Brainstorm Cell Therapeutics has received a $16 million grant from a state agency known as the California Institute for Regenerative Medicine to develop a Phase 3 clinical trial of its ALS stem-cell therapy NurOwn.
NurOwn is based on mesenchymal stem cells, which can transform themselves into various types of cells. Brainstorm transforms stem cells harvested from patients into cells that secrete neurotrophic factors, which promote nerve tissue growth. The treatment can be injected into muscle or the spinal canal to help nerve cells survive.
Brainstorm plans to enroll about 200 patients in the trial, which will be conducted at six ALS specialty centers in the United States.
The primary objective of the study will be to see if NurOwn can improve patients’ functional impairment. The measuring tool investigators will use is the ALS Functional Rating Scale-Revised, or ALSFRS-R.
Researchers will optimize patient population to include the patient profiles that seemed to respond better to NurOwn in the Phase 2 ALS trial (NCT02017912).
The Phase 2 study assessed NurOwn’s safety and effectiveness in 48 patients. The effectiveness yardsticks were ALSFRS-R and slow vital capacity, a measure of breathing capacity.
NurOwn provided the patients with meaningful benefits, compared with a placebo, according to the trial results. It also was safe, and patients tolerated it well.
The study will begin after the company receives U.S. Food and Drug Administration and Institutional Review Board approval. The board looks at ethical considerations of proposed research.
“We are honored to be awarded this CIRM [state agency] grant, and appreciate the support of CIRM in the development of NurOwn,” Chaim Lebovits, Brainstorm’s president and chief executive officer, said in a press release. “This substantial award provides further support for our technology and clinical program, and recognizes the importance of developing effective treatments for patients afflicted with ALS.”
“CIRM is partnering with Brainstorm to follow up on the company’s promising Phase 2 trial in patients with ALS,” said Maria Millan, the agency’s interim president and chief executive officer. “CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurologic condition for which there is currently no cure.”
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