BrainStorm Cell Therapeutics Inc. announced the publication of an article in the Journal of the Academy of Public Health that examines how regulatory frameworks for amyotrophic lateral sclerosis, rare diseases and regenerative medicine could be updated, according to the company.
The piece, co-authored by BrainStorm board member and former FDA associate commissioner Peter J. Pitts and BrainStorm president and CEO Chaim Lebovits, highlights tools the authors say could accelerate development in small, heterogeneous patient populations, including validated biomarkers, surrogate endpoints, adaptive trial designs, Bayesian statistics and earlier sponsor-regulator alignment.
The article also points to advances in genomics, artificial intelligence and precision therapeutics as drivers of change.
BrainStorm describes its NurOwn platform as an autologous mesenchymal stem cell approach for neurodegenerative disease; the company claims NurOwn has orphan drug designations from the U.S. Food and Drug Administration and the European Medicines Agency, that a Phase 3 trial in ALS has been completed, and that a Phase 3b study is set to launch under a Special Protocol Assessment with the FDA.
