A phase 2 clinical trial to evaluate the safety and efficacy of treatment in individuals with prodromal to mild Alzheimer disease (AD) is planned for the autologous neurotrophic factor-secreting mesenchymal stem cell (MSC-NTF)(NurOwn; BrainStorm Cell Therapeutics, New York, NY). The program is being studied for treatments for amyotrophic lateral sclerosis (ALS) (NCT03280056) and multiple sclerosis (MS) (NCT03799718) as well.
"Our newly announced AD program is an important strategic expansion of BrainStorm's clinical pipeline," said Chaim Lebovits, chief executive officer of BrainStorm. "We see this as an ideal complement to our lead ALS program, as there are compelling data suggesting that the benefits observed with NurOwn in ALS patients may extend to other neurologic conditions, including AD. As we move towards completion of our pivotal phase 3 ALS study, the newly announced AD program, combined with our ongoing progressive MS program, will continue to drive the advancement of the NurOwn platform. Through the completion of our clinical trial programs, BrainStorm hopes to advance NurOwn to address unmet needs across a broad population of patients with neurodegenerative disease."
The study will be a 52-week, phase 2 open-label, proof-of-concept clinical trial to evaluate the MSC-NTF in 40 participants with prodromal to mild AD. The study participants will receive 3 intrathecal doses 8 weeks apart. In addition to meeting well-defined clinical criteria for prodromal to mild AD, participants must also meet biomarker defined criteria for AD. The clinical trial will evaluate safety and pharmacodynamics of the treatment, including effects on inflammatory AD-specific neurodegenerative and synaptic biomarkers, as well as a range of key clinical measures of cognition and function.
In the ALS phase 2 trial, treatment-related adverse events were mild to moderate and self-limiting, including injection site and back pain, pyrexia, headache, and arthralgia, consistent with a transient reaction to the transplantation. After treatment, most adverse events were related to progression of the underlying ALS, and the risk-benefit ratio for MSC-NTF cells remains positive.
Allie Massaro, MD
Claire Smyth, BSc; David Roberts, BSc; and Kenneth Monaghan, PhD