Amyotrophic lateral sclerosis (ALS), also known as Motor Neuron Disease (MND) and Lou Gehrig’s Disease, is a progressive neurodegenerative disease that affects 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 that initiate and control voluntary movements. With the progressive loss of voluntary muscle action, ALS patients may lose the ability to speak, eat, move and breathe.
ALS affects tens of thousands of people around the world, with a prevalence of about 4-7 per 100,000 people. Only about 5%-10% of ALS cases are familial, which means the disease is inherited from a parent. The vast majority of cases are sporadic, arising without any prior family history of ALS.
Although scientists are developing an increasingly sophisticated understanding of the disease, these scientific advancements have not yet led to the development of therapies that can halt or slow the progressive loss of motor function in this disease. In the US, the drug riluzole is approved for the treatment of ALS, which can extend survival. Recent years have brought a wealth of new scientific understanding regarding the physiology of this disease.
BrainStorm’s NurOwn® in ALS
We believe NurOwn®, or MSC-NTF cells, may offer promise as a potential therapy for ALS. You can find a detailed description of our technology here, but briefly, NurOwn® is manufactured from a patient’s own bone marrow cells.
We isolate mesenchymal stem cells from the bone marrow, expand them, and then grow them under special, proprietary conditions that induce the cells to secrete growth factors known to help neurons live longer under a variety of conditions. Our hope is that by delivering these cells close to the site of damaged neurons in ALS patients, we can help those neurons live longer. In a mouse model of ALS, NurOwn® cells extended both function and survival.
We have conducted several clinical trials in ALS patients, resulting in compelling findings– click here for more information on these studies.
Statement on Compassionate Use
We are currently not able to offer compassionate use of or expanded access to NurOwn®, owing to limitations on our manufacturing and our need to complete the clinical development program that we hope will prove that NurOwn® is safe and effective for treating ALS.