ALS Patient Pleads With Biogen for the Chance to Extend Her Life Through Tofersen

Lisa Stockman Mauriello of Summit, New Jersey, is not alone in her struggle to gain access to the company’s experimental ALS drug, tofersen. In fact, according to a recent article in Fierce Biotech, over 70,000 people have signed a Facebook petition to Biogen to support her cause. Lisa was diagnosed with amyotrophic lateral sclerosis with the SOD1 mutation (ALS/SOD1) only a few weeks after the close (no longer recruiting) of Biogen’s clinical trial investigating tofersen.

ALS/SOD1 is a debilitating and progressive disease. It is usually fatal within five years after diagnosis. Lisa has this exceptionally rare form of ALS that stems from an SOD1 gene mutation.

Tofersen, an antisense oligonucleotide licensed currently from Ionis Pharmaceuticals, is being developed for this exact purpose.

Dr. Neil Shneider, ALS Center Director who is treating Lisa, believes tofersen offers the best chance to change the course of her ALS.

Response From Biogen

Lisa shared excerpts from Biogen’s March 17th letter to her from Al Sandrock, its R&D chief. He stated that early access to tofersen would be given only after participants in the trial were no longer receiving a placebo. The message was posted on Biogen’s website (please click here for the full article).

According to a statement by Dr. Maha Radhakrishnan, Biogen’s chief medical officer, regulatory approval is the most expeditious way to make tofersen available to ALS patients. That requires the clinical trials to be completed.

Dr. Radhakrishnan added that giving individual access to tofersen could jeopardize Biogen’s ability to complete the study that involves 178 ALS patients with the SOD1 mutation.

She explained the company’s position that it would be unfair to study participants taking placebos if the drug was offered to patients who were not part of the study. Biogen expects initial results of the Phase 3 study mid-year, at which time it plans to conduct an Early Access Program for SOD1 patients.

Lisa’s Response to Biogen

Lisa released a statement in which she wholeheartedly agreed that participants in these clinical trials are to be shown our deep gratitude for the risks they take.

But she disagreed with Biogen’s stance and points out that their decision affects more than just one patient. It is about patients whose form of ALS kills so quickly that they do not have the luxury of waiting until the process plays out. It is about physicians who are recommending tofersen as the only hope for extending their patient’s lives.

Biogen’s Other Phase 3

Biogen’s experience with a failed Phase 3 trial adds weight to both sides of the argument. Its trial for the ALS drug dexpramipexole failed in phase 3. This led Biogen to change their focus from drugs that target mechanisms such as anti-inflammatories to drugs like tofersen that focus on genetic mutations.

Lisa signed off on her message to Biogen by begging the company to allow her the chance to be here long enough to see her sons graduate and begin their own life’s journey.

Twitter: #tofersen4lisa

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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