Patients & Caregivers

Special Protocol Assessment (SPA) for Phase 3b Trial in ALS

Brainstorm received written agreement from the U.S. Food and Drug Administration (FDA), under a Special Protocol Assessment (SPA), on the design for a Phase 3b trial of NurOwn® in amyotrophic lateral sclerosis (ALS). 

The SPA agreement with the FDA validates the clinical trial protocol and statistical analysis of the planned Phase 3b trial of NurOwn, demonstrating their adequacy for addressing objectives that support a future BLA (Biologics License Application) in ALS.

BrainStorm anticipates commencement of the Phase 3b study in 2024, after reviewing the protocol with investigators, securing study site Institutional Review Board approvals, and engaging with appropriate members of the ALS community.

The Phase 3b trial (Study BCT-006-US) will be a two-part, multicenter, study designed to assess the efficacy and safety of NurOwn in patients with ALS. The entry criteria will enroll participants earlier in the course of their disease, having the onset of ALS symptoms, including limb weakness, within the prior 24 months, and upright slow vital capacity >=65% of predicted for gender, height and age. Patients will also be allowed to receive concomitant treatment of an approved standard of care.

Part-A is a double blind, placebo-controlled period of 24 weeks duration. Up to approximately 200 patients are planned to be enrolled and randomized 1:1 to NurOwn or placebo treatment groups. There will be a screening period of six to nine weeks, during which eligible participants will undergo a single bone marrow aspiration procedure to procure the mesenchymal stem cells (MSCs) that will be used to manufacture each participant’s NurOwn treatment for the duration of the trial. Patients will then be randomized 1:1 and treated with NurOwn or placebo via three repeated intrathecal injections, once every eight weeks. All eligible patients who complete Part-A will have the option of entering Part-B, open-label extension period of 24 weeks duration, where all participants will receive three repeated intrathecal injections of NurOwn, once every eight weeks.

The primary efficacy endpoint is the change in the Revised Amyotrophic Lateral Sclerosis Functional Rating (ALSFRS-R) total score from baseline to Week 24. Primary inference from the trial will be based on a p-value from the combined assessment of function and survival (CAFS) to account for mortality observed in the trial. Cerebrospinal Fluid (CSF) and blood samples will be collected for analysis of biomarkers of neuroinflammation, neurodegeneration, and neuroprotection. An independent Data Monitoring Committee (DMC) will monitor the safety of the trial participants.

About ALS

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, is a progressive disease that causes damage to cells in the brain and spinal cord known as motor neurons. Motor neurons transmit signals from the brain to the muscles. When motor neurons become damaged and eventually die, the brain can no longer control muscle actions.

The motor neurons affected in ALS are those that initiate and control voluntary movements. With the progressive loss of voluntary muscle action, patients with ALS will lose their ability to speak, eat, move and breathe.

Clinical Trials

BrainStorm’s investigational cellular therapy is being studied in ALS and progressive MS.

Autologous mesenchymal stem cells secreting neurotrophic factors (MSC-NTFs) are being studied as an investigational treatment for ALS and MS.

Autologous MSC-NTF cells are manufactured from a patient’s own bone marrow cells. Briefly, we isolate mesenchymal stem cells (MSCs) from the patient’s bone marrow and then grow them under special conditions to induce the cells to secrete multiple growth factors known to be important in the nervous system. The autologous MSC-NTF cells are then injected into the cerebrospinal fluid.


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previous clinical trials

Prior clinical studies demonstrated autologous MSC-NTF cellular therapy is safe and well-tolerated in ALS.

BrainStorm has completed four clinical trials of our investigational cellular therapy in ALS. All four trials were designed to determine the safety and tolerability of autologous MSC-NTF cell administration.

Phase Location Design Patients
Phase 1 / 2 Israel A single-arm, open-label phase 1/2 study 12
Phase 2a Israel A single-arm, open-label phase 2a study 14
Phase 2 United States A randomized, double-blind, placebo-controlled phase 2 study 48
Phase 3 United States Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients 200


VIEW STUDY RESULTS

Phase 3 Clinical Trial in ALS

BrainStorm has completed a randomized, double-blind, placebo-controlled Phase 3 trial of autologous MSC-NTF cells following repeat administration in patients in ALS at six U.S. sites (NCT03280056). Learn more.

The trial took place at the following centers.

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Phase 2 Clinical Trial in MS

Our investigational cellular therapy offers promise as a potential treatment option for progressive MS. Find out more about how autologous MSC-NTF cells are developed.

BrainStorm has completed a phase 2 open-label trial using repeat-administration of autologous MSC-NTF cells in progressive MS (NCT03799718). Learn more.

View additional information on the clinical development programs for BrainStorm’s autologous MSC-NTF cell therapy on our Pipeline page.

Pre-approval Access Policies

BrainStorm Cell Therapeutics is dedicated to developing innovative cellular therapies for highly debilitating neurodegenerative diseases with a lead program in Amyotrophic Lateral Sclerosis (ALS).

Expanded Access / Compassionate Use Policy

BrainStorm Cell Therapeutics is devoted to people living with ALS and their loved ones. We acknowledge that some people living with ALS may be unable to participate in a clinical trial and their physicians might seek investigational treatments through Expanded Access also known as Compassionate Use. However, due to the personalized nature of this autologous cellular therapy and the need to complete the Phase 3b trial, access to the investigational therapy is limited to enrollment in this clinical trial. Our commitment to initiating and completing the Phase 3b trial is focused on securing approval, which will ensure the potential therapy becomes available if successful.

If you have additional questions, feel free to reach out to us via our contact page.
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resources

There are numerous organizations that offer valuable education and support to individuals diagnosed with ALS and their families.

ALS Advocacy & Research Organizations

ALS Association

ALS Hope Foundation

ALS ONE

ALS Society of Canada

ALS Therapy Development Institute (ALS TDI)

Answer ALS

Compassionate Care ALS (CCALS)

End the Legacy

EverythingALS

Her ALS Story

Hope Loves Company (HLC)

Hop On a Cure

I AM ALS

International Alliance of ALS/MND

IsrA.L.S.

Les Turner ALS Foundation

Live Like Lou

Motor Neurone Disease Association (MNDA)

Muscular Dystrophy Association (MDA)

Northeast ALS Consortium (NEALS)

Paralyzed Veterans of America (PVA)
Project ALS

Target ALS

Team Gleason

The Mappers

ALS United

ALS Network

ALS Northwest

ALS United Arizona

ALS United Connecticut

ALS United Georgia

ALS United Greater Chicago

ALS United Greater New York

ALS United Mid-Atlantic

ALS United Nevada

ALS United New Mexico

ALS United North Carolina

ALS United Ohio

ALS United Orange County

ALS United Rhode Island

ALS United Rocky Mountain

ALS United San Diego

MS Advocacy and Research Organizations

Accelerated Cure Project (ACP)
Can Do MS
The Consortium of Multiple Sclerosis Centers
Multiple Sclerosis Association of America (MSAA)
Multiple Sclerosis Coalition (MSC)
Multiple Sclerosis Foundation
National Multiple Sclerosis Society

Regenerative Medicine Organizations

Americans for Cures
Alliance for Regenerative Medicine (ARM)
California Institute for Regenerative Medicine (CIRM)

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