Lisa Received Her Long-Awaited ALS Treatment, but Is It Too Late?

Lisa Stockmann Mauriello began her petition to Biogen almost one year ago. In her letter to the developers, Biogen and Ionis Pharmaceuticals, Lisa explained that she has an inherited form of  ALS called SOD1 (superoxide dismutase 1).

Tofersen, an antisense oligonucleotide, targets the mutation in the gene that causes the disease. Lisa was asking for expanded access to the drug. As Biogen’s recent announcement explains, the company felt it could not release the drug prior to completion of its VALOR Phase III trial.

Lisa’s pleas quickly caught the attention of supporters across the nation, as she was joined in her petition by almost 100,000 people on her behalf. But for months Biogen held firm, stating publicly that they cannot offer the drug outside of the trial in fairness to patients who were participating in the trial.

After months of “back and forth,” Biogen opened applications on June 25, 2021. Now it is a matter of waiting to see if there is improvement in Lisa’s condition. Patients succumb from Lisa’s particular variant, AD5, usually within a year after being diagnosed. Lisa’s symptoms became evident about one year ago. As her condition worsened during the year, Lisa pleaded with Biogen for access to Tofersen.

The Early Access Program (EAP)

As part of the EAP, tofersen is being allocated to SOD1-ALS patients who are classified as rapidly progressing according to the ALS Functional Rating Scale. After positive data on the efficacy and safety of the drug, Biogen will expand its distribution to a larger population in the Fall of 2021.

Last week, as planned, patients who were given a placebo during the VALOR Phase III trial switched from the placebo to the drug.

Lisa’s birthday was July 15th, so the family considered this a belated birthday present. Her husband said they hope that this year-long effort will lead to improved development and approval of experimental drugs.

On the other hand, Biogen did not waiver on its ethical argument that it must consider the patients who received the placebo. The company reasserted its belief that it is not fair for patients to receive a placebo while others have access to tofersen. Biogen has agreed that access would be allowed when the study has ended.

Therefore, until efficacy and safety are established, Biogen will allow early access to the most rapidly progressing ALS patients. Biogen agreed that people in the ALS community are looking for a more accountable system for everyone.

Lisa’s family thanked the ALS community and Lisa’s physician. The family expressed their appreciation to Biogen for moving up the timeline but then added that they had wished it had occurred sooner.

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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