Update (March 18, 2013):The Mayo Clinic in Rochester, Minn., has agreed to conduct a clinical trial of BrainStorm Cell Therapeutics’ adult stem cell treatment for ALS. The facility is the third U.S. clinical site to sign a letter of understanding about conducting a trial; the others are the University of Massachusetts and Massachusetts General, BrainStorm said in a press release. The company says it plans to enroll patients in clinical trials at the three facilities as early as the second half of 2013.
Update (Feb. 5, 2013):BrainStorm Cell Therapeutics announced in a Feb. 4, 2013, press release that the first trial participant has begun treatment in the company’s phase 2a dose-escalating clinical trial for ALS.
The Israeli Ministry of Health has given BrainStorm Cell Therapeutics approval to accelerate its current phase 1-2 safety trial of the company’s NurOwn stem cell therapy in amyotrophic lateral sclerosis (ALS).
A new phase 2a dose-escalating trial, designed to evaluate the safety and preliminary efficacy of the experimental therapy in ALS, will be launched immediately at the Hadassah Medical Center in Jerusalem. Last year, the company reported preliminary data that showed the experimental therapy was well-tolerated, and that some functional improvements were seen in participants.
According to a BrainStorm press release issued Jan. 7, 2013, approval for the acceleration was based on evaluation of the first 12 (of an expected total of 24) study participants in the company’s phase 1-2 trial at Hadassah.
In the phase 2a trial, the second group of 12 participants will receive combined intramuscular and intrathecal (into the fluid around the spinal cord) administration of NurOwn cells, with increasing doses. The study participants, who already have been recruited, will be followed for three to six months after transplantation.
Early data indicated treatment effect
Treatment for the first trial participants in the phase 1-2 trial — people with early-stage ALS — involved a single round of multiple injections to the biceps and triceps muscles.
Preliminary data based on results from the first four trial participants in the phase 1-2 trial was reported Jan. 17, 2012. The experimental therapy did not cause any significant side effects; in addition, clinical follow-up of trial participants indicated that the treatment appeared to have improved breathing, swallowing and muscle strength.
NurOwn technology uses participants' own cells
The experimental treatment in BrainStorm's trial uses mesenchymal stem cells, taken from the bone marrow, which are capable of differentiating (maturing) into a number of different cell types. In this trial, mesenchymal stem cells are taken from trial participants’ bone marrow, cultured into healthy cells capable of delivering neurotrophic factors (molecules that support motor neurons, the nerve cells that die in ALS) and then readministered to the participants from whom they were taken.
BrainStorm, with operations in both New York and Petach Tikyah, Israel, was granted orphan drug status for its experimental therapy in February 2011 by the U.S. Food and Drug Administration (FDA). (Orphan drug status provides economic incentives for companies to develop drugs for rare diseases.)
In related news, BrainStorm announced in a Dec. 17, 2012, press release that it has partnered with Octane Biotech of Kingston, Ontario, to develop a bioreactor designed to optimize the NurOwn stem cell production process. The customized bioreactor is expected to enable BrainStorm to significantly increase its production capabilities, while reducing associated costs and time.
BrainStorm plans to add US trial sites
Pending FDA approval, BrainStorm plans to add U.S. trial sites to the ongoing NurOwn clinical trial in 2013. Prospective sites include Massachusetts General Hospital in Boston and the University of Massachusetts Medical School in Worcester, Mass.
Several steps are necessary before NurOwn stem cells can be tested in the United States. These include:
To find out more about the ongoing trial in Israel, view Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF) in ALS Patients (enter NCT01051882 into the search box on ClinicalTrials.gov).