BrainStorm Cell Therapeutics Inc (BCLI)

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Autologous stem cells sound a cord

“Enrichment is the hottest trend in neurodegenerative disease space,” asserts Ralph Kern, MD, chief operating officer and chief medical officer, Brainstorm Cell Therapeutics. “The oncology field was drastically transformed by utilizing genetic insights. In the neurosciences, we can generate a world of analytics from just a few drops of cerebrospinal fluid.”

Success in ALS clinical trials has remained elusive, with only a couple of new treatments approved in the past decade. Kern is certain that this phenomenon is largely due to the considerable heterogeneity in ALS symptoms. To overcome this problem, Brainstorm invests in biomarkers of cerebrospinal fluid to gain insights into disease mechanisms and to optimize patient stratification.

The company’s lead product for ALS, NurOwn®, contains injectable autologous mesenchymal stem cells (MSCs) that have been differentiated in vitro and stimulated to produce neurotrophic factors (NTFs). NTFs are known to promote the survival, repair, and regeneration of neurons. NurOwn® is injected directly into the spinal fluid, bringing it in immediate contact with affected motor neurons in the spinal cord. Changes in NTFs, inflammatory biomarkers, and biomarkers of neurodegenerative damage can be assessed directly from the same port.

The Phase II data demonstrated reduction in rate of ALS disease progression in the NurOwn treatment group. Cerebrospinal fluid biomarkers confirmed the secretion of NTFs and a reduction in inflammatory activity in the patients treated with NurOwn. The treatment effect was statistically significant in a subpopulation of participants with more rapid disease progression.

Kern predicts that the regulatory agencies will have to change the way they shape the drug development process, shifting from a preoccupation with common efficacy signals and large heterogeneous populations, to a willingness to favor unique efficacy signals and small homogeneous populations. “Signal readout is most easily observed in rapid progressors,” notes Kern. “[That is where] we can see meaningful slowing of the disease.”

He envisions that biological “software” solutions will become more prevalent, solutions that attempt to reprogram the disease environment by using the body’s own cells and repeatedly delivering new information. During Brainstorm’s manufacturing process, autologous MSCs are cryopreserved, with multiple doses of treatment being prepared from one bone marrow aspirate. This approach can generate multiple treatment doses while reducing the need for additional aspirates.

Full article link

https://www.genengnews.com/insights/neurodegenerative-treatments-build-to-a-tipping-point/