BrainStorm Launches Phase 3 ALS Clinical Trial in the US

NTFs, coming into its NurOwn? BrainStorm’s NurOwn aims to promote the survival of motor neurons in people with ALS by increasing levels of neurotrophic substances (see September 2017 news). [Image: Neurons in the brain. Public Domain.]

The potential stem cell therapy NurOwn is soon to be evaluated in the ALS clinic at the phase 3 stage. The strategy, developed by BrainStorm Cell Therapeutics in Israel, aims to promote the survival of motor neurons in people with ALS by using mesenchymal stem cells, isolated from their bone marrow, and expanded and differentiated ex vivo, to deliver neurotrophic factors (NTFs) including BDNF, GDNF and HGF into the cervical spinal cord (see Petrou et al., 2016).

The approach builds on previous work, which suggests that a combination of these substances may be needed to help protect motor neurons against the disease (see April 2017 news; Krakora et al., 2013Schaller et al. 2017). A total of 200 people with ALS are expected to participate in the double-blind, randomized, placebo-controlled 28-week clinical trial. Sites will include California Pacific Medical Center, Massachusetts General Hospital, the Mayo Clinic, University of California-Irvine and the University of Massachusetts Medical School. The clinical trial launched at Massachusetts General Hospital on September 11, 2017.

Researchers first turned to NTFs in the early 1990s as a potential treatment for ALS in hopes to promote the survival of motor neurons injured by the disease. But these potential therapies have proved challenging to develop (see Rogers, 2014). A key obstacle is how to deliver these substances to motor neurons affected by the disease. To overcome these challenges, a growing number of researchers, including Brainstorm’s scientists, are turning to stem cells to produce these substances in key tissues damaged by ALS including the spinal cord.

Meanwhile, Cedar Sinai Medical Center’s Clive Svendsen in Los Angeles, California is evaluating a different strategy in hopes to protect motor neurons in people with the ALS. The strategy involves the transplantation of  GDNF-producing human neural progenitor cells into the spinal cord. A phase 1 clinical trial is ongoing.


Petrou P, Gothelf Y, Argov Z, Gotkine M, Levy YS, Kassis I, Vaknin-Dembinsky A, Ben-Hur T, Offen D, Abramsky O, Melamed E, Karussis D. Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis: Results of Phase 1/2 and 2a Clinical Trials. JAMA Neurol. 2016 Mar;73(3):337-44. [PubMed].

Karussis D, Karageorgiou C, Vaknin-Dembinsky A, Gowda-Kurkalli B, Gomori JM, Kassis I, Bulte JW, Petrou P, Ben-Hur T, Abramsky O, Slavin S. Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis.Arch Neurol. 2010 Oct;67(10):1187-94. [PubMed].

Further Reading

Rogers, ML. Neurotrophic Therapy for ALS/MND. New York: Springer New York; c2014. p. 1755-85. (Kostrzewa RM, editor. Handbook of Neurotoxicity.)

Schaller S, Buttigieg D, Alory A, Jacquier A, Barad M, Merchant M, Gentien D, de la Grange P, Haase G. Novel combinatorial screening identifies neurotrophic factors for selective classes of motor neurons. Proc Natl Acad Sci U S A. 2017 Mar 21;114(12):E2486-E2493. [PubMed].

Krakora D, Mulcrone P, Meyer M, Lewis C, Bernau K, Gowing G, Zimprich C, Aebischer P, Svendsen CN, Suzuki M. Synergistic effects of GDNF and VEGF on lifespan and disease progression in a familial ALS rat model. Mol Ther. 2013 Aug;21(8):1602-10. [PubMed].

Gould TW, Enomoto H. Neurotrophic modulation of motor neuron development. Neuroscientist. 2009 Feb;15(1):105-16. [PubMed].


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BDNF brainstorm clinical trial disease-als HGF neurotrophic factor topic-clinic topic-randd
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