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Best Life: ALS patient fighting for access to treatment and more time

Published: Jun. 1, 2021 at 7:21 AM CDT
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SUMMIT, N.J. (Ivanhoe Newswire) – Last summer, Lisa Stockman Mauriello was a busy PR executive, starting her day with 5 a.m. boot camps, workouts and keeping up with her three sons. Today, she is fighting for her life as an aggressive and rare form of ALS, also known as Lou Gehrig’s disease, ravages her system.

Despite her worsening condition, Lisa has been leading the fight for access to an investigational drug that could extend the lives of patients with her form of the fatal disease.

Lisa Stockman Mauriello rented a dance hall for a special moment with each of her three sons, knowing it was unlikely she would dance at their weddings. Lisa was the picture of health, until last August when her muscles suddenly froze, and her voice got very weak.

“They were weird and disparate,” Lisa Stockman Mauriello told Ivanhoe.

“Weird and disparate, yeah,” Bob Mauriello, Lisa’s Husband, confirmed.

Lisa was diagnosed with an aggressive form of ALS.

“So, a normal ALS patient will have about three to five years from when they start to experience symptoms until they pass. If you have the AV4/AV5 variant, it’s more like 12 months,” explained Bob.

There is no cure for ALS, but the Mauriellos hoped a therapy could buy time.

“Three to five months would make me very pleased,” expressed Bob.

Their doctor thought Lisa might respond to a drug called Tofersen being tested in a clinical trial that had just closed enrollment. The Mauriellos were hopeful they’d have access under the 2018 Right to Try Act signed into law by President Donald Trump.

“The right to try act really bypasses the FDA,” Lisa Parker, PhD, director of the Center for Bioethics and Health Law at the University of Pittsburgh, noted.

Experts argue about whether companies can still turn patients down, which Biogen did saying individual access to Tofersen could jeopardize the study. ALS advocates rallied. A change.org petition circulated and gained more than one hundred thousand signatures.

“As special as she is, people realize that this law was put in place for a reason,” commented Bob.

The Mauriello’s say they’ll continue to fight, hoping Lisa’s battle helps others.

“Lisa’s going to die sooner than most of us, but you spend the time with the people you love while you have it,” sobbed Bob.

And time is so important to Lisa and Bob.

The Mauriello’s continued to petition Biogen for access to Tofersen and were denied several more times. Last month, Biogen announced that it will launch an expanded access program for Tofersen in July. Lisa and Bob say that’s likely too late to have any impact on Lisa’s health. The company said they wanted to wait until patients in the placebo arm of the trial were also eligible for the therapy.

Contributor(s) to this news report include: Cyndy McGrath, Producer; Kirk Manson, Videographer; Roque Correa, Editor.

Copyright 2021 WMC. All rights reserved.