Monthly Archives: March 2017

KANNAPOLIS A person could be almost three times more likely to develop multiple sclerosis if they are carrying variants of two particular genes, according to the latest findings from scientists at Duke Health and The University of Texas Medical Branch at Galveston.

The finding, published in the March 23 issue of the journal Cell, could open the way for new tests to identify those at greatest risk of MS and autoimmune disorders, as well as the development of drugs, the researchers said.

The research used biospecimens from the MS cohort of the Measurement to Understand the Reclassification of Disease Of Cabarrus/Kannapolis ( MURDOCK ) Study. The MURDOCK Study is Duke Healths longitudinal clinical research study based at the North Carolina Research Campus in Kannapolis, N.C. The MURDOCK community registry and bio-repository includes more than 12,000 participants and nearly 460,000 biological specimens.

Multiple sclerosis is a major cause of neurological disease in younger adults between the ages of 20 and 50 and disproportionally affects women.

The disease causes the bodys own immune system to attack nerve cells in the spinal cord and brain, causing problems with vision, muscle control, balance and basic body functions. Other symptoms can occur, and could lead to permanent disability.

While treatable, current MS therapies have adverse side effects, as they focus on slowing the progression of the disease through suppression of the immune system. There is no cure for MS.

Our study identifies an interaction with a known MS risk gene to unlock a new MS candidate gene, and in doing so, establishing a novel mechanism that is associated with the risk of multiple sclerosis and other autoimmune diseases, said co-lead author Simon Gregory, Ph.D., director of Genomics and Epigenetics at the Duke Molecular Physiology Institute and principal investigator for the MURDOCK MS Study.

Gregory with colleagues at University of Texas Medical Branch, the University of California, Berkeley, and Case Western Reserve University found two particular DNA variants that appear to play a role in MS. One of these variants is in IL7R, a gene previously associated with MS, and the other in DDX39B, a gene not previously connected to the disease.

When the two are present in a persons genetic code, their interaction can lead to an over-production of a protein called sIL7R. That proteins interactions with the bodys immune system plays an important, but not completely understood, role in MS.

Researchers used MURDOCK biospecimens to examine the chromosomal differences between DDX39B and IL7R.

The researchers said this new information could potentially be used to craft new tests to diagnose multiple sclerosis, or to improve therapeutic toolkits to fight MS and other autoimmune disorders.

One could envision how this type of knowledge will someday lead to diagnose multiple sclerosis sooner and, now that we have promising therapies, a doctor could start the appropriate treatment more quickly. It is not out the realm of possibility to imagine a path for screening for other autoimmune diseases such as Type 1 Diabetes, said co-lead author Mariano Garcia-Blanco, M.D., Ph.D., professor and chair of the Department of Biochemistry and Molecular Biology at University of Texas Medical Branch.

In addition to Gregory and Garcia-Blanco, study authors include lead author Gaddiel Galarza, Farren B.S. Briggs, Irina Evsyukova, Geraldine Schott-Lerner, Edward M. Kennedy, Tinashe Nyanhete, Liuyang Wang, Laura Bergamaschi, Steven G. Widen, Georgia D. Tomaras, Dennis C. Ko, Shelton S. Bradrick and Lisa F. Barcellos.

The research was supported by the National Institutes of Health, National MS Society Pilot Award, Duke University Whitehead Scholarship, Ruth and A. Morris Williams Faculty Research Prize funds from Duke University School of Medicine, start-up funds from University of Texas Medical Branch and funds from Mr. Herman Stone and family for MS research.

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New Gene Interaction Appears to be Associated with Increased MS ... - Independent Tribune

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Despite their focus on rare genetic syndromes, the researchers say their work will be helpful to the millions worldwide who have lost control of their weight for reasons other than genetics.

"If you know the gene and the function of the gene, then you know which biological mechanism is defective," said David Meyre, senior author of the study and an associate professor at McMaster University's School of Medicine in Ontario, Canada. This knowledge of obesity genetics, then, can be applied to more common forms of the condition, he said.

A case in point would be leptin, which is a hormone produced by the body's fat cells and is found to be deficient in some people due to genetic mutations. The gene responsible for producing the "satiety hormone," as leptin is known, was first identified in 1990. Since then, knowledge of this gene has shifted our understanding of fat cells and how weight gain occurs.

"The reason we studied this is very simple," said Meyre. "Every time I was writing a research paper and I was describing what we knew about the genetics of obesity, I didn't have a reference." One day, he decided to write the necessary reference himself, since he suspected the 20 to 30 commonly known genetic syndromes might be, in fact, an underestimation.

"For the study, we focused on monogenic forms of obesity," said Meyre. He explained that monogenic or "Mendelian" forms mean that if you have one mutation, you develop the disease. "It's not that it increases your risk, it's 100% sure you develop the disease," he said. For example, Huntington's disease, a progressive brain disorder that causes uncontrolled movements and loss of thinking ability, is caused by a single inherited gene mutation.

The monogenic obesity syndromes are very rare, Meyre noted, so rare they may collectively represent only 0.5% of the obese population in Canada -- one in a million births.

Meyre and his colleagues from McMaster University and University of British Columbia searched seven databases for papers on the topic. The team adopted a systematic strategy for reviewing the scientific literature, which included two independent reviews of each paper.

All told, the researchers analyzed 161 papers and found 79 obesity syndromes reported in the scientific literature.

"My intuition was correct," said Meyre.

In the monogenic obesity syndromes, not only does the genetic defect result in obesity but it also causes additional abnormal features, including mental disability, characteristic facial features, kidney disease and heart malformation.

Of the 79 syndromes identified, 19 had the genetics worked out completely so that a simple lab test would be able to confirm the condition. Another 11 had been partially clarified, while 27 had been mapped to a chromosomal region. For the remaining 22 syndromes, researchers had not yet identified the genes or location along the chromosomes.

"Identifying genes is very important for the families," said Meyre. He explained that some of the more common syndromes have been treated with a hormone that works very well to improve symptoms. If the genetics are worked out for each of these syndromes, that should enable scientists to find or develop appropriate treatments.

"We also hope that our study will help clinicians to recognize these syndromes," said Meyre. Since in the entire course of their careers, doctors may encounter just one, maybe two patients with these syndromes, most of the time, due to a lack of familiarity and information, the conditions go unrecognized and patients do not get help.

Additionally, the results will help scientists better understand the genes and molecules important to obesity among members of the general population.

Mary Freivogel, president of the National Society of Genetic Counselors, found the comprehensive nature of the new study to be a strength.

"One weakness of the study is that some of these obesity syndromes are so rare that it was not possible for the authors to determine how often the syndrome occurs in the general population nor how reliable the information reported about the syndrome was," said Freivogel, who played no part in the new study.

"Most of the obesity in the United States is NOT syndromic, said Freivogel. She explained that the overwhelming majority of cases are "polygenic and multifactorial," meaning it has resulted from a combination of multiple genetic factors, as well as environmental and lifestyle factors. Freivogel added that anyone wishing to undergo a genetic test might want to talk with a counselor to ensure the test is the right choice and any results are interpreted correctly.

Beales, who was not involved in the new study, also felt pleased that someone had updated this "specialised category of obesity." However, he disagrees with the recommendation proposed by Meyre and his co-authors to name each disease after the scientists who discovered them.

"This is an antiquated notion and unhelpful," said Beales, who observed the preferred name for DiGeorge syndrome -- a common genetic disorder resulting in cognitive impairment and other medical complications -- is now called Deletion22 syndrome, which is a more useful way to look at the disease.

Still, the work has its virtues, said Beales.

"I think where a catalogue of this nature is valuable is that it provides a good resource for researchers who are interested in extrapolating from rare to common," said Beales. "The Mendelian disorders have an untapped potential to reveal mechanistic insights (and possibly new treatments) to common (non-syndromic) obesity."

Dr. Liam R. Brunham, an assistant professor of medicine at University of British Columbia, said the most remarkable finding of the review may be that of the 79 obesity syndromes, the genetic basis of only one-quarter of them is known. Very likely, then, there is "a huge amount regarding the genetics of obesity that remains to be discovered," said Brunham. He was not involved in the study.

Using leptin as an example, Brunham said there's much that can be learned regarding the biology of obesity from even a single gene.

"This suggests that discovering the genetic basis of the remaining obesity syndromes will yield huge advances in our understanding of obesity, which could lead to new opportunities for its treatment and prevention," said Brunham.

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The weirder side of obesity: genetic forms of obesity are rare yet numerous - CNN

Cell Therapy Manufacturing Market, 2027: Partnerships / Collaborations have been Widespread and will Continue to act as Key Enablers

Dublin, March 28, 2017 -- Research and Markets has announced the addition of the "Cell Therapy Manufacturing Market, 2017-2027" report to their offering.

During the course of our study, we identified over 110 organizations that are actively involved in the manufacturing of cell therapies.

The scope of this report primarily includes manufacturing of advanced therapy medicinal products (ATMPs) that involve the use of immune cells such as T-cells, Tregs, dendritic cells, tumor cells and NK cells, and stem cells such as adult stem cells, human embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs).

Several players, including cell therapy developers, research institutes, contract manufacturing organizations, and government and non-profit organizations, are playing a critical role in the development and manufacturing of these cell therapies. In fact, a number of these players have made heavy investments to expand their existing capabilities and establish new facilities for cell therapy products in order to meet the increasing demand.

Additionally, stakeholders have received significant support from governments worldwide, in terms of funding and establishment of consortiums to accelerate the transition of these therapies from laboratories to clinics. It is important to highlight that companies that offer logistics and operational services have developed systems / tools for safer and quicker delivery of therapies from manufacturing sites to patients; this has been identified as one of the key challenges in the overall development process.

Popular examples of approved cell-based therapies include (in order of their year of approval) Carticel, CreaVax-RCC, JACE, ReliNethra, PROVENGE and Prochymal. In addition, over 500 cell-based therapy candidates are currently in different stages of clinical development; these are being evaluated in over 1,000 active clinical studies in various regions across the globe. The growing number of cell therapy candidates, coupled with their rapid progression through the various phases of clinical development, continues to create an increasing demand for facilities that offer manufacturing services for these therapies.

The market already has a wide array of well-established players, mid-sized companies and start-ups. Several industry players as well as academic institutes are significantly contributing to the production of GMP grade cell types. In addition, the market has witnessed the entry of several players that offer novel technology solutions, aimed at improving and upgrading existing cell-based therapies and their manufacturing processes. We have observed that such players have signed multiple partnerships / collaborations with an aim to optimize, scale-up and expand the capabilities for production of cell-based therapies.

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. CELL THERAPY MANUFACTURING: INTRODUCTION 3.1. Context and Background 3.2. Cell-based Therapies: Introduction 3.3. Cell Therapy Manufacturing: An Introduction 3.4. Cell-based Therapies Manufacturing: Key Challenges 3.5. Cell Therapy Manufacturing: Types of Manufacturers 3.6. Cell Therapy Manufacturing: Other Important Considerations 3.7. Cell Therapy Manufacturing: Regulatory Landscape

4. MARKET OVERVIEW 4.1. Chapter Overview 4.2. Cell Therapy Manufacturing: Overall Market Landscape 4.3. Cell Therapy Manufacturing: Role of Logistic Service Providers

5. ROADMAPS: POTENTIAL STRATEGIES TO OVERCOME EXISTING CHALLENGES 5.1. Chapter Overview 5.2. Roadmap for the United States 5.3. Roadmaps for Other Geographies

6. CELL THERAPY MANUFACTURING: IN-HOUSE MANUFACTURERS 6.1. Chapter Overview 6.2. Argos Therapeutics 6.3. Bavarian Nordic 6.4. Cytori Therapeutics 6.5. Juno Therapeutics 6.6. MEDIPOST 6.7. SOTIO (Acquired by PPF Group) 6.8. Stemedica Cell Technologies

7. CELL THERAPY MANUFACTURING: INDUSTRY PLAYERS 7.1. Chapter Overview 7.2. Cell and Gene Therapy Catapult 7.3. CELLforCURE 7.4. Lonza 7.5. PharmaCell 7.6. PCT, a Caladrius Company 7.7. Roslin Cell Therapies 7.8. Waisman Biomanufacturing

8. CELL THERAPY MANUFACTURING: NON-INDUSTRY PLAYERS 8.1. Chapter Overview 8.2. Center for Cell and Gene Therapy, Baylor College of Medicine, US 8.3. Centre for Cell Manufacturing Ireland, National University of Ireland, Ireland 8.4. Clinical Cell and Vaccine Production Facility, University of Pennsylvania, US 8.5. Guy's And St. Thomas' GMP Facility, Guy's Hospital, UK 8.6. Newcastle Cellular Therapies Facility, Newcastle University, UK 8.7. Rayne Cell Therapy Suite, King's College London, UK 8.8. Scottish National Blood Transfusion Services Cellular Therapy Facility, Scottish Centre of Regenerative Medicine, UK 8.9. Laboratory of Cell and Gene Medicine, Stanford University, US

9. ROLE OF NON-PROFIT ORGANIZATIONS 9.1. Chapter overview 9.2. Cell Therapy Manufacturing: List of Non-Profit Organizations 9.3. Cell Therapy Manufacturing: International Societies

10. RECENT DEVELOPMENTS 10.1. Chapter Overview 10.2. Collaboration / Agreement Models 10.3. Cell Therapy Manufacturing: List of Collaborations 10.4. Cell Therapy Manufacturing: Partnership Analysis

11. MARKET SIZING AND FORECAST 11.1. Context and Background 11.2. Forecast Methodology 11.3. Cell Therapy Manufacturing Market, 2017-2027 11.4. Cell Therapy Manufacturing Market: Regional View

12. SWOT ANALYSIS

13. CONCLUSION 13.1. A Growing Pipeline of Cell Therapy Products is Likely to Increase the Demand for Manufacturing of Cell-based Therapies 13.2. Stakeholders are Continuously Striving to Overcome Existing Challenges 13.3. Developed Economies have Emerged as Prominent Hubs for Cell Therapy Manufacturing 13.4. Both Industry and Academia have Jointly Led the Initiatives; The Trend is Likely to Persist in the Near Term 13.5. Partnerships / Collaborations have been Widespread and will Continue to act as Key Enablers 13.6. The Manufacturing of Cell-based Therapies is Likely to Become a Multi-billion Dollar Market in the Coming Decade

14. SURVEY ANALYSIS 14.1. Chapter Overview 14.2. Seniority Level of Respondents 14.3. Type of Cell Therapy 14.4. Scale of Operation 14.5. Source of Cells 14.6. Type of Cell Culture System 14.7. Fill / Finish Service

15. INTERVIEW TRANSCRIPTS 15.1. Chapter Overview 15.2. Tim Oldham, CEO, Cell Therapies 15.3. Brian Dattilo, Manager of Business Development, Waisman Biomanufacturing 15.4. Mathilde Girard, Department Leader, Cell Therapy Innovation and Development, YposKesi 15.5. Dr. Gerard J Bos (CEO, CiMaas)

16. APPENDIX: TABULATED DATA

17. APPENDIX: LIST OF COMPANIES AND RESEARCH ORGANIZATIONS

For more information about this report visit http://www.researchandmarkets.com/research/bvlctq/cell_therapy

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Cell Therapy Manufacturing Market, 2027: Partnerships / Collaborations have been Widespread and will Continue to ... - EconoTimes