Stem Cell Therapies For ALS, or Lou Gehrig’s disease: The Problem And New Clinical Trials

Two of our Stem Cell Sector companies, Neuralstem (CUR) and Brainstorm Cell Therapeutics (BCLI.OB) are in clinical trials with stem cell therapies for Amyotrophic lateral sclerosis (ALS), also known as Lou Gherig's disease. Neuralstem (CUR), market cap $60M, began its ALS trial in 2008 in the United States.

BrainStorm, market cap $57M, is the first company to receive approval from the Israeli Ministry of Health (MOH) for a differentiated stem cell therapy and approval of its Phase I/II clinical trial utilizing its proprietary NurOwn™ treatment for patients with Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig's Disease. BrainStorm's trial effectively began in Isreal on June 6,2011.

On July 19, 2011, BrainStorm announced it will partner with UMass Medical School and its chair of neurology, Robert H. Brown Jr., DPhil, MD, in anticipation of applying for FDA approval to begin amyotrophic lateral sclerosis (ALS) human clinical trials in the United States. The trial, which will be conducted at Massachusetts General Hospital, will investigate the use of the company’s NurOwn treatment for ALS.

We've included a video talk by Fred Gage of the Salk Institute on the subject of how ALS works in the body.

Nueralstem's first patient in Phase I clinical trial was dosed on January 21, 2010 at Emory University in Atlanta Georgia.  The trial will ultimately consist of up to 18 ALS patients.  To date, 12 patients have been treated.  With the completion of the first three patient groups (totaling 12 patients) Nueralstem has reached a planned pause in the trial while the FDA reviews the results to date. The Principal Investigator for the trial presented interim safety data on the first nine patients in April.  All nine ALS patients remain alive and there were no unresolved serious adverse reactions related to surgery. Of the three ambulatory patients who were treated, all remain ambulatory with no serious adverse events secondary to surgery. The Company hopes to get clearance to resume the trial by the end of the summer.

Fred Gage, Professor of Genetics at the Salk Institute and a stem cell research pioneer, is not related to either trial or the company conducting it. He did, however, deliver the 13th annual Kenneth Myer Lecture in Australia in December of 2009 captured in a five part video series. In part 3 of that series, he speaks of the problems associated with a stem cell therapy for ALS or Lou Gherig's disease. Watching him discuss it gives a context for the clinical trials on ALS now being conducted by the two Stem Cell Sector companies. Gage's lab had previously shown that, contrary to accepted dogma, human beings are capable of growing new nerve cells throughout life. Small populations of immature nerve cells are found in the adult mammalian brain, a process called Neurogenesis.

Here is part 3 of the 5 part video of his Kenneth Myer Lecture:

 

 

This is what Neuralstem says about the basis for the stem cell therapy it's developed:

Our technology enables the isolation and large-scale expansion of human neural stem cells from all areas of the developing human brain and spinal cord, thus enabling the generation of physiologically relevant human neurons of all types. What differentiates our stem cell technology from others is that our patented processes do not require us to direct our cells towards a certain fate by adding specific growth factors. Our cells actually “become” the type of cell they are fated to be.

This process and the resulting cells comprise a technology platform that allows for the efficient isolation and production, in commercially reasonable quantities, of neural stem cells from the human brain and spinal cord.

This is what Brainstorm Therapeutics says about its stem cell therapy:

Our NurOwn™ technology processes adult human Mesenchymal stem cells that are present in bone marrow and are capable of self-renewal as well as differentiation into many cell types. The research team is among the first to have successfully achieved the in vitro differentiation of adult bone marrow cells (animal and human) into cells capable of releasing neurotrophic factors, including glial-derived neurotrophic factor (GDNF) by means of a specific differentiation-inducing culture medium.

We wish both companies the best of luck in these important trials.

 

 

 

 

Comments

[Susan]

I know that at least one patient has had extremly positive results from these trials.

[Susan]

The science works. The issue is and it sounds the same for both studies is they have not been able to replicate it as of yet. I am heavily involved with one of these studies and can assure you that with one patient miraculous results have been seen.