Patients & Caregivers
AN OPEN LETTER TO THE ALS COMMUNITY
Over the past few years, tremendous progress has been achieved in terms of increasing both ALS funding and research, due in large part to the ALS community’s tireless efforts and dedication. This commitment has brought much-needed and long-overdue attention and resources to this heartrending condition. And as a result, after decades of clinical failure, there are now three recently approved therapies available and more than a dozen investigational treatments in the clinic.
At BrainStorm Cell Therapeutics, we have experienced the community’s energy and emotion through both good and hard times, and particularly now as we near a decisive moment in the development of NurOwn. In the past few months, we have witnessed with great concern as community discussions about NurOwn, particularly via social media, have become increasingly more heated and personalized. To that end, we feel the need to articulate our stance around communications about NurOwn.
• We firmly believe that the appropriate place to evaluate NurOwn is at the upcoming FDA Advisory Committee meeting on 9/27/23. As is the case with most ALS research, the NurOwn clinical program produced extremely complex results, which required significant time and resources as well as intensive engagement with the study’s investigators and ALS experts. Through this process, the FDA, with counsel from its Advisory Committee members, will be in a position to thoughtfully evaluate the full body of scientific evidence around NurOwn and consider whether to apply regulatory flexibility, given the continued dire need for new ALS treatments.
• It is impossible to have a productive dialogue about NurOwn via social media, and that is why we have avoided engaging in social threads. The topic is complex, and these platforms simply do not have the capacity or ability to support a thoughtful conversation. That said, we are committed to transparency. To that end, we have publicly shared new data and regulatory updates as soon as we were able to do so, including publication of the full data package summarizing all pre-specified endpoints in all participants. We have subsequently reviewed this information with advocates and advocacy groups with the goal of keeping our key constituents informed.
• We appreciate the reality that the ALS community will continue to discuss matters that are of great interest to all, and experience shows that these discussions will be robust. However, we ask that anyone engaged in discussions about NurOwn express their views not only with passion, but also with compassion and respect. Disagreements are not only expected, they are helpful. We ask for mutual respect be shown to everyone, whether or not they agree with your view. We will model this behavior ourselves and sincerely hope others will refrain from personal attacks.
Moving forward, we will continue to post relevant information via social media, but will not engage in debates about NurOwn. We believe the best way to serve the ALS community is to focus our full attention on preparation for NurOwn’s upcoming FDA Advisory Committee meeting and the next steps that will follow. We hope to address any outstanding concerns in this forum.
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 may lose their ability to speak, eat, move and breathe.
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.
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 1 / 2
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.
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.
Pre-approval Access Policies
BrainStorm Cell Therapeutics is dedicated to developing innovative cellular therapies for highly debilitating neurodegenerative diseases.
BrainStorm Cell Therapeutics offered an expanded access program which enrolled by invitation only, to our investigational agent, autologous MSC-NTF cellular therapy for patients who completed the Phase 3 trial and met inclusion criteria. This program is now closed. Learn More