The ALS News Today articles that showed up in my inbox a couple of weeks ago – SOL-257 Gene Therapy Targeting TDP-43 Shows Promise in Mice and Arimoclomol Fails to Show Efficacy in Phase 3 Trial, Topline Data Show – pretty much sum up the state of ALS research. That is, there are studies underway that sound hopeful, but which are a long way from being on the market, and there are many studies that fail. Sometimes I’m amazed and heartened to read about the various clinical trials taking place around the world. Lately, however, I’m frustrated by the slow pace of science.
There are only two ALS drugs on the market today. Riluzole received FDA approval in 1995. It can prolong lung function and increase survival by two to three months. Yes, two to three months. The other drug, Radicava, was approved in 2017, and slows progression, but not necessarily for more than a year or for people with advanced ALS. In fact, insurance won’t approve Radicava for pALS whose symptoms are beyond a certain threshold.
There is a new drug seeking FDA approval. I was fortunate to be a part of the clinical trial. I don’t know if I received the study drug or the placebo, but once my participation ended, I had the opportunity to take the actual drug, which I’m still doing. People ask me if it helps. I have no way of knowing if my progression would be worse without it. The results of the clinical trial were statistically significant, but very modest. We are still nowhere near having a drug that stops the death of motor neurons.
The fact that ALS has many causes further complicates things. Even if scientists develop a successful treatment, it will likely help only some ALS patients. We will probably need multiple treatments in order to stop ALS for all who have it. (See Why ALS is more like a Syndrome than a Disease, and what that Means for Treatments.)
The ALS Association just announced a goal to make ALS livable by 2030. To achieve this goal will take enormous resources, but it is possible. If you would like to help, please donate to the ALS Therapy Development Institute or to the ALS Association.
May is ALS awareness month. Please spread the word!