Stem Cell Clinic

Foundation Medicine Receives Medicare Payment in Non-Small Cell Lung Cancer under a Local Coverage … – Business Wire (press release)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. (NASDAQ:FMI) today announced that it has received payment from Palmetto GBA, the Companys Medicare Administrative Contractor (MAC) in North Carolina, for its FoundationOne comprehensive genomic profiling assay when used in the clinical course of care for individuals in the United States with Stage IIIB/IV non-small cell lung cancer (NSCLC) who meet the eligibility requirements under Palmetto GBAs Local Coverage Determination L36143 (LCD). The LCD was most recently updated on December 22, 2016. Foundation Medicine began submitting an initial set of claims to Palmetto GBA in January 2017 for FoundationOne, and received its first payments for claims under this LCD on March 1, 2017.

Coverage and payment for FoundationOne under Palmetto GBAs LCD is a positive step toward advancing access to precision medicines for individuals living with non-small cell lung cancer, said Troy Cox, chief executive officer for Foundation Medicine. We look forward to continuing to work with Palmetto GBA as we gain additional payment experience under this LCD for non-small cell lung cancer. We will continue to work with FDA and CMS as they review our universal companion diagnostic test through the Parallel Review process with the goal of being the first pan-cancer, universal companion diagnostic test to receive FDA approval and a National Coverage Determination from CMS.

About Foundation Medicine Foundation Medicine (NASDAQ:FMI) is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patient's cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Cautionary Note Regarding Forward-Looking Statements for Foundation Medicine

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding reimbursement of the Companys comprehensive genomic profiling assays, the benefits provided by anFDA-approved and CMS-covered version of the Companys universal companion diagnostic test, and progress with the Parallel Review process with FDAand CMS; the scope and timing of any approval of FoundationOne as a medical device by FDAand any potential national coverage decisions by CMS; and strategies for achievingMedicarecoverage decisions at the local or national level and new and expanded coverage from third-party payers.All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that FDA does not approve our universal companion diagnosic test as a medical device or that CMS does not decide to offer our universal companion diagnostic test as a covered benefit underMedicare; FDAor CMS is delayed in the completion of the Parallel Review process; and the risks described under the caption "Risk Factors" inFoundation Medicine's Annual Report on Form 10-K for the year endedDecember 31, 2016, which is being filed with the Securities and Exchange Commission on the date hereof, as well as other risks detailed inFoundation Medicine'ssubsequent filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andFoundation Medicine undertakes no duty to update this information unless required by law.

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Foundation Medicine Receives Medicare Payment in Non-Small Cell Lung Cancer under a Local Coverage ... - Business Wire (press release)

Gene therapy lets a French teen dodge sickle cell disease – Sioux City Journal

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 teen was the first in the world to get the treatment for sickle cell, which affects 90,000 Americans, mostly blacks. It's caused by a defective gene for hemoglobin (HE-muh-glow-bin), the stuff in red blood cells that carries oxygen. The cells curl up and clog small blood vessels, causing pain and serious problems.

The boy was given a gene to prevent the sickling, and now half of his red cells work normally. Three similar gene therapy studies are underway in the U.S.

The case is detailed in Thursday's New England Journal of Medicine.

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Gene therapy lets a French teen dodge sickle cell disease - Sioux City Journal

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

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

Cell Death in Gut Implicated in IBD | Newsroom | Weill Cornell … – Cornell Chronicle

The natural lifecycle of cells that line the intestine is critical to preserving stable conditions in the gut, according to new research led by a Weill Cornell Medicine investigator. The findings may lead to the development of new therapies to alleviate inflammatory bowel disease (IBD) and other chronic inflammatory illnesses.

In the study, published Nov. 9 in Nature, the scientists investigated the healthy turnover of epithelial cells, which are born and die every four to five days, to better understand how the gut maintains a healthy equilibrium. Because cells, called phagocytes, can clear dying cells so quickly in the body, it had been difficult to study this process in tissues. The inability to clear dying cells has been linked to inflammation and autoimmunity. Dying epithelial cells are shed into the gut lumen, so their active clearance is not necessary and they were thought to have no role in intestinal inflammation.

The investigators sought to understand whether phagocytes can take up dying epithelial cells in the gut and, if so, how these phagocytes respond. Specifically, the study tried to ascertain which genes are expressed by phagocytes after the uptake of dead cells. To answer these questions, the scientists engineered a mouse model where they could turn on apoptosis and catch phagocytes in the act of sampling dying cells. Through a series of experiments, they found that several of the genes modulated up or down in phagocytes bearing dead cells overlapped with the same genes that have been associated with susceptibility to IBD.

The mouse model used in the study enables the visualization of a dying red cell within the green fluorescently-labeled small intestinal epithelial cells. The green outline of villi shown delineates the single cell layer of the intestinal epithelium. Cell nuclei are shown in blue. Weill Cornell Medicine investigators tracked dying intestinal epithelial cells into the underlying phagocytes (not visible), and asked how their uptake modulates gene expression in those phagocytes.

The fact that there was an overlap shows that apoptosis must play a role in maintaining equilibrium in the gut, said Dr. Julie Magarian Blander, a senior faculty member in the Jill Roberts Institute for Research in Inflammatory Bowel Disease at Weill Cornell Medicine who was recently recruited as a professor of immunology from Mount Sinai. This study identified cell death within the epithelium as an important factor to consider when thinking about therapeutic strategies for patients with IBD.

In their experiments, the scientists expressed a green fluorescent protein fused to the diphtheria toxin receptor within intestinal epithelial cells of mice, which made them visible under a microscope and sensitive to diphtheria toxin. They injected into these mice a carefully titrated dose of toxin into the intestinal walls of mice to induce cell death. Then the team examined the phagocytes that turned green after they internalized dead cells. Macrophages, one kind of phagocyte, expressed genes that help process the increased lipid and cholesterol load they acquired from dying cells. Dendritic cells, another type of phagocyte, activated genes responsible for instructing the development of regulatory CD4 T cells, a class of suppressive white blood cells. Notably, both phagocytes expressed a common suppression of inflammation gene signature.

Because the same genes that confer susceptibility to IBD were modulated in response to apoptotic cell sampling, the research indicates that a disruption of the phagocytes immunosuppressive response would have consequences for homeostasis or stable conditions in the gut.

We know there is excessive cell death, inflammation and microbial imbalance in IBD, so the prediction is that the immunosuppressive program in phagocytes, associated with natural cell death in the gut epithelium, would be disrupted, Dr. Blander said. The goal in the treatment of IBD is to enhance healing in the gut, but now we know that this also helps phagocytes restore their immunosuppressive and homeostatic functions. We think this would translate into helping patients stay in remission. Theres a lot to learn from phagocytes and we may be able to target the same pathways they use to suppress inflammation in patients with IBD.

The study validates the importance of healing in the mucosa, or lining, of the intestine as a therapy and enhances the understanding of that process. The next phase of Dr. Blanders research will be to investigate how the inflammatory conditions of IBD alter cell death and the homeostatic immunosuppressive functions of intestinal phagocytes, and to do so in both mouse models and different groups of IBD patients undergoing anti-TNF therapy at the Jill Roberts Center for Inflammatory Bowel Disease at New York-Presbyterian and Weill Cornell Medicine.

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Cell Death in Gut Implicated in IBD | Newsroom | Weill Cornell ... - Cornell Chronicle

D-backs’ De La Rosa counting on stem-cell therapy to avoid second Tommy John – Arizona Sports (registration) (blog)

Rubby De La Rosa had already undergone Tommy John surgery. So when his right elbow became an issue again, he had a tough decision to make.

He could have the surgery again and risk that it might not be as effective the second time around. Or he could venture down another avenue.

Either way, his career was at stake.

De La Rosa was in his second season as a Diamondback when it was interrupted in September by a familiar pain in his elbow.

His rookie season with the Dodgers in 2011 was cut short for the same reason. He ended up having his first Tommy John procedure.

So when the issue arose again last season, he and Dr. James Andrews who performed De La Rosas first Tommy John surgery discussed the options. They decided stem-cell therapy would be more effective than a second surgery. De La Rosa then received one stem cell injection in late September and another in December.

He has been playing catch and said he feels 100-percent healthy, but he will not begin throwing off the mound until mid-March.

What I see from it right now, its working, said De La Rosa, who will be 28 years old March 4. No more pain, no more soreness. Just waiting for my time.

Dr. Keith Jarbo is an orthopedic surgeon with OrthoArizona who specializes in elbow surgery. Jarbo, who has performed many Tommy John surgeries to repair the ulnar collateral ligament, said the procedure is less effective the second time around.

Stem-cell therapy has become more popular in orthopedics over the past five to 10 years, Jarbo said. Some doctors even use it in addition to Tommy John surgery to accelerate the healing process. He said stem cells are used because they are pluripotent, meaning they can develop into every type of cell that makes up the body.

They have a lot of the growth factors that are necessary for healing, Jarbo said. We think they can be important adjuncts for healing.

However, there is a risk. Jarbo said there is no research that compares the efficacy of stem-cell therapy to that of Tommy John surgery. With the lack of research, Jarbo said one doctor may be using different types of stem cells than another.

He said he doesnt use stem cells and wont until there is more research that shows it is effective. Until then, he cant assure his patients that stem-cell therapy will produce a ligament that has similar characteristics to what it did before the injury.

Its high-risk in the sense that we dont know exactly what its going to do, Jarbo said. We think that it promotes healing, but we dont know exactly what growth factors are within or whats going to happen.

I dont think youre necessarily getting a new, better tissue as if youre developing a robot. Youre really just trying to get good healing and strong appropriate tissue, whether thats with surgery or not.

Jarbo estimated Andrews conducts over 100 Tommy John surgeries per year. He said doctors like Andrews may be helpful in researching stem-cell therapys effectiveness if they can use stem cells on half of their patients and compare the results with the half that received Tommy John surgery.

De La Rosa is part of a group of trendsetters. Last season, Los Angeles Angels of Anaheim pitchers Garrett Richards and Andrew Heaney both received stem cell injections to stave off Tommy John surgery. Heaney ended up needing the surgery anyway.

D-backs manager Torey Lovullo said De La Rosa seems encouraged by his health.

We just know that Rubby is in a really good place, hes been throwing the ball really well hes smiling, and hes back to himself, which is always a good sign for him, he said.

Diamondbacks pitching coach Mike Butcher said stem-cell therapy is unpredictable, but seems to helping De La Rosa.

Its obviously helped out Rubby, Butcher said. The results have been good so far; he feels great. Now its just a matter of working toward the progression of where we can get him off the mound.

Jarbo said the largest risk with using stem-cell therapy may be that players are rushed back to action through an accelerated rehab program.

However, De La Rosa has been patient throughout his now five-month recovery.

He hopes it means the end of his elbow pain.

Its frustrating because sometimes you want to pitch but you cannot pitch because theres a lot of soreness, he said. When you do it right and you get hurt and everything switches, now you cant pitch with your whole motion and you have to limit a lot of things.

You get bad pitching.

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D-backs' De La Rosa counting on stem-cell therapy to avoid second Tommy John - Arizona Sports (registration) (blog)