The ALS Association

February 2, 2015

Dear ALS Community,

We are excited and enthusiastic about the new approaches and new drugs that are being tested for people with ALS and remain very hopeful that one or more of these will become a therapy with substantial impact on slowing down or perhaps even stopping ALS. We remain grateful to the brave volunteers with ALS who are willing to take new medications to help in the battle to find an effective therapy. We understand the urgency to find treatments for a disease that has such a rapid course and are committed to identifying promising treatments more rapidly through initiatives to improve clinical trial design and encourage the testing of many compounds in phase II studies through programs such as ALS ACT.

As established advocates for the neuromuscular disease community, we are often asked to make comments on a treatment in the early stage of development. This is challenging. For all of the drugs currently being developed, exciting results in a small trial spark enthusiasm and urgency for widespread availability. We do hear the voices that say, “This is exciting, let’s make sure this drug is available to all patients with ALS!” We also hear, “OK, but what is the downside?  Why not make it available?”

Every medication has potential risks and costs. Recent excitement about lithium in ALS and followed by no effect (or worsening) in subsequent studies underscores this point. Why is this, you might ask? ALS is a disease that can be quite different from one individual to the next. These differences in how ALS progresses are particularly apparent when looking at small numbers of people for short periods of time. Therefore, well-controlled, relatively large studies are the best way to test whether a treatment works. Small studies play important roles in bringing a new treatment forward in ALS. They can give information on dosing and initial safety, information on potential biomarkers and can help in making a decision on whether to move forward to testing in more people and for a longer duration.

One such example is Genervon’s GM604, also known as GM6, which has drawn a great deal of attention. GM6 has received fast track and orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of ALS. GM6 has been tested in 12 people living with ALS (8 drug, 4 placebo, or a drug look-alike with no active ingredients) for two weeks and followed for an additional 10 weeks. The study was designed to test safety and tolerability in people with ALS. We hear the rally to hasten GM6's approval; however the clinical study is very small and short in duration, and like other studies of this size, it is difficult to determine whether there would be any benefit to people with ALS if we were to repeat this in a larger study or the long term impact on quality of life. The many experienced clinicians and scientists in the groups below encourage a larger and longer study to determine both safety and efficacy for those with the disease.

While this is a very hopeful time for ALS research, giving a medication broadly without a clinical trial exposes those with ALS to possible side effects  that may reduce their quality of life, risks making them worse, and pulls money, personnel, and effort away from finding the cure that all of us are working together to find. Be assured that we will be the first to get on board to assist with trials to test promising new therapies.


(In alphabetical order)
ALS Hope Foundation
Jeffrey Rothstein, M.D., Ph.D., NEALS Science Advisory Board Co-Chair
Jeremy Shefner, M.D., Ph.D., Past Co-Chair NEALS
Les Turner ALS Foundation
Muscular Dystrophy Association
Nicholas Maragakis, M.D., NEALS Science Advisory and Executice Committee Member
Robert H. Brown, Jr., D.Phil., M.D., NEALS Science Advisory and Executice Committee Member
Robert Packard Center for ALS Research
Terry Heiman, M.D., NEALS Co-Chair
The ALS Association
The North American ALS Research Group (ALSRG)
Timothy Miller, M.D., Ph.D., NEALS Science Advisory and Executive Committee Member
WALS Executive Team

Powered by Blackbaud
nonprofit software