Wholesome Residing: Saving Lisa from ALS: A Dying Affected person’s Proper to Attempt
A busy PR manager last summer, Lisa Stockman Mauriello started her day with 5am bootcamps, workouts and kept 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 deteriorating condition, Lisa leads the battle to gain access to an investigational drug that could extend the lives of patients with her form of the deadly disease.
Lisa Stockman Mauriello rented a dance hall with each of her three sons for a special moment, knowing that it was unlikely that she would dance at their wedding. Lisa was the picture of health until last August her muscles suddenly froze and her voice became very weak.
Lisa was diagnosed with an aggressive form of ALS.
“A normal ALS patient has about three to five years from the onset of symptoms to death. If you have the AV4 / AV5 variant, it’s more like 12 months, ”explained Lisa’s husband Bob. There is no cure for ALS, but the Mauriellos hoped therapy could buy time. “I would be very happy for three to five months,” he said.
Her doctor thought Lisa might respond to a drug called Tofersen, which is being tested in a clinical trial that has just completed enrollment. The Mauriellos hoped they would have access under President Trump’s 2018 Right to Negotiate Act.
“The right to tryout law really goes by the FDA,” noted Lisa Parker, PhD, director of the Center for Bioethics and Health Law at the University of Pittsburgh.
Experts argue over whether companies can still refuse patients, which Biogen said individual access to Tofersen could jeopardize the study. ALS advocates rallied. A petition from change.org circulated and received more than 100,000 signatures.
“As special as she is, people know that this law was introduced for a reason,” commented Bob.
The Mauriellos say they will keep fighting in the hopes that Lisa’s fight will help others.
“Lisa will die sooner than most of us, but you spend time with the people you love as long as you have them,” Bob sobbed.
And time is so important to Lisa and Bob.
The Mauriellos continued to ask Biogen for access to Tofersen and were refused several times. Last month, Biogen announced that it would launch an expanded access program for Tofersen in July. Lisa and Bob say this is likely too late to have any impact on Lisa’s health. The company said it would wait for patients in the study’s placebo arm to qualify for therapy.