Patients & Caregivers
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.
About Progressive MS
Multiple sclerosis (MS) is a chronic neurological disease that causes damage to nerve cells in the central nervous system (CNS). Damage to the myelin sheath insulating the nerve fibers and to the nerve fibers themselves disrupts the transmission of signals between the CNS and the rest of the body. MS can affect numerous areas of the CNS and cause a wide variety of neurologic symptoms.
There are multiple types of MS. The most common form of MS is relapsing-remitting MS (RRMS, 85% of MS patients), characterized by periods of relapse, with flare-ups or exacerbations of symptoms, and periods of remission. Progressive MS, includes both primary progressive MS (PPMS) and secondary progressive MS (SPMS), is characterized by a worsening of neurologic function and increase in disability over time. About 10% of people with MS are diagnosed with PPMS and about half of those diagnosed with RRMS will eventually transition to SPMS.
A number of disease-modifying therapies approved for treatment of MS have been found to reduce the number of relapses, modestly delay progression of disability, and limit new disease activity. Unfortunately, there are currently no available therapies that halt the progression of established disability or result in significant functional improvement in progressive MS.
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.
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.
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
There are numerous organizations that offer valuable education and support to individuals diagnosed with ALS and their families.
ALS Advocacy & Research Organizations