Clinical development programs
There is significant unmet need for effective therapies for people living with highly debilitating and often fatal neurodegenerative diseases. Using the NurOwn® platform, BrainStorm is focused on realizing the potential of autologous cellular therapies.
Program overview
Program
Indication
PreclinicalPhase 1Phase 2Phase 3
Status
Preclinical studies are ongoing to determine the potential for autologous MSC-NTF cells in Huntington’s disease, Parkinson’s disease and autism spectrum disorder.
Programs in detail
- 01
NurOwn in ALS
The Phase 3b trial design agreed with the FDA under a Special Protocol Assessment, the four completed studies, and the ALSFRS-R endpoint explained.Read more - 02
Progressive multiple sclerosis
A completed Phase 2 open-label trial of repeat administration in primary and secondary progressive MS.Read more
References
Peer-reviewed publications- 1 Brown RH, Al-Chalabi A. Amyotrophic lateral sclerosis. N Engl J Med. 2017;377:162-172.
- 2 Cedarbaum JM, Stambler N, Malta E, et al. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. J Neurol Sci. 1999;169(1-2):13-21.
- 3 Leigh PN, Swash M, Iwasaki Y, et al. Amyotrophic lateral sclerosis: a consensus viewpoint on designing and implementing a clinical trial. Amyotroph Lateral Scler Other Motor Neuron Disord. 2004;5:84-98.
- 4 Kaufmann P, Levy G, Thompson JL, et al. The ALSFRSr predicts survival time in an ALS clinic population. Neurology. 2005;64:38-43.
- 5 Castrillo-Viguera C, Grasso DL, Simpson E, et al. Clinical significance in the change of decline in ALSFRS-R. Amyotroph Lateral Scler. 2010;11:178-180.
- 6 Lublin FD, et al. Defining the clinical course of multiple sclerosis. Neurology. 2014;83:278-286.
- 7 Confavreux C, Vukusic S. Natural history of multiple sclerosis: a unifying concept. Brain. 2006;129:606-616.
- 8 Chataway J. Tackling progression in multiple sclerosis. Editorial. Lancet Neurol. June 2018.Volume and page numbers are not given on the live site.
- 9 Harris VK, et al. Characterization of autologous mesenchymal stem cell-derived neural progenitors as a feasible source of stem cells for central nervous system applications in multiple sclerosis. Stem Cells Transl Med. 2012;1:536-547.
- 10 Martins LF, et al. Mesenchymal stem cells secretome-induced axonal outgrowth is mediated by BDNF. Sci Rep. 2017;7:4153. doi:10.1038/s41598-017-03592-1
- 11 Rivera FR, Aigner L. Adult mesenchymal stem cell therapy for myelin repair in multiple sclerosis. Biol Res. 2012;45:257-268.
BrainStorm's autologous MSC-NTF cell therapy (NurOwn®, debamestrocel) is investigational and has not been approved by the FDA or any other regulatory authority.