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!
Dina Towbin
We are with you Jess in this battle.
Kathi
Thanks for keeping us informed and sharing the link to make the donation easier.
You are an inspiring warrior for the cause.
Rachel Nussbaum
Thanks, Jessie, for sharing your take on where the research stands, and on the complexity of ALS. I continue to learn so much from you… not least of which is due to the emotional openness and vulnerability you model as you write and teach. Sending virtual hugs your way tonight.
Kirstan
You amaze me daily, Jessie. Your writing is so full of knowledge and eloquence, and is helpful for us to understand what’s really going on out there. I miss you!
Julia Chase
Dear Jessie, Thank you. This was a very informative post. I appreciate the time, effort and research you put into it. I Iearned a lot. Your writing and teaching are so engaging. I am just sorry you are having to go through this syndrome! I hope they can speed up the science too. I will make a donation to ALS research! Warmly, Julia
Jenny Love
Dear Jess,
Thank you for your thoughtful post and the helpful links. And thank you for spreading the word about how to get involved and support–and hopefully, help quicken the pace of–ALS research. I am so glad that you have had an opportunity to be part of a clinical trial for a new medication. The goal of making ALS livable by 2030 is encouraging and exciting!
Love,
Jenny
Kimiyo Schau
Hi, Jessie, thank you for the great information. It is frustrating that ALS research is slow progressing when we, human beings, are so smart enough to create effective COVID vaccines within a short period of time, invent a fully automatic driving system, and shoot ourselves into space…… I am sorry I cannot do anything to speed up the process rather than pitching in some money. It is so unfair that you have to go through this journey, but I can see your spirit is so free and boundless. You are a remarkable writer!!!
Leonard Podis
Thanks for this informative post, Jessie. JoAnne and I think of you often and send you our warmest regards. The slow pace of progress toward more effective treatments and therapies is certainly frustrating and disappointing, but it does seem that there are very substantial, ongoing efforts underway. Today (June 2), by the way, has been designated as Lou Gehrig Day across Major League Baseball. 🙏 Len & JoAnne
Sara Esrick
Jessie! You are such an inspiration to me! I am so grateful for the links to learn more about ALS. Here with you through this journey. XOXO
David Lasky
Thank you for taking the time to educate us about ALS. I have know of it for most of my life, but now realize that I knew very little about it.
With gratitude,
David