Tomer Kornfeld, Calcalist
ALS, also known as Lou Gehrig’s Disease, is a incurable disease that attacks suddenly and causes degeneration as a result of damage to the motor-nerve cells. Many companies have tried to find a cure for a disease that about 10,000 patients are diagnosed with annually in the U.S. alone, but without success. One company that does think it’ll be the first to find a cure for ALS patients, however, is the Israeli biotech firm Brainstorm Cell Therapy.
Brainstorm, established in 2004, is developing an ALS treatment using stem cells, recently deciding to focus its energies to treating ALS as part of continuing research into the use of technology to treat degenerative diseases of the nervous system such as Parkinson’s and damage to the spinal column.
The company’s offices are located in the Kiryat Aryeh neighborhood of Petach Tikvah, with the majority of its employees research and development people headed by Prof. Eldad Melamed, Director of Neurology at Beilinson. Brainstorm’s chairman is Rami Efrati, who joined about a year ago. Previously, Efrati served as vice chairman at Nayis in the field of business-development and marketing sales. The main shareholder at the company is Chaim Liebovitz, a religious businessman who dealt in crude oil and gold sales and decided a year-and-a-half ago to invest five million dollars in the company.
Trials on only eight patients
In an interview with Calcalist, Lebovitz and Efrati explain why Brainstorm decided to focus on treating ALS, discussing the energies expended to find an investor who would enable the company to succeed with clinical trials and why they believe that Brainstorm’s technology will succeed where other companies failed.
How does the company’s technology work, and what are its advantages?
Efrati: “The technology is based upon taking stem cells from the bone marrow of the patient and, using our exclusive technology, turning them into cells that produce proteins that have a vital function in the nervous system. Those cells, after being re-injected into the patient, will change the environment of growth for the nerve cells and make it possible to continue their function and by doing so, stop the downward progression of the disease.”
The company’s chief advantage is the use of adult stem cells which are considered very reliable for use, in contrast to fetal stem cells which carry a heavy risk of side effects. The firm’s technology introduces special properties to the stem cells extracted from patients, which make protection of the nerve cells possible. The fact that the cells are taken from the patient represents an important advantage in preventing complications and avoiding the need for immunosuppressant medications.
Why do you believe that your technology is really bound to succeed helping ALS patients?
“The technology is based on numerous tests carried out in the company’s labs, at Tel Aviv University, and jointly with Rutgers University in New Jersey. In tests we found that the stem cells which we isolated and grew with the exclusive technology that we developed, produces large quantities of essential growth agents for the function of nerve cells, including those with ALS.”
In addition, injections made with various models in animals showed that in fact, the injected cells which survived for a long time protected the nerve cells from damage and even led to their proliferation. In additional tests we found that we can isolate stem cells from the bone marrow of ALS patients, and we hope that injections to affected areas will stop degeneration and maybe improvement of nerve function.”
Despite that Brainstorm’s lab results seem rosy, treating humans is far more problematic. More than one treatment that demonstrates effectiveness in animals does not demonstrate those same results in humans. To this day, Brainstorm has not tried treating ALS patients, since it is based on research results at Tel Aviv University that were intended for treating Parkinson’s Disease, which is why it was centered primarily on this disease. When Liebovitz came to the company, he decided to change the company’s focus from Parkinson’s to ALS patients, but without abandoning Parkinson’s.
Chaim Liebovitz, why did you decide to focus more on ALS over Parkinson’s?
Because ALS is an incurable disease which strikes young people and is considered an orphaned disease—the FDA gives many breaks to companies that develop treatments for it. Thus, for example, in ALS safety trials, the trial is only carried out on eight patients, in contrast to the large number of patients used in treatment trials for Parkinson’s, which already has about 40 drugs to treat it on the market.”
Do you have enough money to fund continued development?
“Since July of 2007 I have invested about 4 million dollars out of the 5 which I have committed to them. In the near future I’ll inject more cash into the company per our agreement. In addition to funds coming from me as an investor, the company gets support from the chief scientist for its 2008 activities, and will get support in 2009 as long as the company has enough resources to become independent at the end of 2009.”
When do you plan to move from laboratory-testing phase to human-trial phase?
We are making every effort to allow us to go to human clinical tests in the second or third quarter of 2009. Human trials are more expensive and because of that, we’ll need additional funding. In the coming week Rami and I will join Prof. Eldad Melamed at an important medical conference in New York. Prof. Melamed will summarize our technology and the para-clinical Parkinson’s and ALS trials we conducted. After that, we will be meeting with 10 companies, among them a number of big firms that expressed interest in investing in the company. To be able to go from the research phase to the trial phase, we are seeking to collect 15 million dollars, which will allow us to pave the whole way up to Phase III in ALS trials.”
Brainstorm is a public company you won’t find in the limelight. It trades under BCLI in the millions on the NASDAQ exchange. Liebovitz would almost certainly prefer if it weren’t publicly traded at all. He even doesn’t get excited by the stock price’s recent skydive together with the entire market, asserting that “if our trial succeeds, our company won’t sell for five million but for at least 100 times as much.”