BrainStorm Cell Therapeutics has just announced that they have completed enrollment in their Phase 3 clinical trial for amyotrophic lateral sclerosis (ALS). Enrollment was initiated way back in October of 2017.
ALS is a rare disease that currently has no approved treatment. The neurodegenerative condition is debilitating and fatal. Hopefully, this new trial will bring renewed hope to patients.
This Phase 3 investigation will examine the effects of Autologous MSC-NTF cells, called NurOwn, in ALS.
NurOwn is BrainStorm Cell Therapeutics Autologous Mesenchymal Stem Cells (MSCs)-NTF cells. These cells have already received Orphan Drug Designation from both the FDA and the EMA for ALS. The MSCs are derived from bone marrow. These cells are then expanded ex vivo and differentiated. Next, they are converted into the MSC-NTF cells by being placed under conditions that cause the cells to secrete high amounts of neurotrophic factors.
The NurOwn cells deliver NTFs as well as immunomodulatory cytokines to tissues in the body which are damaged. This direct delivery is supposed to allow the cells to slow or at least stabilize the progression of disease.
Researchers believe this therapy has promise for treating the disease pathways of neurodegenerative disorders, including ALS.
About The Trial
This trial includes 200 participants. It is a randomized and placebo-controlled trial that will examine the effect of repeat intrathecal administration of the NurOwn cells in ALS patients.
Researchers say that a “significant percentage” of the patients have already received three of the NurOwn treatments. They expect that by October of next year, all participants will be finished with the treatment.
This investigation is being conducted at six different trial sites.
- UC Irvine
- Cedars-Sinai Medical Center
- UMass Medical School
- Massachusetts General Hospital
- Mayo Clinic
- California Pacific Medical Center
This trial is funded by the California Institute for Regenerative Medicine (CIRM) CLIN2-0989. CIRM works specifically in stem cell therapies for underserved patient populations. They provided 15.9 million dollars for this Phase 3 trial. The support from CIRM is the reason three trial sites are located in California.
Multiple Sclerosis Trial
NurOwn is also being examined in a Phase 2 clinical trial for progressive multiple sclerosis (MS). Approval for this trial was granted by the FDA back in December of 2018. Since March 2019, enrollment has been open. This trial is still recruiting patients.
This is an open-label and multi-center trial. It will examine repeat intrathecal dosing of the cells in MS patients.
BrainStorm Cell Therapeutics expects to report their findings from this Phase 3 trial for ALS before the end of 2020. Their hope, and expectation, is that they will be able to file a biological license application for NurOwn following the completion of this investigation.
If this application is approved by the FDA, ALS patients could finally have an effective treatment option.
You can read more about this trial here.