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Mother with ALS asking Cambridge-based Biogen for access to experimental drug

CAMBRIDGE, Mass. — A mother from New Jersey who has been diagnosed with a rare form of ALS is taking her fight against the deadly disease to Cambridge.

With what could be only a few months more to live, 51-year-old Lisa Mauriello is asking the biotechnology company Biogen for access to an experimental drug that could potentially prolong her life.

“It’s a cliché, but she’s been my soulmate, my partner for almost 30 years, the mother of my children,” her husband Bob Mauriello told Boston 25 News Sunday night.

Bob Mauriello explained that, since Lisa’s diagnosis on January 6, her health has been declining rapidly. Still, he said the mother of three has a lot to live for.

“You think about not being there for your first grandchild or the boys’ weddings, and the tears start coming,” he said.

The couple is now pushing for access to the drug Tofersen after being denied by Biogen. The problem, they said, was that Lisa’s diagnosis came just a few weeks after the deadline for the drug’s clinical trial.

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“Then to know that clinical trials will likely finish up this summer, and then they may start to move forward with it for access this fall or winter…but by then, unfortunately, she’ll most likely be gone,” Bob Mauriello said. “We want them to try to find a way to say yes. It seems like they’ve been focusing on trying to meet with folks and try to find a way to politely say no, but we want them to try to find a way to say yes.”

The Mauriello’s started an online petition, calling for Biogen to change its position.

On Sunday night, nearly 100,000 people had signed it. On Tuesday, the couple is planning to hold a march from Kendall Square to Biogen Headquarters to raise public awareness.

“I know they have decisions to make and I know they want to help as many patients as they can. I just want them to try to help one more,” Bob Mauriello said.

Boston 25 News reached out to Biogen Sunday night and asked about Lisa’s desire to receive the experimental drug. They released the following statement:

We brought together people across the organization with expertise in bioethics, medicine, research and regulatory guidelines as well as our most senior leadership to challenge our own policies in light of the situation for Lisa and other people with ALS.

We share the urgent desire to bring new medicines to people suffering from ALS. Obtaining approval for a new drug from regulatory authorities around the world is the fastest way to help the largest number of people with a specific disease.

Our Phase 3 placebo-controlled study of tofersen, VALOR, is designed to investigate the safety and efficacy of tofersen in people living with SOD1-ALS. The study has completed enrollment and data are expected in the second half of 2021.

We are working as fast as we can and preparing to open an Early Access Program after VALOR patients are no longer randomized to placebo and if VALOR data show that tofersen is safe and effective.

Providing individual access to tofersen at this time could jeopardize access to tofersen for hundreds of SOD1-ALS patients by impeding our ability to complete the study and seek subsequent regulatory approvals.

We are acting as quickly as possible with the interests of all patients in mind within the current regulatory framework, with the hope of bringing new medicines to the ALS community.