Brainstorm Recycles ALS Stem Cell Data to Boost Sagging Stock
January 11, 2016 - als
“Breakthrough,” “revolutionary” and “dramatic” were adjectives deployed by Brainstorm Cell Therapeutics (BCLI) in an email representation mouth-watering reporters to a press discussion being hold Monday morning. The association is announcing results of a tiny clinical hearing of an initial stem-cell therapy in patients with amyotrophic parallel sclerosis (ALS), a degenerative and deadly haughtiness illness also famous as Lou Gehrig’s disease.
A some-more honest outline of a tangible Brainstorm ALS investigate information would be recycled, vague and rarely promotional.
Israel-based Brainstorm initial announced formula of a investigate one year ago. In 12 patients receiving an injection of their possess cultivated branch cells, a course of ALS during 6 months was slowed compared to baseline, totalled by an alleviation in an ALS illness astringency measure or an boost in lung function.
As we wrote during a time, Brainstorm’s stem-cell therapy looked earnest in ALS patients though a information were early and distant from decisive since a investigate usually enrolled a handful of patients and all were treated during a same Israeli hospital. Most importantly, a investigate lacked a comparator arm so there was unequivocally no approach to decider objectively how good a branch dungeon therapy was working.
In April, Brainstorm updated a same investigate during a neurology meeting, stretched to embody research of 14 ALS patients. The formula were similar, again display a negligence of ALS course when patients were assessed 6 months after injection of a branch dungeon therapy compared to baseline.
Which brings us to Monday’s press discussion in San Francisco, where investors are entertainment for a J.P. Morgan Healthcare Conference. The usually new expansion is a announcement of a same investigate in this week’s book of JAMA Neurology. The branch dungeon therapy formula are unvaried from what was reported by Brainstorm in April.
Brainstorm is creation a large understanding about a recycled stem-cell information since a batch cost is in difficulty — down 30% over a past year. The association has prolonged found it formidable to remonstrate institutional investors in a U.S to buy a stock. Brainstorm’s financier family bid grew some-more severe in Sep when U.S.-based CEO Tony Fiorino quiescent and was transposed by Chairman (and largest shareholder) Chaim Lebovits.
Brainstorm’s ALS therapy, NurOwn, starts with a harvesting of bone pith from any ALS patient. Mesenchymal branch cells are removed from a bone pith representation and sent to a lab, where a exclusive Brainstorm routine initial expands their numbers and afterwards transforms them into specialized cells secreting haughtiness expansion factors. [Neuralstem (CUR – Get Report) is building a identical stem-cell therapy for ALS though ran in difficulty final year with unsatisfactory clinical hearing results.]
After a duty and potential of a NurOwn cells are confirmed, a “drug” is delivered behind into a ALS studious around injections into flesh and a spine. Once injected, a NurOwn branch cells wash a shop-worn neurons of ALS patients with secretions of haughtiness expansion factors.
When Fiorino was CEO of Brainstorm final year, a Nurown proviso I/II investigate formula were earnest though inconclusive. Under Lebovitz, a same information are insubordinate and a breakthrough for ALS patients.
In a company’s email representation to reporters final week, Dr. Dimitrios Karussis of Hadassah Medical Organization in Israel, where a investigate was conducted, is quoted as saying, “Close to 90% of patients who were injected intrathecally [in a spine] were responders to a diagnosis possibly in terms of their respiratory duty or their ubiquitous engine disability.”
The “close to 90%” response to NurOwn, however, includes any ALS studious in a investigate who showed any negligence of their illness progresson. And Brainstorm was usually means to consider 10 of a 14 enrolled ALS patients during 6 months after a NurOwn injection.
Without a remedy arm for comparison, interpreting a formula of a proviso I/II investigate is challenging, during best.
Brainstorm is conducting a larger, proviso II investigate in a U.S. right now that randomizes ALS patients to diagnosis with NurOwn or a placebo. Results are approaching after this year.