Yesterday Biogen denied my 5th request for Expanded Access of tofersen. We are not even sure how to feel right now. We are pleased our efforts have contributed to some people with ALS getting access to tofersen faster than they would have otherwise. Unfortunately, it still falls short for SOD1 ALS A5V patients like me who are deteriorating rapidly. For us, this is a death sentence because we likely will not make it to mid-July when the tofersen Expanded Access trial will start, and if we do, we will be in such bad shape that we may not see any benefit.

The frustrating thing is that this is avoidable because all we are asking for is equal, not superior, treatment relative to those in the trial. I told Biogen I would be fine being randomized into tofersen or the placebo arms at the same ratio (2:1) as study participants so I am not receiving any unfair or unethical advantage. This would have given me, and others, the same 67% chance of getting the medicine that we so desperately need. Then in mid-July, when the trial participants that are getting the placebo are switched over to tofersen, those of us who are receiving the placebo through Expanded Access could also be switched over.

It’s not as if Expanded Access is new. It started in the late 1980’s to help those dying of AIDS and continues to be offered today to help people with cancer. On the FDA website they cite more than 10,000 cases of Expanded Access so any ethical argument rings hollow. 

Potentially life-extending treatments need Expanded Access programs running alongside Phase 3 trials for patients who do not meet study criteria (but still can benefit) or miss the enrollment window and are experiencing rapid progression. The HEALEY ALS Platform Study at Mass General is a good example of this approach.

We are thankful that this fight may help some people with ALS. Since we began our efforts just seven weeks ago, we have met so many wonderful, generous, caring people. We have been helped in our efforts by our elected officials, journalists, doctors, friends, family, and even complete strangers. 

We will also be forever grateful to the support offered by those with ALS, their families and the ALS organizations. Your willingness to educate us, share resources, make connections and amplify our messages means the world to us. Thank you for reaching out and sharing your stories, they are an inspiration to us. We belong to a sad club, but it has the best members in the world.

Lisa Stockman Mauriello