"Breakthrough," "revolutionary" and "dramatic" were adjectives deployed by Brainstorm Cell Therapeutics (BCLI) in an email pitch inviting reporters to a press conference being held Monday morning. The company is announcing results of a small clinical trial of an experimental stem-cell therapy in patients with amyotrophic lateral sclerosis, or ALS, the degenerative and fatal nerve disease also known as Lou Gehrig's disease.
A more honest description of the actual Brainstorm ALS study data would be recycled, inconclusive and highly promotional.
Israel-based Brainstorm first announced results of its study one year ago. In 12 patients receiving an injection of their own cultivated stem cells, the progression of ALS at six months was slowed compared to baseline, measured by an improvement in an ALS disease severity score or an increase in lung function.
As I wrote at the time, Brainstorm's stem-cell therapy looked promising in ALS patients but the data were early and far from definitive because the study only enrolled a handful of patients and all were treated at the same Israeli hospital. Most importantly, the study lacked a comparator arm so there was really no way to judge objectively how well the stem cell therapy was working.
In April, Brainstorm updated the same study at a neurology meeting, expanded to include analysis of 14 ALS patients. The results were similar, again showing a slowing of ALS progression when patients were assessed six months after injection of the stem cell therapy compared to baseline.
Which brings us to Monday's press conference in San Francisco, where investors are gathering for the J.P. Morgan Healthcare Conference. The only new development is the publication of the same study in this week's edition of JAMA Neurology. The stem cell therapy results are unchanged from what was reported by Brainstorm in April.
Brainstorm is making a big deal about the recycled stem-cell data because its stock price is in distress -- down 30% over the past year. The company has long found it difficult to convince institutional investors in the U.S to buy its stock. Brainstorm's investor relations effort grew more challenging in September when U.S.-based CEO Tony Fiorino resigned and was replaced by Chairman (and largest shareholder) Chaim Lebovits.
Brainstorm's ALS therapy, NurOwn, starts with the harvesting of bone marrow from each ALS patient. Mesenchymal stem cells are isolated from the bone marrow sample and sent to a lab, where a proprietary Brainstorm process first expands their numbers and then transforms them into specialized cells secreting nerve growth factors. (Neuralstem (CUR) is developing a similar stem-cell therapy for ALS but ran in trouble last year with disappointing clinical trial results.)
After the function and potency of the NurOwn cells are confirmed, the "drug" is delivered back into the ALS patient via injections into muscle and the spine. Once injected, the NurOwn stem cells bathe the damaged neurons of ALS patients with secretions of nerve growth factors.
When Fiorino was CEO of Brainstorm last year, the Nurown phase I/II study results were promising but inconclusive. Under Lebovitz, the same data are revolutionary and a breakthrough for ALS patients.
In the company's email pitch to reporters last week, Dr. Dimitrios Karussis of Hadassah Medical Organization in Israel, where the study was conducted, is quoted as saying, "Close to 90% of patients who were injected intrathecally [in the spine] were responders to the treatment either in terms of their respiratory function or their general motor disability."
The "close to 90%" response to NurOwn, however, includes any ALS patient in the study who showed any slowing of their disease progression. And Brainstorm was only able to assess 10 of the 14 enrolled ALS patients at six months after the NurOwn injection.
Without a placebo arm for comparison, interpreting the results of the phase I/II study is challenging, at best.
Brainstorm is conducting a larger phase II study in the U.S. right now which randomizes ALS patients to treatment with NurOwn or a placebo. Results are expected later this year.