At CIRM we don’t have a disease hierarchy list that we use to guide where our funding goes. We don’t rank a disease by how many people suffer from it, if it affects children or adults, or how painful it is. But if we did have that kind of hierarchy you can be sure that Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, would be high on that list.
ALS is a truly nasty disease. It attacks the neurons, the cells in our brain and spinal cord that tell our muscles what to do. As those cells are destroyed we lose our ability to walk, to swallow, to talk, and ultimately to breathe.
As Dr. Maria Millan, CIRM’s interim President and CEO, said in a news release, it’s a fast-moving disease:
“ALS is a devastating disease with an average life expectancy of less than five years, and individuals afflicted with this condition suffer an extreme loss in quality of life. CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurological condition for which there is currently no cure.”
Having given several talks to ALS support groups around the state, I have had the privilege of meeting many people with ALS and their families. I have seen how quickly the disease works and the devastation it brings. I’m always left in awe by the courage and dignity with which people bear it.
I thought of those people, those families, today, when our governing Board voted to invest $15.9 million in a Phase 3 clinical trial for ALS run by BrainStorm Cell Therapeutics. BrainStorm is using mesenchymal stem cells (MSCs) that are taken from the patient’s own bone marrow. This reduces the risk of the patient’s immune system fighting the therapy.
After being removed, the MSCs are then modified in the laboratory to boost their production of neurotrophic factors, proteins which are known to help support and protect the cells destroyed by ALS. The therapy, called NurOwn, is then re-infused back into the patient.
In an earlier Phase 2 clinical trial, NurOwn showed that it was safe and well tolerated by patients. It also showed evidence that it can help stop, or even reverse the progression of the disease over a six month period, compared to a placebo.
CIRM is already funding one clinical trial program focused on treating ALS – that’s the work of Dr. Clive Svendsen and his team at Cedars Sinai, you can read about that here. Being able to add a second project, one that is in a Phase 3 clinical trial – the last stage before, hopefully, getting approval from the Food and Drug Administration (FDA) for wider use – means we are one step closer to being able to offer people with ALS a treatment that can help them.
Diane Winokur, the CIRM Board Patient Advocate member for ALS, says this is something that has been a long time coming:
CIRM Board member and ALS Patient Advocate Diane Winokur
“I lost two sons to ALS. When my youngest son was diagnosed, he was confident that I would find something to save him. There was very little research being done for ALS and most of that was very limited in scope. There was one drug that had been developed. It was being released for compassionate use and was scheduled to be reviewed by the FDA in the near future. I was able to get the drug for Douglas. It didn’t really help him and it was ultimately not approved by the FDA.
When my older son was diagnosed five years later, he too was convinced I would find a therapy. Again, I talked to everyone in the field, searched every related study, but could find nothing promising.
I am tenacious by nature, and after Hugh’s death, though tempted to give up, I renewed my search. There were more people, labs, companies looking at neurodegenerative diseases.
These two trials that CIRM is now funding represent breakthrough moments for me and for everyone touched by ALS. I feel that they are a promising beginning. I wish it had happened sooner. In a way, though, they have validated Douglas and Hugh’s faith in me.”
These therapies are not a cure for ALS. At least not yet. But what they will do is hopefully help buy people time, and give them a sense of hope. For a disease that leaves people desperately short of both time and hope, that would be a precious gift. And for people like Diane Winokur, who have fought so hard to find something to help their loved ones, it’s a vindication that those efforts have not been in vain.