, and you need to work with a doctor to retest to see if the diagnosis is accuratethen find if what youre doing to ward off a disease is adequate.

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Understanding this rare disease could help treat aspects of aging – Ottawa Citizen

Young Indian progeria sufferer Rupesh (C) sits with his mother Shanti Devi (L) and younger brother Vakil (R) at Allahabad District Magistrates Office in Allahabad on May 2, 2017, as they seek government financial support for his treatment. 21 year old Rupesh who comes from a village on the outskirts of Allahabad suffers from progeria disease, also known as Hutchinson-Gilford progeria syndrome (HGPS), which causes premature aging as a result of a genetic condition it affects some 1 in every 4 million births across the world.SANJAY KANOJIA / AFP via Getty Images

Progeria, also known as Hutchinson-Gilford Progeria syndrome, is a very rare disease, representing about one in six million births.

Progeria is aging on steroids. Babies with the syndrome are born without complications, but within a year or two, theyre not growing normally. They lose their hair and develop wrinkles. Most patients die before they leave their teens of heart attacks or stroke.

It would appear that understanding progeria and figuring out how to slow down the clock for progeria patients could be a key to treating aging itself. But while progeria has offered researchers a number of insights, the parallels between progeria and aging are not exact, said William Stanford, a senior scientist in regenerative medicine at the Ottawa Hospital Research Institute and a professor of cellular and molecular medicine at the University of Ottawa.

For example, plaque build-ups in the arteries of progeria patients in their teens look like those of a heart patient in their 90s. Progeria patients produce an abnormal form of a structural protein expressed in all cells. This abnormal protein known as progerin is also found in almost everyone in their 70s and 80s.

There are other differences between progeria patients and aging people. People with progeria dont show the same kidney and liver decline as older people, for example.

Research by Stanford and his colleagues has found it is possible to reprogram the progeria cells and essentially reset the clock. However, while this works in a culture dish, it has not progressed to animal studies, let alone human trials. A drug can slow down the process of aging on a cellular level, but it corrects only some of the cellular defects, not all.

The way I see it, theres a whole laundry list of things that go wrong with aging, said Stanford.

In the past decade, a cocktail of three different drugs that reduces Progerin accumulation combined with better medical care has helped extend the lives of progeria patients by at least two years. But treating ordinary people with the same drugs wont extend their lives.

Some of the insights gained by studying progeria may have benefits for treating heart disease in the general population, and thats a good thing, considering that heart disease is one the top causes of death in Canada, said Standford. But that knowledge may not extend to treating other age-related illnesses.

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UVA Honors Its Leading Researchers at Boar’s Head Banquet – University of Virginia

The University of Virginias top leaders gathered Wednesday evening at the Boars Head Resort to honor faculty members from across Grounds for their outstanding contributions to their fields of study and societal impact through their research and scholarly activities.

University of Virginia President Jim Ryan presented the 2019 Research Achievement Awards to 13 UVA faculty members at the dinner event.

This is the first year of the Research Achievement Awards, Vice President for Research Melur Ram Ramasubramanian said. We believe that as a university, we are what we celebrate. We want to acknowledge the talented UVA faculty who are leaders in their fields and are impacting the world in positive ways.

Provost Elizabeth Liz Magill said, Were delighted to have a chance to celebrate the accomplishments and achievements of our faculty. From education policy to precision medicine to police-community relations, there are many different fields and individuals being honored by these awards.

Im awed and immensely grateful for the contributions the award winners have made to their respective fields and to the University of Virginia, Ryan said. Our strategic plan focuses a good deal of attention on supporting research. ... Our ultimate goal is to make it possible for researchers at UVA to do their very best work.

The awards covered excellence in research, collaboration, mentorship, public impact and innovation.

Pompano arrived at UVA in 2014 and assembled a robust research team in her lab. Pursuing new technologies and new questions, she is developing new approaches to study immunity. In the areas of immunoanalysis and immunoenineering, she is working to map out cellular activity in live tissues. Her group was recently awarded a large grant from the National Institutes of Health to develop an artificial lymph node on a microfluidic chip.

Dr. Pompano chose the road less travelled by pursuing entirely new technologies and questions, rather than the safer route of building on the experiences from her Ph.D. or postdoc work, Jill Venton, chair of the Department of Chemistry, said. This strategy required spending the first 2.5 years of her professorship laying new groundwork. Dr. Pompano is a research leader in the fields of analytical chemistry and immunoengineering.

Bassoks work is in early childhood education, and her focus has been to find a way for it to both meet high standards and make a difference in the lives of young children. To do this, she has partnered with policymakers and school districts in Virginia and Louisiana to study how early childhood education opportunities can happen at scale.

In the past four years, her work has accelerated. She has published 16 articles and received more than $6 million in grant funding. In 2017, Bassok was honored with the Presidential Early Career Award for Scientists and Engineers.

Daphna Bassok has raised the bar for the field and will motivate other scholars to do more insightful and rigorous work, said Katherine Magnuson, director of the University of Wisconsins Institute for Research on Poverty.

Alhusens research focuses on improving maternal and early infant health outcomes for disabled women and women living in poverty. Her research has been funded by the National Institutes of Health, the Health Resources and Services Administration and numerous foundations, and the goal of her work is to provide higher quality care to vulnerable populations.

She has received numerous awards for her work, including the Southern Nursing Research Society Early Science Investigator Award; the Association of Womens Health, Obstetric, and Neonatal Nurses Award for Excellence in Research; and School of Nursings Faculty Research Mentor Award.

Walsh is Lockhart B. McGuire Professor of Internal Medicine and directs the School of Medicines Hematovascular Biology Center. His research is focused on clonal hematopoiesis of indeterminate potential, or CHIP.

In his lab, he is looking at how mutations in blood cells lead to chronic diseases like heart attack and stroke. Through precision medicine, he is identifying and combatting the out-of-control multiplying process in these mutations to fight age-related diseases, as well as blood cancers like leukemia.

Walsh has published more than 350 scientific articles and he is the recipient of multiple research grants from the National Institutes of Health, including a MERIT Award. In 2011, the American Heart Association designated him a Distinguished Scientist by for his contributions to cardiovascular research.

Throughout his career, Scullys research, scholarship and teaching have focused on the science of how corrosion occurs and the engineering required to prevent it. He has conducted research and collaborated with scientists around the world in numerous industries such as energy, transportation, infrastructure, aerospace, maritime and microelectronics.

His projects include two U.S. Department of Energy Energy Frontier Research centers, two Department of Defense multi-university research initiatives, as well as grants from the National Science Foundation, PPG Industries and Axalta (formerly DuPont), and the U.S. Office of the Undersecretary of Defense.

Scully, the Charles Henderson Chaired Professor and chair of the Department of Materials Science and Engineering, also co-directs the Center for Electrochemical Science and Engineering, one of the leading centers in the world focusing on materials degradation. The center has generated more than $30 million in research funding in the last 10 years and graduates on average four to five Ph.D. students per year.

Scully is technical editor in chief of CORROSION, The Journal of Science and Engineering, the premier international research journal for the field. He serves in several capacities as an ambassador for the materials-corrosion field, including several meetings to debrief the U.S. Congress on materials degradation issues of national importance.

John Scullys contributions to corrosion can be characterized by quality, quantity and longevity, said Gerald S. Frankel, Ohio State University distinguished professor in materials science and engineering and a member of CORROSIONs editorial board. It is clear that he is a world leader, if not the world leader, in metal passivity, passivity breakdown and localized corrosion, and stress corrosion cracking.

In more his more than 20 years at UVA, Lambert has advanced the science of risk analysis and systems engineering. He has led more than 60 projects related to advanced logistics systems for a total of approximately $25 million in research funding.

Lambert, a professor in the Department of Engineering Systems and Environment, has focused on the disruption of system priorities by emergent and future conditions, including technologies, regulations, markets, environments, behaviors and missions. His work has been applied to disaster resilience, energy infrastructure, coastal protection, economic development, transportation, biofuels and Olympics planning, among other challenges.

His research has been cited more than 5,000 times across more than 200 publications. In 2019, he chaired the Fifth World Congress on Risk, convening more than 300 scientists in Cape Town, South Africa.

Professor Lambert is among the most accomplished and respected scientists of systems engineering and risk analysis in the world today, said Igor Linkov, Risk and Decision Science Team Lead for the U.S. Army Engineer Research and Development Center. Lambert in his research invented the application of scenario-based preferences in risk analysis.

Connelly, Morris and Grossman worked together on a multi-disciplinary project to examine how early life experiences affect the brain and social behaviors. The team studies the brain, as well as social and cognitive development, during the first two years of life, focusing on oxytocin and its role in social behavior. Their research has helped to illuminate gaps in our knowledge about behavioral development in humans, and helps us better understand healthy and atypical development.

They received a National Science Foundation Research Award in 2017 for their cutting-edge approach in combining epigenetic, neuroscience and behavioral methods across their three labs, and their work has led to several publications.

Moore is a busy physician-scientist with his own lab, and has recently become the division chief of Pediatric Gastroenterology, Hepatology, & Nutrition at UVA. He also co-wrote the application for a Trans-University Microbiome Initiative grant, which was funded last year by the Universitys Strategic Investment Fund in an effort to make UVA a center for microbiome research. But that has not stopped him from repeatedly aiding his colleagues and providing them with key resources when they needed them.

Three colleagues joined forces to nominate Moore for the mentorship award, mentioning his critical support, his generous sponsorship and advice, and his guidance as they dealt with grant applications and the logistics of their first accepted grants. Moore went above and beyond, donating lab space and reaching out to his networks to help them make the connections and give them a leg up in their careers.

Williams only arrived at Batten two years ago, but after the violent Unite the Right rally in Charlottesville in August 2017 he was able to immediately show the value of his research and public service engagement to the University community.

Starting before he came to the University, he has spent two decades doing research in the field on police-community relations around racial profiling, community policing and the need for law enforcement officers to work with their community on issues of public safety. In all his work, he strives to make an impact on communities by building relationships and tackling problems wherever they crop up.

Dr. Williams consistently uses his knowledge, experience and passion for the good of our city, Mindy Goodall, executive director of the Charlottesville Police Foundation, said. Charlottesville is fortunate to have gained him as a citizen and champion of police and community reconciliation.

The award for Innovator of the Year was presented to Dillingham and Ingersoll for their creation of PositiveLinks, an application designed to improve health outcomes and care for people living with HIV. They will give deliver a keynote lecture Feb. 18 in the Rotunda Dome Room.

Other researchers (in alphabetical order by school) were honored for being the top 25 in sponsored funding, top cited, national award winners, named to a national academy, or named as an outstanding researcher for their school:

Timothy Beatley, PlanningBarbara Brown Wilson, PlanningMona El Khafif, Urban & Environmental Planning

Jessica Connelly, PsychologyRita F. Dove, EnglishKevin Everson, ArtTobias Grossman, PsychologyL. Ilse Cleeves, AstronomyNitya Kallivayalil, AstronomyLee M. Lockwood, EconomicsJames P. Morris, PsychologyKen Ono, MathematicsRebecca R. Pompano, ChemistryMarilyne Stains, ChemistryAlan S. Taylor, History

Christopher Barrett, Director

David G. Mick, Marketing

Derrick P. Alridge, Leadership, Foundations and PolicyDaphna Bassok, Leadership, Foundations and PolicyRobert Q. Berry, Instruction and Special EducationCatherine Bradshaw, Human ServicesBenjamin L. Castleman, Leadership, Foundations and PolicyNancy L. Deutsch, Youth-NexJason Downer, Human ServicesSara E. Rimm-Kaufman, Leadership, Foundations and PolicyWilliam J. Therrien, Instruction and Special EducationArt Weltman, KinesiologyJoanna Lee Williams, Leadership, Foundations and PolicyAmada P. Williford, Human Services

Thomas H. Barker, Biomedical EngineeringHilary Bart-Smith, Mechanical and Aerospace EngineeringCraig H. Benson, Environmental EngineeringSteven M. Bowers, Electrical and Computer EngineeringJames T. Burns, Materials ScienceBenton H. Calhoun, Electrical and Computer EngineeringJoe C. Campbell, Electrical and Computer EngineeringGeorge J. Christ, Biomedical EngineeringJason L. Forman, Center for Applied BiomechanicsJeffery W. Holmes, Biomedical EngineeringPatrick E. Hopkins, Mechanical and Aerospace EngineeringKevin A. Janes, Biomedical EngineeringJames H. Lambert, Systems and EnvironmentXiaodong (Chris) Li, Mechanical and Aerospace EngineeringPamela M. Norris, Mechanical and Aerospace EngineeringElizabeth J. Opila, Materials ScienceMatthew B. Panzer, Mechanical and Aerospace EngineeringJohn R. Scully, Materials ScienceKevin Skadron, Computer ScienceMary Lou Soffa, Computer ScienceJohn A. Stankovic, Computer ScienceMalathi Veeraraghavan, Electrical and Computer Engineering

Brian N. Williams, Public PolicyJay Shimshack, Research Dean

Jayakrishna Ambati, OphthalmologyRuth Bernheim, Public Health SciencesAlison K. Criss, Microbiology /GIDIRebecca Dillingham, Infectious DiseasesLinda R. Duska, Obstetrics/Gynecology OncologyAnindya Dutta, Biochemistry/Molecular GeneticsW. Jeff Elias, NeurosurgeryEdward H. Egelman, Biochemistry/Molecular GeneticsRobin A. Felder, Clinical PathologyEric R. Houpt, Infectious DiseasesKaren Ingersoll, Psychiatry and Neurobehavioral SciencesKaren C. Johnston, NeurologyJaideep Kapur, NeurologyAnne K. Kenworthy, Molecular Physics and BiophysicsJonathan Kipnis, NeuroscienceRobert C. Klesges, Public Health SciencesBoris P. Kovatchev, Psychiatry and Neurobehavioral SciencesThomas P. Loughran, Oncology and MedicineColeen A. McNamara, Internal and Cardiovascular MedicineWladek Minor, Molecular Physics and BiophysicsSean R. Moore, PediatricsJames P. Nataro, PediatricsImre Noth, Internal and Pulmonary MedicineMark D. Okusa, NephrologyGary K. Owens, Cardiovascular Research, Molecular Physiology and Biological PhysicsKevin A. Pelphrey, NeurologyWilliam A. Petri, Internal Medicine and Infectious DiseasesKodi S. Ravichandran, MicrobiologyPatricio E. Ray, PediatricsStephen S. Rich, Public Health SciencesLukas K. Tamm, Molecular Physics and BiophysicsGregory C. Townsend, Internal Medicine and Infectious DiseasesKenneth Walsh, Internal and Cardiovascular MedicineKatharine Hsu Wibberly, Public Health SciencesMichael C. Wiener, Molecular Physics and BiophysicsMark Yeager, Molecular Physics and BiophysicsJames C. Zimring, Pathology

Jeanne L. Alhusen, Nursing

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UVA Honors Its Leading Researchers at Boar's Head Banquet - University of Virginia

Meet the Ex-Dallas Doctor Now Playing a Doctor on TV During the Super Bowl – D Magazine

When you briefly glimpse Calvin Peters in a Verizon commercial during Super Bowl LIV on Sunday, his face might seem familiar.

Maybe you remember him from his performances during the audition stage of American Idol. Perhaps you recognize him from guest appearances in television dramas such as 9-1-1 or Snowfall. Or possibly he was your doctor during an appointment at UT Southwestern Medical Center.

The diverse resume for the former Dallas physician now finds him splitting much of his time between treating patients and auditioning for roles on stage and screen.

The art caters to my right brain, and the medicine and science is challenging and fostering my left brain, Peters said. I love helping people. Every time I look in a patients eyes and theyre hurting, that tugs at my heart in a way that nothing else can. But then I love to be on stage and perform.

Appropriately enough, Peters plays a surgeon in the Verizon spot that will debut Sunday. Its a continuation of the companys ongoing campaign saluting first responders and was directed by Hollywood filmmaker Peter Berg (Friday Night Lights).

Hes a visionary. It was so encouraging and motivating to see this guy work, Peters said. I want to continue to grow to where Im working with all kinds of creative talent.

Peters developed a passion for medicine and for performing while growing up in the Stop Six neighborhood in Fort Worth. He acted in his first play at age 9, and later graduated from North Side High School.

At Hampton University in Virginia, Peters was involved in musical theater before graduating with a degree in molecular biology in 2006.

He returned to Texas to attend medical school at UT Southwestern, then stayed to complete his residency in 2014, specializing in physical medicine and rehabilitation.

During his residency, he impulsively auditioned for American Idol, eventually making the show in 2013. Later that year, he appeared on an episode of The Dr. Oz Show spotlighting the Most Eligible Doctors. He was hooked.

I was still going to work every day, and everybody at the hospital was really supportive, but I still felt empty, he said.

So Peters took some classes and hired an agent in Dallas, and began acting in local commercials and short films. In 2015, he relocated to Los Angeles and set up his practice primarily as a therapist for nursing home patients, which allowed him to have a flexible schedule.

As his exposure grew, Peters refined his craft with improvisation classes, voice classes, and dance classes. He hired a manager and began booking jobs, including an episode of Snowfall that aired in August and a small role on 9-1-1 in November. Peters also plays a waiter in the low-budget romantic comedy What Love Looks Like, which began streaming on Amazon Prime earlier this month.

Juggling his two careers has become more of a challenge. Peters recently had an opportunity to play Harpo in a traveling stage production of The Color Purple, but couldnt commit to the full year away from his practice.

His circumstances mean Peters isnt be as desperate as other up-and-coming actors in California, but he said hes just as driven. He spends 30-40 hours per week reading scripts and rehearsing.

My bills are all paid. Im still saving money and have health insurance. I have an assistant. I live in Beverly Hills. My life is not bad, Peters said. But when I go into an audition, Im still going hard. Im giving it my all. Im putting my foot in the water just as fast and hard as somebody whos waiting tables.

Eventually, he would like to give back to the rough neighborhood in eastern Fort Worth where he was raised, and where his family has been victimized by a pervasive cycle of drugs and violence.

Ill always be connected to that community. Im never going to act like Im too good for them. Fort Worth will always be in my heart, he said. Sometimes its so difficult to go be around some of the situations because the energy and the vibes are not right. The way I grew up was not ideal, but I feel so fortunate.

As his success in show business grows and he plans for the long-term future, Peters said he hasnt thought about abandoning his career in medicine.

I dont think you can ever stop somebody from being a doctor, Peters said. I try not to think about that right now, because I dont have to make that choice yet. If the situation got big enough, I probably wouldnt be able to go into a hospital and practice, but theres always a way to stay connected to healthcare.

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Meet the Ex-Dallas Doctor Now Playing a Doctor on TV During the Super Bowl - D Magazine

Realizing The Full Potential Of Precision Medicine In Oncology – Contract Pharma

Precision medicine promises a new paradigm in oncology where every patient receives truly personalized treatment. This approach to disease diagnosis, treatment and prevention utilizes a holistic view of the patientfrom their genes and their environment to their lifestyleto make more accurate decisions.

Growing at a rate of 10.7 percent, the precision medicine market is expected to exceed $96 billion by 2024.1 Bioinformatics represent a significant share of the market, as bioinformatics tools enable the data mining necessary for rapid identification of new drug targets and repurposing of existing treatments for new indications.1 (Reuters) The oncology segment of the precision market is expected to experience an 11.1 percent compounded annual growth rate (CAGR) leading up to 2024 due to the success of recent targeted therapies and subsequent high demand.

Still, precision medicine is in its infancy, and making personalized treatment a reality for all patients requires a transformation in how novel therapies are developed and delivered. New regulatory, technical, clinical and economic frameworks are needed to ensure that the right patients are able to access the right therapy at the right time. In this article, we review the current state of precision medicine in oncology and explore some of the challenges that must be addressed for precision medicine to reach its full potential.

Great strides toward precision medicine are being made in the area of cancer immunotherapy, which is designed to boost a patients own immunity to combat tumor cells. The introduction of immune checkpoint inhibitors (PD-1/PD-L1 and CTLA-4 inhibitors) revolutionized treatment for certain hematologic malignancies and solid tumors. To date, immune checkpoint inhibitors have been approved by the U.S. Food and Drug Administration (FDA) for more than 15 cancer indications, but their widespread use has been hampered by unpredictable response rates and immune-related adverse events.

The approvals of the first chimeric antigen receptor (CAR)-T cell (CAR-T) therapies in 2017 were the next leap forward in precision medicine. These immunotherapies demonstrated that it was possible to take out a patients own T-cells, genetically modify them, and then put them back in to target cancer cells. With complete remission rates as high as 83 percent within three months of treatment, CAR-T therapies represent a seismic shift in our approach to cancer, bringing the elusive possibility of a cure one step closer. However, longer-term follow-up has shown that these remissions may not be durable2 and prevention of relapse must still be studied.

Ultimately, the goal of cancer immunotherapy is to stimulate the suppressed immune system of a patient with cancer so that it can launch a sustained attack against tumor cells.3 This is complicated, as the interactions between tumors and immune systemsometimes called the Cancer-Immunity Cycle (see Figure 1 in the slider above)4are complex and dynamic. The Cancer-Immunity Cycle manages the delicate balance between the immune systems ability to recognize non-self and the development of autoimmunity.

In some cases, the immune system may fail to recognize tumor cells as non-self and may develop a tolerance to them. Moreover, tumors have an armamentarium of methods for evading the immune system. Given this elaborate interplay between cancer and immunity, there is a wide range of potential cancer immunotherapy approaches:

The immune response to cancer involves a series of carefully regulated events that are optimally addressed as a group, rather than individually.4 The complexity of the immune response to cancer provides a strong rationale for combination therapies, for instance:

Increasingly, the development and deployment of immunotherapy relies on harnessing genomic data to identify the patients most likely to respond to immunotherapy and to customize immunotherapy for a given patient.6 Thus, molecular profiling technologies, such as next-generation sequencing, have become integral to drug development and patient selection. At the same time, researchers are focusing on identifying molecular alterations in tumors that may be linked to response.7 The molecular fingerprints of a tumor can be quite complex and heterogeneous, not only across tumors, but also within a single patient. Consequently, molecular tumor characterization requires both multidimensional data from laboratory and imaging tests and advanced software and computational methods for analyzing these data.8 This emergence of computational precision oncology is associated with both opportunities and challenges, from validation and translation to regulatory oversight and reimbursement.

The regulatory landscape is evolving to keep pace with technological advances in cell engineering and gene editing. Since 2013, the FDA has published four guidance documents on cellular and gene therapy products, as well as two guidance documents providing recommendations on regenerative medicine advanced therapies (RMATs). Specifically, their Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, published in November 2017, provides guidance on the expedited development and review of regenerative medicine therapies for serious or life-threatening diseases and conditions. This document also provides information on the use of the accelerated approval pathway for therapies that have been granted the RMAT designation.9

In the EU, the European Medicines Agency (EMA) published a draft revision of its Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells in July 2018.10 This draft revision includes current thinking on the requirements for nonclinical and clinical studies, as well as specific sections on the scientific principles and clinical aspects of CAR-T products.

Precision medicines such as CAR-T therapies require manufacturers to transform a complex, individualized treatment into a commercial product. In conventional manufacturing, the entire manufacturing process occurs within the confines of the manufacturing facility. With cell therapies, however, the process begins with the collection of cells from the patient and ends with administration of the final product (see Figure 2 in the slider above). In between, the cells are handed off multiple times for the process of genetic modification, creating a complex supply chain that blends manufacturing and administration.11

Moreover, in contrast to traditional manufacturing where the starting materials are standardized or well-defined, the starting materials for cell therapies are derived from patients and, thus, highly variable.

As evidenced by the manufacturing challenges that plagued the launch of Kymriah (tisagenlecleucel), even pharmaceutical giants have struggled with meeting label specifications for commercial use.13 To help address its manufacturing hurdles, Novartis acquired CellforCure, a contract development manufacturing organization, and plans to transform by focusing on data and digital technologies.14,15 What this means for sponsors is that robust, scalable manufacturing must be incorporated into clinical developing planning at its earliest stages.

The high price tags associated with CAR-T therapies illustrate how expensive targeted therapies are in comparison to their traditional counterparts.16 Existing health insurance models have not been structured to reimburse for costly treatments that offer the potential for long-term benefit or even cure. The pricing model for CAR-T therapies may be especially challenging for private insurance companies, which have higher turnover and shorter coverage windows than national health insurance programs. For sponsors of precision medicine therapies, one way to address the challenge of reimbursement is to create innovative, value- or outcomes-based pricing models, rather than focusing on sales volume. The success of these new pricing models will rely on patient selection. To demonstrate value and optimizing outcomes, sponsors will need to develop profiles of patients who are most likely to respond and provide tools for identifying these patients.8

Of note, on August 7, 2019, the Centers for Medicare & Medicaid Services (CMS) finalized the decision to cover FDA-approved CAR-T therapies when provided in healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies (REMS) for FDA-approved indications. Medicare will also cover FDA-approved CAR-T treatments for off-label uses that are recommended by CMS-approved compendia.17

Beyond the pharmaceutical companies that are working to develop personalized treatments, the precision medicine ecosystem has a number of other key stakeholdersregulators, payers, diagnostic companies, healthcare technology companies, healthcare providers and, of course, patients. Pharmaceutical companies need to engage with each of these stakeholders by providing education or developing partnerships that help demonstrate the need for high-quality data collection, the value of precision medicine, and the process for identifying the right patients.

Sponsors may also benefit from engaging with patient advocacy groups as these groups play a critical role in connecting patients and caregivers with scientific and healthcare experts to learn about how new immunotherapy breakthroughs are changing the standard of care.

Empowered patients pushing for the latest innovations are propelling precision medicine forward, but we still have a way to go before the full potential of precision medicine is realized. In its maturity, precision medicine will not only enable the personalization of treatments for individual patients, but also inform public health at a population level as insights from the genetic and molecular data collected are used to advance our understanding of disease. Robust data collection and analysis, along with standardization, are required for building this foundation of precision medicine, and multi-stakeholder buy-in is necessary for addressing issues around data integration and privacy.

While significant challenges remain, the opportunity to transform patient outcomes and population health with precision medicine is tantalizing. Increasingly, we are seeing advanced technologiessuch as artificial intelligence and machine learningbeing incorporated into the drug discovery and development process. This underscores the critical need for a multidisciplinary approach to precision medicine, from discovery at the bench all the way through to delivery at the bedside, to help ensure that more patients can access the right therapy at the right time, and the right price.

References

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Realizing The Full Potential Of Precision Medicine In Oncology - Contract Pharma

Triple negative breast cancer might not actually be ‘breast cancer’ – Clinical OMICs News

A new study from New York might completely change how breast cancer is classified and treated. Researchers have discovered, in a study of triple negative breast cancer in black women, that the molecular mechanisms involved are more closely related to non-breast cancers, and two specific gene mutations may be responsible for the tumor development.

This study would signal a potential treatment breakthrough for patients with this form of breast cancer, which disproportionately affects and tends to develop more aggressively in black women. Triple negative breast cancer is a form of breast cancer that is not hormone driven, and it does not express the estrogen receptor, progesterone receptor, or HER2/neu genesunlike other forms of the disease, which rely on hormone-driven therapies to control the cancer.

The paper, authored by researchers at The Graduate Center of The City University of New York, Hunter College, Memorial Sloan Kettering Cancer Center, and the University of Chicago, details findings about the interaction between two specific genes during DNA replication that appear to drive growth of malignant cells in patients with triple negative breast cancer.

The research team found the presence of a mutated p53 (a tumor suppressor gene whose mutation often leads to cancer development) and a mutated PARP (a gene that maintains DNA integrity) in a large majority of patients with triple negative breast cancer. Specifically, they found that p53 would interact with replicating DNA and PARP, driving cell growth and division.

This information would suggest that if drugs could specifically target these two mutated genes and the proteins they produce, it would be possible to suppress their actions and slow or stop tumor growth, so the researchers tested this in lab, using drugs developed for other types of cancer.This was indeed the case.

Our new findings suggest that the presence of both [p53] and PARP could serve as a good identifier of breast cancers that would respond to combined treatment with talazoparib a PARP inhibitor that was developed to treat breast cancers with the BRCA mutation and temozolomide a chemotherapy agent that is used to treat some brain cancers, said Professor Jill Bargonetti, whose lab conducted the research. This is an exciting finding because it could lead to the first targeted therapy for triple negative breast cancer, enabling more precise and effective treatment of a very aggressive form of the disease.

To conduct this study, researchers investigated a variety of breast cancer cell lines, patient-derived xenographs, tissue microarray samples, and data from The Cancer Genome Atlas to tease out the association and interaction between p53 and PARP in triple negative breast cancer.

Our findings that mutant p53 and PARP participate in the DNA replication pathway will provide mechanism-derived dual biomarkers that aid in the diagnosis and treatment of these therapeutically elusive subsets of breast cancer, said Gu Xiao, a research associate with Bargonettis lab and the papers first author.

The scientists had discovered that high levels of these proteins are present on replicating DNA in these types of tumors, suggesting that the unusual presence and levels of these proteins may drive tumor growth. When these researchers reviewed the therapeutic mechanisms of existing cancer drugs, it led the research team to identify talazoparib and temozolomide as existing drugs that could potentially be combined to create a targeted therapy that could effectively suppress the growth of triple negative breast cancer tumors.

Looking to the future, this group plans to test if this combination of drugs can successfully block replication of triple negative breast cancer cells in xenographed animal models. If the therapy can prove to be successful, it would very likely lead to the reclassification of triple negative breast cancer to a category of cancers called mutant p53/PARP1 positive cancers, which are treatable with a combination PARP inhibitor therapies.

With so little in positive news for this particular subset of breast cancer, and many previous clinical trials being disappointing, these new findings are both welcome news to the scientific community, as well as a good explanation as to why this disease has been so hard to treat compared to other forms of breast cancer.

This study gives doctors two new biomarkers to test for in patients, and many new potentials in clinical trials, which can be fast tracked since so many drugs already exist that target these specific genes. While this study only tested out two drugs, there are many other drugs that specifically target p53 and PARP, and many more which can be developed, which may give even better results. Hopefully, there will be many new, positive developments for this disease following this discovery.

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Triple negative breast cancer might not actually be 'breast cancer' - Clinical OMICs News

Is Predictive Technology Group Inc (OTCMKTS:PRED) Ready for Another Run? – The Oracle Dispatch

Predictive Technology Group Inc (OTCMKTS:PRED) just announced that it and Atrin Pharmaceuticals LLC areentering into a collaboration agreement to develop molecular diagnostic toolsto facilitate improved selection of cancer patients who would most benefit fromtreatment with DNA Damage and Response (DDR) inhibitors, including Atrins andother small molecule ATR inhibitors.

According to the release, Atrinand Predictive will jointly utilize Predictive Laboratories state-of-the-artsequencing capabilities and genomics expertise to identify cancer patients withspecific molecular markers that predict the level of clinical response toAtrins, and other, targeted therapies. This is intended to improve patientoutcomes as well as improve Atrins ability to successfully progress itsproduct pipeline, and upon commercialization, improve on the treatments forwomen with cancer.

Predictive Technology GroupInc (OTCMKTS:PRED) promulgates itself as a company that, together with itssubsidiaries, develops and commercializes discoveries and technologies involvedin novel molecular diagnostic and pharmaceutical therapeutic/human cells,tissues, and human cellular and tissue-based products (HCT/Ps).

The company operates throughtwo segments, Regenerative Medicine Products and HCT/Ps, and Diagnostics andTherapeutics. It offers ARTguide, a genetic diagnostic and prognostic test forwomen experiencing infertility as a result of endometriosis and other healthconcerns; and regenerative medicine products, including AmnioCyteT, AmnioCytePlusT, PolyCyteT, and CoreCyteT.

The company was formerly knownas Global Enterprises Group, Inc. and changed its name to Predictive TechnologyGroup, Inc. in July 2015. Predictive Technology Group, Inc. was founded in 2005and is headquartered in Salt Lake City, Utah.

According to companymaterials, Predictive Technology Group aims to revolutionize patient carethrough predictive data analytics, novel gene-based diagnostics and companiontherapeutics through its subsidiaries Predictive Therapeutics, PredictiveBiotech, and Predictive Laboratories. These subsidiaries are focused onendometriosis, scoliosis, degenerative disc disease and human cell and tissueproducts. The subsidiaries use genetic and other information as cornerstones inthe development of new diagnostics that assess a persons risk of illness andtherapeutic products designed to identify, prevent and treat diseases moreeffectively.

Find out when $PRED reaches critical levels. Subscribe toOracleDispatch.com Right Now by entering your Email in the box below.

As noted above, PRED just announced that it and Atrin Pharmaceuticals LLC are entering into a collaboration agreement to develop molecular diagnostic tools to facilitate improved selection of cancer patients who would most benefit from treatment with DNA Damage and Response (DDR) inhibitors, including Atrins and other small molecule ATR inhibitors.

Weve witnessed 36% piled onfor shareholders of the listing during the trailing month. In addition, thename has witnessed a pop in interest, as transaction volume levels haverecently pushed 11% above the average volume levels in play in this stock overthe longer term.

We are very pleased to workwith Atrin Pharmaceuticals, a recognized leader in the development ofanti-cancer therapeutics targeting DDR, said Bradley Robinson, president andchief executive officer of Predictive Technology Group. We see an opportunityto develop a precision medicine approach to address unmet medical needs bycombining our state-of-the-art sequencing capabilities, genomics expertise andcompanion diagnostics with Atrins targeted therapeutics. This collaboration isconsistent with our vision of building a leading womens health platform, andwe look forward to working together on this important initiative.

Currently trading at a marketcapitalization of $276M, PRED hasa chunk ($842K) of cash on the books, which stands against about $8.6M in totalcurrent liabilities. PRED is pulling in trailing 12-month revenues of $43.7M.In addition, the company is seeing major top-line growth, with y/y quarterlyrevenues growing at 2.4%. You can bet we will update this one again as newinformation comes into view. Sign-up forcontinuing coverage on shares of $PRED stock, as well as other hot stock picks,get our free newsletter today and get our next breakout pick!

Disclosure: we hold no position in $PRED, either long orshort, and we have not been compensated for this article.

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Is Predictive Technology Group Inc (OTCMKTS:PRED) Ready for Another Run? - The Oracle Dispatch

OpGen (NASDAQ:OPGN) and Avant Diagnostics (NASDAQ:AVDX) Head-To-Head Review – Riverton Roll

OpGen (NASDAQ:OPGN) and Avant Diagnostics (OTCMKTS:AVDX) are both small-cap medical companies, but which is the better stock? We will contrast the two businesses based on the strength of their profitability, valuation, risk, dividends, analyst recommendations, institutional ownership and earnings.

Profitability

This table compares OpGen and Avant Diagnostics net margins, return on equity and return on assets.

Volatility and Risk

OpGen has a beta of 0.64, suggesting that its stock price is 36% less volatile than the S&P 500. Comparatively, Avant Diagnostics has a beta of 9.42, suggesting that its stock price is 842% more volatile than the S&P 500.

Earnings & Valuation

This table compares OpGen and Avant Diagnostics revenue, earnings per share and valuation.

Avant Diagnostics has lower revenue, but higher earnings than OpGen.

Analyst Recommendations

This is a breakdown of current ratings and target prices for OpGen and Avant Diagnostics, as reported by MarketBeat.com.

OpGen presently has a consensus target price of $7.93, suggesting a potential upside of 405.31%. Given OpGens higher probable upside, equities research analysts plainly believe OpGen is more favorable than Avant Diagnostics.

Insider & Institutional Ownership

0.3% of OpGen shares are held by institutional investors. Comparatively, 0.0% of Avant Diagnostics shares are held by institutional investors. 5.0% of OpGen shares are held by insiders. Comparatively, 38.0% of Avant Diagnostics shares are held by insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a stock will outperform the market over the long term.

Summary

Avant Diagnostics beats OpGen on 7 of the 11 factors compared between the two stocks.

About OpGen

OpGen, Inc., a precision medicine company, engages in developing molecular information products and services to combat infectious diseases in the United States and internationally. The company utilizes molecular diagnostics and informatics to help combat infectious diseases. It also helps clinicians with information about life threatening infections, enhance patient outcomes, and decrease the spread of infections caused by multidrug-resistant microorganisms. The company's products include Acuitas AMR Gene Panel, a vitro diagnostic test for the detection and identification of various bacterial nucleic acids and genetic determinants of antimicrobial resistance in urine specimens or bacterial colonies isolated from urine and other body sites; and QuickFISH and PNA FISH products, which are FDA-cleared and CE-marked diagnostic test designed to detect antimicrobial- resistant pathogens. In addition, it offers Acuitas Lighthouse informatics systems, a cloud-based HIPAA compliant informatics offerings, which combine clinical lab test results with patient and hospital information, and provide analytics and insights to enable manage MDROs in the hospital and patient care environment. The company was incorporated in 2001 and is headquartered in Gaithersburg, Maryland.

About Avant Diagnostics

Avant Diagnostics, Inc., a commercial-stage molecular data-generating company, focuses on the development and commercialization of proprietary data-generating assays that provide information for physicians and patients in the areas of cancers. It owns license and distribution right for OvaDx, a noninvasive proteomics diagnostic screening test for the early detection of ovarian cancer. The company was founded in 2009 and is based in Washington, District of Columbia.

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OpGen (NASDAQ:OPGN) and Avant Diagnostics (NASDAQ:AVDX) Head-To-Head Review - Riverton Roll