The muscle wasting caused by Lou Gehrig's disease - amyotrophic lateral sclerosis (ALS) - is quick and fatal, with many not surviving five years after diagnosis. Internal organs eventually shut down, leading to respiratory failure. But promising stem cell research from laboratories at Tel Aviv University is the basis of a new drug that could stop the wasting process and improve survival odds.

 

It's not a long shot but a treatment based on 10 years of research with a new "orphan drug" status allocated by the US Food and Drug Administration. Clinical trials are now underway in Jerusalem's Hadassah University Medical Center under the supervision of Prof. Dimitrios Karussis, a renowned stem-cell transplant expert and head of Hadassah's Multiple Sclerosis Center. Early clinical evidence being collected by Israeli company BrainStorm Cell Therapeutics, which owns the license to commercialize the research if it succeeds, suggests that the specially modified stem cells - a treatment called NurOwn - can help slow the progression of the disease.

 

According to the American ALS Association, about 5,600 people in the United States are diagnosed with ALS each year and as many as 30,000 Americans may have the disease at any given time. The only drug on the market as of 2011 is Riluzole, improving survival by only a few months. It cannot reverse neuron damage and it causes liver damage in about 10 percent of people taking the drug.

 

 

More effective solutions are needed. But with little known about the cause of ALS and how it develops in the body, new treatments have been hard to come by.

 

BrainStorm, a Petah Tikva company originally founded to commercialize a drug for Parkinson's, has developed a methodology useful for a number of motor-brain related disorders, using adult stem cells from a person's own body to treat dysfunction. This niche area presents greater possibilities for success in the short term. Earning "orphan drug" status - for formulations that address diseases for which there are no solid treatments - also accelerates the process through regulatory hurdles. BrainStorm is currently testing NurOwn, an injectable active compound, on 24 patients in Jerusalem. Half have just been diagnosed with ALS, and the other half are in a later stage of the disease.

 

Those willing to travel to Israel and commit to at least nine months, if they meet eligibility requirements, will have the opportunity to be part of this clinical study, says BrainStorm's acting CEO Adrian Harel. As is the case with all clinical trials, a third party is managing the applicants.

 

 

Unlike embryonic stem cell therapies, NurOwn's use of a patient's own stem cells comes with "no issue of rejection or danger of evolving into cancer," Harel says. Developed in the labs of Professors Daniel Offen and Eldad Melamed of Tel Aviv University's Sackler Faculty of Medicine and Felsenstein Medical Research Center, NurOwn helps the body produce neuroprotectors, proteins that protect the brain against neurodegenerative disorders and will hopefully slow the progression of ALS. Research supporting this revolutionary advance was published by the professors two years ago in the Journal of Stem Cells. Developed over a period of 10 years, the drug could be available in as little as three years if the clinical trials prove successful.
 
After testing in a mouse model, "we were able to show that the bone marrow-derived stem cells prevent degeneration in the brain following injection of selective neurotoxins," says Offen. "Researchers also demonstrated that transplantation of these cells increased the survival rate in the mouse model of ALS and significantly delayed the progress of motor dysfunction."
 
After the Jerusalem clinical trials are complete and safety and efficacy evaluated, BrainStorm plans to run trials at Massachusetts General Hospital in collaboration with the Medical School of the University of Massachusetts.

 

NurOwn may also hold promise for treating both Parkinson's and Huntington's diseases, Harel says. Typical treatments today only address the symptoms, not the underlying cause.