SUMMIT, N.J. (CBSNewYork) — Thousands of people from all over the world are rallying behind a New Jersey mom who is fighting an aggressive form of ALS.
Over the last few months, the Union County woman has lost her ability to walk and talk.
As CBS2’s Lisa Rozner reported Thursday, doctors say one drug is her last hope, but she’s being denied the opportunity to take it and time is running out.
Years after Lisa Stockman-Mauriello participated in the ALS “Ice Bucket Challenge,” she started losing her breath easily, herself a victim of the disease.
Worse, in January she learned she’s one of 15% of ALS patients with a rare genetic mutation.
“Some people have three to five years before they lose the functionality and she’s losing it over weeks,” husband Bob Mauriello said.
After helping others for so long, she now has to lean on her husband and sons ages 14 to 22 for basic tasks.
“She couldn’t sign our taxes last week,” Mauriello said. “Now, just walking out here for the interview she lost her balance. Two months ago, she was doing mother-son dances, like you would normally do at a wedding. We recorded those for the boys’ weddings.”
The 51-year-old’s cognition is intact and after a career doing communications for pharma companies, now her life-saving pleas to one, Biogen, have been rejected five times.
The company is in Phase 3 clinical trials of the medicine Tofersen.
“This is Lisa’s best hope, we feel,” Dr. Neil Schneider said.
But she was diagnosed one week after enrollment closed.
Schneider, the director of the world renown Eleanor and Lou Gehrig ALS Center at Columbia University’s Irving Medical Center, is asking Biogen to give Lisa the treatment immediately.
The Food and Drug Administration‘s Expanded Access program provides the option to help patients in serious condition with no alternative.
More than 100,000 people have signed a petition of support and recently the company said after the trial, in July, patients like Lisa can get Tofersen.
READ MORE: Biogen’s Statement On Tofersen
An interfaith coalition is praying she makes it to her July 15 birthday.
“We’re getting prayer quilts and rosary beds and … it has been amazing,” Mauriello said.
Biogen declined CBS2’s multiple requests for an on-camera interview. In a statement, the company said it is not fair to ask study participants to continue to receive placebo while access is offered to others.
“That’s an excuse,” said Peter Pitts, a former FDA commissioner. “There isn’t a single patient out there with ALS that I believe would deny the use.”
Pitts added her data would not be included in the FDA’s final review.
But another expert speculated that the company doesn’t want to risk negative publicity if it fails.
“And they may not be ready to set the actual cost or price,” said Bruce Y. Lee, professor at CUNY School of Public Health.
The family isn’t giving up hope, but is already fundraising to establish a health communications program at Lisa’s alma mater, the University of North Carolina.
“And just kind of make more Lisas, the world would be a better place with more Lisas,” Mauriello said.
The FDA says there have been thousands of instances where companies have offered the experimental drug to patients before a trial ends.
One bill in congress would help drug companies cover the cost of offering experimental drugs.
“Waiting a month or six months or two years for approval, they’re not going to be around or not living the life they’re living today, so if we can stop this disease in its tracks, we want to do it for one patient at a time,” said Dr. Danielle Carnival, CEO, I AM ALS, a patient-led nonprofit.
She said the bipartisan Promising Pathway Act was would require the FDA to create a prioritized high-speed lane for evaluating drugs intended to treat, prevent, or diagnose serious or life-threatening diseases.
“It provides a pathway where FDA would be able to say ‘We’re conditionally approving this drug.’ We’re saying there’s enough of a need in the patient community that this should be out there while we continue to do testing and that was introduced last week,” said Carnival.