The potential stem cell therapy NurOwn is soon to be evaluated in the ALS clinic at the phase 3 stage. The strategy, developed by BrainStorm Cell Therapeutics in Israel, aims to promote the survival of motor neurons in people with ALS by using mesenchymal stem cells, isolated from their bone marrow, and expanded and differentiated ex vivo, to deliver neurotrophic factors (NTFs) including BDNF, GDNF and HGF into the cervical spinal cord (see Petrou et al., 2016).
The approach builds on previous work, which suggests that a combination of these substances may be needed to help protect motor neurons against the disease (see April 2017 news; Krakora et al., 2013; Schaller et al. 2017). A total of 200 people with ALS are expected to participate in the double-blind, randomized, placebo-controlled 28-week clinical trial. Sites will include California Pacific Medical Center, Massachusetts General Hospital, the Mayo Clinic, University of California-Irvine and the University of Massachusetts Medical School. The clinical trial launched at Massachusetts General Hospital on September 11, 2017.
Researchers first turned to NTFs in the early 1990s as a potential treatment for ALS in hopes to promote the survival of motor neurons injured by the disease. But these potential therapies have proved challenging to develop (see Rogers, 2014). A key obstacle is how to deliver these substances to motor neurons affected by the disease. To overcome these challenges, a growing number of researchers, including Brainstorm’s scientists, are turning to stem cells to produce these substances in key tissues damaged by ALS including the spinal cord.
Meanwhile, Cedar Sinai Medical Center’s Clive Svendsen in Los Angeles, California is evaluating a different strategy in hopes to protect motor neurons in people with the ALS. The strategy involves the transplantation of GDNF-producing human neural progenitor cells into the spinal cord. A phase 1 clinical trial is ongoing.
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