Thousands of People Have Joined Lisa in Her Appeal to Biogen Asking for Access to ALS Drug

A note from Lisa:

Please read and share this piece with anyone living with ALS or any life-threatening condition that can benefit from compassionate use: 

Lisa’s friend of thirty years Daniel McIntyre, a Boston public affairs consultant, wrote an article in an April 2021 issue of the Timmerman Report on behalf of Lisa and her plea to Biogen to allow her access to the experimental ALS drug Tofersen.

McIntyre had worked for Biogen and begins his article by stressing that he has the highest regard for the company. He adds a note of caution that Lisa’s petition, which now numbers approximately 103,000 signatures, could impact Biogen for many years to come.

As Lisa said in her note introducing the McIntyre article, not only could Biogen’s allowing her access to Tofersen stabilize her rapidly deteriorating condition, but it could impact thousands of other patients who are waiting to gain access to drugs under the FDA’s Compassionate Use designation.

An Update from Lisa’s Husband Bob

They were still going on family trips as recently as two months ago when they first noticed Lisa was having a little trouble breathing. Two months later Lisa is wheelchair-bound.

With Lisa’s disease, bulbar amyotrophic lateral sclerosis (ALS), patients experience problems swallowing or with speech rather than typical symptoms in their legs or arms.

Lisa’s form of ALS progresses so rapidly that now Bob has to feed her soft food through a tube. Lisa is now in a wheelchair. Yet according to Lisa’s doctor, who told them about Tofersen, there is still time. Toferson treats Lisa’s rare SODI 1 mutation. There are no other options.

Bob quotes Biogen as saying that Lisa and Bob should be patient and wait for the trial to conclude next year. But Bob says Lisa does not have even a year as her ALS is taking hold so rapidly.

The Basics

The drug at the center of the controversy is Tofersen, which is designed as a barrier against the SODI 1 protein associated with a rare type of ALS.

Lisa has worked in the biopharma industry for years prior to her diagnosis.

Biogen had just closed enrollment for its VALOR Phase 3 trial investigating Tofersen several days before Lisa received her life-altering diagnosis. Since then, Biogen’s door has been shut.

Lisa’s physician’s request for Tofersen under the FDA’s policy for expanded access as well as Lisa’s own request was denied by Biogen.

By the time Biogen issued a statement outlining its position and reasons for denial, a campaign that included over 100,000 signatures plus print, and media coverage had already begun.

At this point, the issue is no longer just about Lisa and saving her life. It is about other patients with diseases such as cancer, HIV, and certain rare diseases fighting for survival.

The Company’s Viewpoint

An appeal for compassionate use may be made by a physician for one patient or expanded access may be applied for and granted to hundreds of patients before the FDA’s final approval of a drug.

Companies have concerns that a patient who is taking a drug outside of the clinical trial setting may have an adverse event and suspect the drug. In that situation, the companies claim that a trial may be closed down and a good drug may never reach the market.

Although the FDA has shown support for the concept of compassionate use, the decision to provide such therapy is made by the drug company.

Companies developing drugs are in a catch 22 situation by either denying an application and run contrary to public opinion, or if the company approves an application it could have a negative effect on the company. This would also harm patients who may have otherwise benefited from the drug had the trial not been canceled.

Looking Back

McIntyre acknowledges that Biogen has shown its willingness to develop drugs that will benefit patients with difficult-to-treat diseases but no treatment currently available.

If a company like Biogen can end up in this tug-of-war, other companies are also vulnerable.  Although there may never be a clear-cut answer, it would be helpful to establish policies for compassionate use that are just, ethical, humane, and cannot be abused.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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