A graduate of the University of North Carolina and Apex native diagnosed with ALS (or amyotrophic lateral sclerosis, also known as Lou Gehrig's disease) is not only fighting the degenerative disease, she is fighting for access to a potentially lifesaving treatment.
Lisa Stockman Mauriello is asking Biogen to allow her to try Tofersen, a drug currently in clinical trials, but the drug maker is denying early use until the trials are complete.
Mauriello may not have that long to wait.
Lisa Mauriello got her diagnosis in January.
“A week ago, she could type with both hands, and now she can only really type with her right hand,” her husband, Bob Mauriello, said.
He has become her voice as the disease has stolen her ability to talk and walk.
“I am doing her hair and makeup. I am feeding her by tube everyday,” he said. “Within months, if she is still with us, she is not going to be able to do anything for herself really. That’s what we are trying to prevent.”
Dr. Neil Schneider, of Columbia University, is Lisa Mauriello’s doctor. He believes she would benefit from Tofersen. But her diagnosis came just weeks after enrollment ended for the drug's phase three trial.
“I think there’s evidence to suggest this therapeutic would be helpful to her. In ALS, unfortunately, we do not have a lot of therapeutic options. We are very limited in what we can offer our patients. So this, I feel, is her best chance for a therapeutic that could make a meaningful difference in the course of her disease,” Schneider said.
The
2018 Right To Try Act allows Biogen to make a trial drug available to a patient under "compassionate use," but it does not require that.
In a statement, Biogen said:
“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.
“If we offered the certainty of access to Tofersen to patients outside of our study, we could not in good faith ask patients in our study to continue to receive placebo, without offering them the same opportunity for access to Tofersen.”
Lisa Mauriello's family and friends rallied outside Biogen’s headquarters, and 100,000 people have signed an online petition asking Biogen to change its mind.
Bob Mauriello said, “If there is something proven to be effective that is so close and people can’t wait, that’s why that act was put in place.”
The Mauriellos are hoping that Lisa can live to see her three sons graduate this spring - from college, high school and the 8th grade.
“We’re hoping for an Easter miracle here," Bob Mauriello said. "That someone at Biogen says, 'Yes, we can do this without endangering the approval of this important drug.'”
