Stacy Lindborg, PhD, on Addressing Unmet Needs in ALS With Cell Therapy

The co-CEO of BrainStorm Cell Therapeutics spoke about the current standards of care in ALS and the potential of NurOwn to address unmet needs.

“I think we all appreciate that ALS is a devastating disease... The typical survival from onset of symptoms to death is 2 to 5 years on average. There are 3 products approved: riluzole which was approved in 1995 and showed about a 90 day survival benefit; edaravone was approved in 2017—so almost 22 years later—and showed a reduction in the progression as measured by the ALS Functional Rating Scale; and then Relyvrio was approved last year, which also showed a reduction in the rate of decline and function as measured by the scale. We're excited to see new therapies coming to the market, but there remains unmet need.”

Amyotrophic lateral sclerosis (ALS) is characterized by degeneration and loss of cortical and spinal motor neurons, resulting in progressive muscle atrophy, weakness, and respiratory failure. While there are currently 3 FDA-approved products for the treatment of ALS, significant unmet needs remain for patients with the disease.

BrainStorm Cell Therapeutics recently announced that it will have an Advisory Committee Meeting with the FDA to discuss its biologics license application for NurOwn, an autologous bone marrow-derived mesenchymal stem cell therapy intended to treat ALS.

In an interview with CGTLive™, Stacy Lindborg, PhD, the co-CEO of BrainStorm Cell Therapeutics, spoke about the current standard of care for patients with ALS, going over the effects of riluzole, edaravone, and Relyvrio. She noted that while these options do provide some benefit, significant unmet needs remain. Next, Lindborg discussed how NurOwn, which was recently evaluated in a phase 3 clinical trial (NCT03280056), could potentially address some of these unmet needs. She touched on how NurOwn functions, noting that it is engineered to secrete high levels of neurotrophic factors, and some of the preclinical research regarding the therapy. Afterwards Lindborg went over the efficacy data generated during NurOwn’s clinical development and discussed the impact of the floor effect, which is of particular relevance for the upcoming Advisory Committee Meeting.

1. Brainstorm Cell Therapeutics announces FDA advisory committee meeting to review biologics license application for NurOwn® for the treatment of ALS. News release. BrainStorm Cell Therapeutics Inc. March 27, 2023. Accessed March 27, 2023.
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