A New Jersey woman fighting for her life with a rare, rapidly progressing form of ALS is taking on a new fight — to receive an experimental treatment when all other options seem to be off the table.
Lisa Mauriello, 51, of Summit, New Jersey, was diagnosed with ALS in January, but it’s progressing quickly. Already, she’s lost her ability to speak due to the rare form of the disease she’s been diagnosed with, with a prognosis much less encouraging than the average ALS case, which is devastating to families on its own.
Her family and doctors are pleading with Biogen Pharmaceuticals to give her access to Tofersen, a drug currently in clinical trials.
“I think Tofersen offers the best chance to extend her life in a meaningful way,” said Dr. Neil Shneider of the Gehrig ALS Center at Columbia University.
But the drug company wants to wait until phase three trials are complete, the family said, with access to be expanded this fall or winter.
Unfortunately, the way her disease is progressing, Mauriello likely won’t be able to wait.
The company, though, said providing her access to the drug could put their trial in jeopardy.
The family is fighting back, saying she meets the criteria for compassionate use, and they’re looking for others to join in.
More than 100,000 people have signed a petition to convince the pharmaceutical company to allow Mauriello to receive the possibly-life-saving drug.