On October 11th, Brainstorm Cell Therapeutics announced that the Israeli Ministry of Health had granted clearance for a Phase I/II clinical trial using the company’s autologous NurOwn™ stem cell therapy in patients with ALS (pending completion of validation studies).
To find out more about what this means to the field, and why Brainstorm has decided to focus on ALS research, I spoke with the Director and CEO of the company, Mr. Rami Efrati. Mr. Efrati has worked with Brainstorm for three years, having previously served for seven years as Vice President of Sales, Business Development, and Marketing at NICE Systems.
In a nutshell, Brainstorm’s therapy involves extracting stem cells from a patient’s bone marrow, inducing these cells to differentiate into supportive neurological cells, and injecting these cells back into the same patient at the site of damage. Because the stem cells are taken from a patient’s own marrow, there is a reduced risk of the body rejecting them once they are re-transplanted. The adult nature of the stem cells also allows Brainstorm to sidestep any ethical or moral controversy associated with embryonic stem cell research. While the therapy has potentially far-reaching applications to a variety of neurological diseases, Brainstorm’s focus remains, for the time being, on ALS.
“Brainstorm originally started working on Parkinson’s disease. Two years ago, our company made the decision to focus our efforts on diseases which would bring us as quickly as possible to clinical trials in order to help people,” said Mr. Efrati. As ALS was among the diseases that were candidates for this targeted approach, “…we wanted to invite ALS patients to come to discuss their experience with the disease. I kept hearing the name Avichai Kremer [CEO and co-founder of Prize4Life,] and I said, ‘who is he?’ We invited him in, and we became good friends. I came to believe in the mission of Prize4Life, and believe in Avi’s fight against ALS.”
Avi’s enthusiasm that ALS research was at a tipping point was apparently quite infectious. Choosing to focus on a disease that was ripe for a breakthrough was not just a marketing decision; it was an attempt to get a promising therapy to suffering patients quickly. As Chaim Lebovits, President of Brainstorm, said shortly after the announcing of the trial, “…the greatest satisfaction comes from saving a human life, not to mention thousands of lives; G-d willing, I am hopeful we can achieve that.”
But the decision was not received well by those wary of the return on investment. ALS is considered an orphan disease, meaning that because it is so rare (a disease with orphan designation must affect fewer than 200,000 individuals according to US criteria,) there is little financial incentive for companies to invest in the research. “People asked me, ‘Are you crazy? This is not logical,’” Mr. Efrati recalled. But the figures seemed more encouraging to Brainstorm. “I explained to our skeptical investors ‘There are at least 100,000 people with ALS in the Western World [author’s note: and 600,000 worldwide]. This means an estimated $6 billion market. And our therapy has the potential to be applicable to many disorders of the central nervous system, such as Multiple Sclerosis and Parkinson’s disease’. I think they found this persuasive and worth the many risks.” All told, Brainstorm estimates the potential global market for these diseases at $214 billion.
The trial will begin after the screening of patients. “The clinical trial will be headed by medical doctors at Hadassah Medical Center, under the direction of Prof. Dimitrios Karussis,” explained Mr. Efrati. “Because this is the first time we are treating a human being, the first phase of our trial will be focused entirely on safety.”
But researchers are hopeful that after the safety of the procedure is assured, they will be able to track the efficacy of the treatment. “Imagine that soon, if a doctor tells you that you have ALS, he or she can also say that there is a treatment that will slow or stop the progression of this disease. This will be an exciting moment.”
Certainly there are thousands of eyes on this trial, eager to share in that excitement. The trial will recruit 24 patients and take place in Jerusalem. Potential candidates will be screened by a panel of doctors affiliated with the Hadassah Medical Center.
For more information on the trial, visit the National Institute of Health’s database of clinical trials here.
To learn more about Brainstorm Cell Therapeutics, you can visit their website at www.brainstorm-cell.com.
For more information about the Hadassah University Medical Center, go to www.hadassah.org.il/english.