Category Archives: Gene Medicine

WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

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Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Montana Standard

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In world-first breakthrough, French doctors use gene therapy to treat rare blood disease - RFI

Scientists and doctors will gather in La Jolla this week for a conference about how medicine could change and in some cases, how it's already starting to change thanks to advances in genomics.

The 10th annual Future of Genomic Medicine conference organized by the Scripps Translational Science Institute and held at the Scripps Institution of Oceanography is being held at a time when genomic breakthroughs have increasingly become the subject of high-profile intellectual property disputes, ethical debates and major industry investments.

One panel focussed on the gene editing tool CRISPR will feature MIT scientist Feng Zhang, who recently came out ahead of UC Berkeley scientists in a patent battle over who deserves credit for developing a tool that could be used to fight cancer, malaria and a host of other genetic diseases.

"I think it can't really be overstated how big of a revolution CRISPR is in terms of manipulating the genome," said Scripps Translational Science Institute researcher Ali Torkamani, who will moderate the CRISPR panel.

"It's exciting to the public and it is really exciting to scientists as well," he said.

Other panels will cover the federal Precision Medicine Initiative, which is still in nascent stages, and attempts to develop blood-based screening tests meant to catch cancer in early stages. Grail, a spinoff company of San Diego-based Illumina, announced on Wednesday that it has raised $900 million to develop such "liquid biopsies."

Razelle Kurzrock of the UCSD School of Medicine will give a presentation about fighting cancer by better understanding a patient's unique DNA and the unique DNA of their tumor.

"By understanding genomics and each patient's cancer, we are learning how to customize precision treatments for patients," Kurzrock wrote in an email to KPBS. "The immune system, once reactivated by our new drugs, differentiates between tumor and normal cells by the mutations that tumor cells harbor."

More than 600 people are scheduled to attend the conference, which begins Thursday morning and wraps on Friday afternoon.

To view PDF documents, Download Acrobat Reader.

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San Diego Genomic Medicine Conference Kicks Off Thursday - KPBS

March 1, 2017 by Marilynn Marchione This 2009 colorized microscope image made available by the Sickle Cell Foundation of Georgia via the Centers for Disease Control and Prevention shows a sickle cell, left, and normal red blood cells of a patient with sickle cell anemia. Researchers say a French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder. The case is detailed in the March 2, 2017 issue of the New England Journal of Medicine. (Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia via AP)

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

The first-in-the-world case is detailed in Thursday's New England Journal of Medicine.

About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found. Worldwide, about 275,000 babies are born with it each year.

"Vexing questions of race and stigma have shadowed the history of its medical treatment," including a time when blacks who carry the bad gene were urged not to have children, spurring accusations of genocide, Keith Wailoo of Princeton University wrote in a separate article in the journal.

The disease is caused by a single typo in the DNA alphabet of the gene for hemoglobin, the stuff in red blood cells that carries oxygen. When it's defective, the cells sickle into a crescent shape, clogging tiny blood vessels and causing bouts of extreme pain and sometimes more serious problems such as strokes and organ damage. It keeps many people from playing sports and enjoying other activities of normal life.

A stem cell transplant from a blood-matched sibling is a potential cure, but in the U.S., fewer than one in five people have a donor like that. Pain crises are treated with blood transfusions and drugs, but they're a temporary fix. Gene therapy offers hope of a lasting one.

The boy, now 15, was treated at Necker Children's Hospital in Paris in October 2014. Researchers gave him a gene, taken up by his blood stem cells, to help prevent the sickling. Now, about half of his red blood cells have normal hemoglobin; he has not needed a transfusion since three months after his treatment and is off all medicines.

"It's not a cure but it doesn't matter," because the disease is effectively dodged, said Philippe Leboulch, who helped invent the therapy and helped found Bluebird Bio in Cambridge, Massachusetts, the company that treated the boy. The work was supported by a grant from the French government's research agency.

Bluebird has treated at least six others in the U.S. and France. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Researchers think they know why and are adjusting methods to try to do better.

Two other gene therapy studies for sickle cell are underway in the U.S.at the University of California, Los Angeles and Cincinnati Children's Hospitaland another is about to start at Harvard and Boston Children's Hospital using a little different approach.

"This work gives considerable promise" for a solution to a very common problem, said Dr. Stuart Orkin, a Boston Children's Hospital doctor who is an inventor on a patent related to gene editing.

"The results are quite good in this patient," he said of the French teen. "It shows gene therapy is on the right track."

Explore further: BCL11A-based gene therapy for sickle cell disease passes key preclinical test

More information: Gene therapy: ghr.nlm.nih.gov/primer/therapy/availability

2017 The Associated Press. All rights reserved.

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children's ...

Sickle cell disease and the blood disorder beta thalassemia affect more than 180,000 Americans and millions more worldwide. Both diseases can be made milder or even cured by increasing fetal hemoglobin (HbF) levels, but current ...

Scientists at the Center for Regenerative Medicine (CReM) at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM) are creating an induced pluripotent stem cell (iPSC)-based research library that opens ...

UCLA stem cell researchers have shown that a novel stem cell gene therapy method could lead to a one-time, lasting treatment for sickle cell diseasethe nation's most common inherited blood disorder.

A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward ...

Scientists have developed a new approach to repair a defective gene in blood-forming stem cells from patients with a rare genetic immunodeficiency disorder called X-linked chronic granulomatous disease (X-CGD). After transplant ...

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

A research team, led by the University of Minnesota, has discovered a groundbreaking process to successfully rewarm large-scale animal heart valves and blood vessels preserved at very low temperatures. The discovery is a ...

Working with yeast and human cells, researchers at Johns Hopkins say they have discovered an unexpected route for cells to eliminate protein clumps that may sometimes be the molecular equivalent of throwing too much or the ...

By changing one small portion of a stimulus that influences part of one molecule's function, engineers and researchers at Washington University in St. Louis have opened the door for more insight into how the molecule is associated ...

A minimally invasive, fiber-optic technique that accurately measures the passive stretch and twitch contraction of living muscle tissue could someday be an alternative to the painful muscle biopsies used to diagnose and treat ...

An in-depth computational analysis of genetic variants implicated in both schizophrenia and rheumatoid arthritis by researchers at the University of Pittsburgh points to eight genes that may explain why susceptibility to ...

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Gene therapy lets a French teen dodge sickle cell disease - Medical Xpress

In 2017, Sangamo is conducting a Phase 1/2 clinical trial evaluating SB-913 as an in vivo genome editing treatment for MPS II. Sangamo is also conducting Phase 1/2 studies this year evaluating in vivo genome editing treatments SB-318 for MPS I, another rare lysosomal storage disorder, and SB-FIX for hemophilia B, a rare blood disease. Data from these studies and from a clinical trial for a fourth lead program, SB-525, a gene therapy approach for hemophilia A, are expected in late 2017 or early 2018.

Sangamo's In Vivo Genome Editing Approach Sangamo's ZFN-mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific site that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein. The ability to permanently integrate the therapeutic gene in a highly specific, targeted fashion significantly differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy. Ultimately, the target population for these programs will include pediatric patients, and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient.

About Sangamo Therapeutics Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in hemophilia A and hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has a strategic collaboration with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at http://www.sangamo.com.

Forward Looking Statements This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation references relating to research and development of therapeutic applications of Sangamo's gene therapy and ZFP technology platforms, the potential of Sangamo's technology to treat hemophilia and lysosomal storage disorders, the expected timing of these clinical trials and the release of data from these trials, the impact of Sangamo's clinical trials on the field of genetic medicine and the benefit of orphan drug status. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to substantial dependence on the clinical success of lead therapeutic programs, the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this press release.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-receives-orphan-drug-designation-from-the-fda-for-sb-913-genome-editing-treatment-for-mps-ii-300415719.html

SOURCE Sangamo Therapeutics, Inc.

http://www.sangamo.com

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Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-913 Genome Editing Treatment for ... - PR Newswire (press release)

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During his address to Congress last night, President Donald Trump introduced Megan Crowley, a 20-year-old woman with a rare disease.

Crowley, who has a neuromuscular disorder known as Pompe disease, credits her survival to her father's small biotechnology company for developing and producing a therapy for the rare genetic mutation.

During the speech, the President suggested that Crowleys story highlighted the need to scale back regulations and to streamline the processes of the Food and Drug Administration. While Crowley's doctor welcomed the spotlight shown on Pompe disease and on rare diseases, in general, others cautioned that rushing experimental treatments through the Food and Drug Administration could have disastrous results.

Related: Speed up drug approvals? The FDA already did

The big problem isnt with the FDAs procedures, but rather a shortage of funds and the small numbers of people in whom scientists can study the ailments, researcher Dr. Puneet Opal told TODAY.

FDA drug approval procedures are intended to prevent patient deaths from experimental treatments, said Opal, a professor of neurology at Northwestern Universitys Feinberg School of Medicine.

"You do need the FDA to be the police dog," said Opal.

Crowleys physician, Dr. Priya Sunil Kishnani, division chief of medical genetics at the Duke University Medical Center, was excited to have rare diseases given such high-profile attention.

We diagnosed Megan at Duke in 1998 when she was just 15 months old. Its been an amazing journey to see how she has not just survived but is thriving despite her limitations, Kishnani, who is also professor of pediatrics and division chief of medical genetics, told TODAY.

Pompe disease occurs in an estimated 1 in 12,000 to 20,000 babies and people with an adult-onset form, according to Duke University experts. Crowley's fathers struggle to find a cure for the disease was profiled in the book The Cure: How a Father Raised $100 Million and Bucked the Medical Establishment in a Quest to Save His Children and also inspired a 2010 movie called Extraordinary Measures.

Megan Crowley listens as President Donald Trump addresses Congress.

Related: Parents race to save girl with rare, deadly disease

For those not familiar with Pompe, here are five things to know:

That means that a child must inherit two copies one from each parent to be affected by it. Children born with just one copy of the mutated gene are healthy and generally are completely unaware they have it.

When someone has both copies of the mutated gene, the body lacks enough of an enzyme that breaks down the starch glycogen into glucose, a form of sugar that fuels the muscles. The result is a damaging overabundance of glycogen.

Because the heart is also a muscle, damage to it is what generally kills patients even before there is respiratory distress, Opal said.

In babies, the earliest symptoms are not meeting motor milestones, Kishnani said. Babies might not hold their heads up or start rolling over at the expected age. They may not have the same strength as other babies of similar age.

"When you pick them up you feel like theyre almost slipping through your fingers," said Kishnani.

Respiratory symptoms or pneumonia might reveal a massively enlarged heart.

The severity of the symptoms and when they appear depend on the exact type of mutation. There are children who show up with worse symptoms than Crowley and others who show up with milder ones, Kishnani said.

The treatment isnt a cure for the disease, but it is lifesaving. Crowley is confined to a wheelchair and must use a ventilator to breathe.

These individuals do have a life," said Kishnani, talking about Megan's spunk and passion.

"She has life goals. People should not just look past her ... and think there are not going to be any cognitive abilities.

Read more here:
What is Pompe disease? 5 things to know about the rare gene mutation - Today.com

Could This Treatment Cure Cancer?

Despite the many advances in medicine over the last century, a cure for one of the most prevalent and devastating diseases in the world todaycontinues to evade us. But thanks to new research, that could soon change.

Kite Pharma, a US pharmaceutical company, just released the groundbreaking results of their six-month gene therapy trial: terminal cancer patients incomplete remission after just a single round.

The treatment filters a patients blood to remove T-cells, immune system cells that can be genetically engineered in a lab to identify cancer cells. Cancer cells thrive because of their ability to evade the immune system.This new therapy boosts immune cells so that they are able to eliminate cancer cells more effectively.

Patients who participated in the trial had one of three types of non-Hodgkin lymphoma. The advanced stageof their conditions meant all of them were given only a few months to live. However, following the first round of gene therapy, which took place nine months after the trial began, half the patients are not only still alive, but a third of themappear to be cured.

Among them is a 43- year-old named Dimas Padilla from Orlando, Florida whose cancer had stopped responding to chemotherapy. He completed the first round of the trials treatment last August, and his cancer is now in remission.

These results are promising and suggest that one day CAR-T cells could become a treatment option for some patients with certain types of lymphoma, said head cancer information nurse, Martin Ledwick from the Cancer Research UK, in an interview with The Telegraph.

While the results are promising and could prove to be life- changing for patients with terminal cancer, the treatment is not without risks.

Because the therapy essentially puts the human immune system to go into overdrive by radically altering human cells, complications are certainly possible some of which could be fatal. In fact, during the trial, two people diedas a resultof the therapy not because of the cancer. Some patientsimmune systems overreacted in its effort to kill the cancer cells, while others developed blood-count related issues such as anemia. Reports of patients suffering from neurological problems were also cited, but these side effectsapparently only lasted a few days.

More studies are needed tounderstand the therapys side effects, potential complications, and long-term benefits.

The trials full results wont be presented until April, and the pharmaceutical company still has to get approval from the European regulatory boards which means it will be a while yet before the therapy becomes available. Given the possible risks, it might give them enough time to study the therapy further and refine the process hopefully eliminating any adverse effects.

Although, as the Cancer Institutes Dr. Steven Rosenberg points out: Its a safe treatment, certainly a lot safer than having progressive lymphoma.

Excerpt from:
A New Cancer Treatment Has Given Terminal Patients a Second Chance at Life - Futurism

Science Daily

Many genetic changes can occur early in human development Science Daily "The diagnostics lab Baylor Genetics is one of the pioneers in this new era of clinical genomics-supported medical practice and disease gene discovery research," Lupski said. "They are developing the clinical genomics necessary to foster and support ...

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Many genetic changes can occur early in human development - Science Daily

ValueWalk

Scientists Favor Gene Editing, But Only For 'Fixing Diseases' ValueWalk An international body of scientific experts has stated, with caution, that gene editing technologies should be allowed for treating diseases or disabilities. The US National Academy of Sciences and the National Academy of Medicine said in a 200-page ... Gene Editing Could Make You SmarterFuturism all 7 news articles »

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Scientists Favor Gene Editing, But Only For 'Fixing Diseases' - ValueWalk

Researchers may be one step closer to determining the cause of schizophrenia, after uncovering an abnormal genetic process associated with the disease that begins in the womb.

By transforming skin cells from patients with schizophrenia into neuronal progenitor cells - cells that form neurons in early development - researchers identified an abnormal gene pathway called nuclear FGFR1 (nFGFR1) that impairs early brain development.

Senior study author Michal K. Stachowiak, Ph.D., of the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo in New York, and colleagues say that their findings may bring us closer to treatments that could prevent schizophrenia in utero.

The researchers recently reported their results in the journal Schizophrenia Research.

According to the National Institute of Mental Health, around 1.1 percent of adults in the United States have schizophrenia - a mental health disorder characterized by hallucinations, delusions, and abnormal thoughts.

While the exact causes of schizophrenia remain unclear, researchers have long known that the condition can run in families, suggesting a genetic origin. Furthermore, an increasing number of studies have uncovered genetic mutations associated with an increased risk of schizophrenia.

For their study, Stachowiak and colleagues sought to learn more about the genomic processes that occur in utero that might influence the risk of schizophrenia development.

To reach their findings, the researchers collected skin cells from four adults with schizophrenia and four adults without the disorder.

The skin cells were reprogrammed into induced pluripotent stem cells, and these differentiated into neuronal progenitor cells. This enabled the team to assess the processes that occur during early brain development in people with schizophrenia.

The researchers pinpointed a dysregulated nFGFR1 pathway that targets and mutates numerous genes associated with schizophrenia. The team explains that just one of these gene mutations can impact brain development.

According to the authors, these findings provide proof of concept that schizophrenia may be caused by a dysregulated genomic pathway that influences the brain before birth.

"In the last 10 years, genetic investigations into schizophrenia have been plagued by an ever-increasing number of mutations found in patients with the disease. We show for the first time that there is, indeed, a common, dysregulated gene pathway at work here."

Michal K. Stachowiak, Ph.D.

Furthermore, the team says that these findings open the door to new schizophrenia treatments. For example, a drug could be administered to expectant mothers, whose offspring has a high risk of developing schizophrenia, that prevents processes related to the disease occurring in the developing fetus.

In future studies, the researchers plan to grow "mini brains" using the same processes used in the current study, with the aim of gaining a deeper understanding of how dysregulation of the nFGFR1 pathway influences early brain development, as well as to provide a model to test possible treatments.

Learn how B vitamins might improve symptoms of schizophrenia.

Read more:
Schizophrenia begins in the womb, study suggests - Medical News Today