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Having a brain storm

Local doctor works on Israeli stem-cell project to fight ALS

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Dr. Tony Fiorino with Dr. Yossef Levy, BrainStorm’s vice president of cell production, at the clean room facility at Hadassah Medical Center.

While people across the world raised huge sums for ALS research this summer by dousing themselves in the Ice Bucket Challenge, Teaneck physician Dr. Tony Fiorino has become chief executive officer of an Israeli company whose revolutionary stem-cell therapy technology could be an answer for neurodegenerative diseases, including ALS.

The progressive neuromuscular disease amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a death warrant. There is no known cure. Victims gradually lose the ability to move, swallow, and breathe. According to the ALS Association, every year two in 100,000 people are diagnosed in the United States. They usually are between 55 and 65 years old.

Petach Tikva-based BrainStorm Cell Therapeutics is in phase 2 testing of NurOwn, a novel technique innovated at Tel Aviv University for growing and modifying adult stem cells to treat ALS. Stem cells from the patients’ own bone marrow are enhanced in order to secrete elevated levels of nerve-growth factors that protect existing motor neurons, promote motor neuron growth, and reestablish nerve-muscle interaction.

Following clinical trials in Israel, where about 60 percent of patients treated with NurOwn showed improvement, a larger three-site safety trial approved by the U.S. Food and Drug Administration is beginning at Massachusetts General Hospital, the University of Massachusetts Memorial Hospital, and the Mayo Clinic.

We spoke with Dr. Fiorino, 46, a member of Teaneck’s Congregation Rinat Yisrael, about his involvement with BrainStorm and the potential of NurOwn.

Jewish Standard: What did you do before BrainStorm?

Dr. Tony Fiorino: I earned my MD and Ph.D. from the Albert Einstein College of Medicine, and I studied liver stem cells for my Ph.D. thesis, which was long before stem cells became so popular. But I actually have spent most of my career on Wall Street, as a biotechnology and pharmaceuticals analyst and portfolio manager at firms including JP Morgan, Citigroup, and Pequot Capital. In 2008 I left Pequot to run a start-up biotechnology company that we financed, and I really enjoyed the operational side of drug development. With BrainStorm, I was given the opportunity to join an Israeli biotech company with amazing science and really promising early clinical data, which was simply too interesting to pass up.

JS: How is NurOwn distinct from other companies developing stem-cell therapeutics for treating diseases like ALS?

TF: We have a directed approach. Many other companies in the stem-cell space take a shotgun approach, in which they use early stem cells that possess the potential to turn into many different cell types. By contrast, we have taken adult stem cells and converted them into factories that pump out growth factors known to promote the survival of neurons. Thus we know exactly where we want to use them; there are no mysteries. We’re focused on diseases in which neurons are dying. This is really a platform technology with applications outside of ALS, such as Parkinson’s disease and multiple sclerosis, though ALS is our lead indication.

JS: Please describe the study now beginning in Massachusetts and Minnesota.

TF: This is primarily a safety study involving 48 patients at sites headed by important thought leaders in the field of ALS. In keeping with FDA guidelines for a randomized, double-blind study, 36 of the subjects will randomly be chosen to receive NurOwn and 12 will receive a placebo. Enrollment will take 12 to 15 months, and there will be six months of follow-up, so we expect the study to be completed in early 2016.

JS: What did previous studies find?

TF: The first study, led by Professor Dimitrios Karussis at Hadassah Medical Center, looked at patients getting NurOwn through intramuscular injections or in the spinal column via lumbar puncture. In the second study, everyone received both types of injections. That was partially driven by not understanding if ALS spreads from the central nervous system to the periphery or vice versa, so we treated both. The study also looked at a range of doses, and we’re still following up on the last few cases.

In both Israeli studies, some patients showed unexpected stabilization or even temporary improvements in terms of their function and respiration. Some had several months of stabilization, although unfortunately some did not respond. About 60 percent of subjects in the two studies getting NurOwn by lumbar puncture had slowing of progression, stabilization, or improvement, and we saw that injecting NurOwn intramuscularly alone doesn’t do much. In the current U.S. studies we’re getting a much bigger data set.

JS: What is your role in taking BrainStorm to the next level?

TF: BrainStorm has never had a U.S.-based employee, much less a U.S.-based CEO, so my joining the company represents a real shift. My immediate tasks are, first, to make sure the phase 2 clinical trial is running smoothly, and second, to tell the BrainStorm story to U.S. biotech investors, most of whom haven’t heard of BrainStorm. Beyond that, it is my goal to move additional programs into clinical trials over the next one to two years. We have established an excellent safety profile on the basis of more than 30 ALS patients treated in Israel at Hadassah, and we have quite interesting data in animal models of other neurodegenerative diseases. As a small company, our resources are limited, so we can’t do everything we want at once. That is why the dialogue with investors is so important. They ultimately provide the capital that biotechnology companies like ours need in order to develop innovative therapies.

JS: As a practicing Jew, how do you feel about the Israeli role in pioneering new hope for ALS patients?

TF: It is a source of great pride. Our technology came out of the lab of Professor Dani Offen at Tel Aviv University and was nurtured and transformed into a clinical program by BrainStorm entirely in Israel. But, of course, ALS and other neurodegenerative diseases do not observe national, ethnic, or religious borders, and drug development is a global business, so the time had come to move our clinical development beyond its Israeli roots. We certainly hope and pray that NurOwn will eventually emerge as a therapy that offers benefits to ALS patients around the world, and in that way will reflect back positively on Israel.

JS: Your thoughts on the Ice Bucket Challenge?

TF: I think the Ice Bucket Challenge has been an amazing thing to watch – and on a personal note, a very wet and cold thing to participate in. A substantial amount of money has been raised that would have gone elsewhere, so that is a great thing for ALS research, and more importantly down the road, for ALS patients. BrainStorm probably will not directly benefit from the Challenge. But the ALS Association, which has generated so much publicity and galvanized so much charitable giving through this campaign, supports a good deal of important research in ALS – and they have a much bigger pie to slice up now. If they will offer grant programs relevant to us, then we will certainly pursue those opportunities. In any case, we benefit indirectly from the attention that the Ice Bucket Challenge has brought to this relentless and devastating disease.

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