Lisa Stockman-Mauriello with her husband Bob and their three sons.Photo: Courtesy Lisa Stockman-Mauriello
A New Jersey mother of three is in desperate need of a new drug that could be a game changer in her battle with amyotrophic lateral sclerosis, the deadly neurological disease that takes away one’s ability to control muscles.
In an emotional interview with PEOPLE, Lisa Stockman-Mauriello and her husband Bob Mauriello detail their race against time. Unfortunately for the 51-year-old — who was diagnosed with Bulbar ALS earlier this year — she says her chances of survival hinge on whether she can obtain Tofersen.
For Lisa, waiting until the pharmaceutical company’s study is complete may not be possible, asBulbar ALS— which leads to loss of movement in the face and neck — is known for a quick progression. Those who suffer from Bulbar ALS first experience speech or swallowing problems, as opposed to symptoms in the arms or legs, according to theNational Institute of Neurological Disorders and Stroke.
“They keep saying, ‘Oh, well, just wait until late this year, early next year, and we’ll be able to open it, expand access,'” Bob, also 51, says of the drugmaker. “And that would be fine if she had three to five years to live. But we’re talking months, and it’s going to be too late.”
“It’s heartbreaking,” adds Lisa.
Biogen addressed the difficult decision in a letter to Lisa in March, NBC affiliateWCNCreported.
Lisa Stockman-Mauriello and husband Bob Mauriello.Courtesy Lisa Stockman-Mauriello
It was less than a year ago that Lisa’s life was forever changed.
The mom of three, who worked in public relations and advertising forSyneos Health, says she noticed something was wrong in August, when she had trouble calling in her dogs. Around that time, Lisa was also experiencing difficulty raising her arm. After seeing a variety of doctors, Lisa says she was officially diagnosed with Bulbar ALS in January.
“She knew enough about it to know that there’s no treatment for it and the life expectancy is fairly short,” says Bob, who often speaks for his wife when she is unable to speak for herself. “So she cried the whole way home… and when she got home, she couldn’t even bring herself to get out of the car because she didn’t know how she was going to tell us.”
Lisa Stockman-Mauriello with her 3 sons when they were younger.Courtesy Lisa Stockman-Mauriello
Dr. Neil Shneider, Lisa’s physician and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University, told NBC affiliateWCNCthe drug is Lisa’s “best chance.”
“I think there is evidence to suggest that this therapeutic would be helpful to her, and you know, in ALS, we don’t have a lot of therapeutic options — we are very limited as to what we can offer our patients,” Shneider said. “So I feel this is her best chance, for a therapeutic that could make a meaningful difference in her disease.”
With each day that passes, Bob feels an increased sense of urgency.
“Two months ago, when we were still doing some family trips, her voice was a little bit hoarse. She was losing her breath more easily than normal for someone who’s always been really active. But we had no idea what was going on,” he says. “Now, I’m feeding her every meal through tubes. She can’t eat any solid food. I’m pushing her around on the wheelchair on the beach. I have to help her get ready for bed.”
Lisa Stockman-Mauriello with her family.Courtesy Lisa Stockman-Mauriello
When Lisa and Bob approached Biogen about the drug, they claim they were denied access, despite Lisameeting the FDA’s criteriato obtain it through a pathway called compassionate use.
According tothe FDA, compassionate use applies to patients who have a life-threatening condition with no other treatment options available, and want to gain access to an investigational medical product.
“We’re tremendously appreciative of the research that they’ve done and we know that Biogen’s goal is to get something approved and out to the market as quickly as possible so they can help people,” Bob says. “They absolutely could allow this, and they’re choosing not to.”
“Biogen has this tagline — [Where Science Meets Humanity] — and they’ve done an awesome job with the science part,” he adds. “But the humanity part, unfortunately, at least to us, feels like it’s lacking in this specific case.”
Lisa Stockman-Mauriello at UNC Chapel Hill with her husband.Courtesy Lisa Stockman-Mauriello
To help their cause, Lisa and Bob set upan online petition, where they are asking for Biogen to grant her access to Tofersen. So far, the petition has garnered over 100,000 signatures.
“It’s almost hard to really imagine this outpouring,” Bob says. “We’re proud of the lives that we’ve had… But didn’t realize quite how much of an impact we’d had on people and how much support we have.”
Bob has also spearheaded theLegacy for Lisa fundat the Hussman School of Journalism and Media at their alma mater, the University of North Carolina, Chapel Hill.
The goal is to cover endowed professorships, summer internships, guest speakers and scholarships to help the next generation of healthcare communicators.
“We want to celebrate the life that she’s led and the legacy that she leaves behind,” Bob says. “She’s tremendous. I joke that I’m the President of the Lisa Stockman-Mauriello fan club now for 25 years, and I’m proud of that role because she’s an amazing person.”
While the family waits to see what will happen, Lisa says she is finding strength in Bob and their sons, Scott, 22, Luke, 18, and Dean, 14. Her dream is to see them graduate from college, high school and eighth grade in just a few months.
RELATED VIDEO: Father of Two With ALS Gives Update on His 600-Mile Hike & Shares Inspiring Message
“The boys are supportive, but they have big lives ahead of them, and you don’t want them to focus too much on this,” Bob adds. “For us, we want to spend our time witnessing their achievements and taking pride in our children because that’s part of Lisa’s legacy.”
The couple tells PEOPLE that they encourage readers who want to help them tosign the petitionandcontact Biogen.
In the drugmaker’s statement to PEOPLE, Biogen addresses the complexities of the situation and reiterates the points made in their initial letter to Lisa:
We brought together people across the organization with expertise in bioethics, medicine, research and regulatory guidelines as well as our most senior leadership to challenge our own policies in light of the situation for Lisa and other people with ALS.
We share the urgent desire to bring new medicines to people suffering from ALS. Obtaining approval for a new drug from regulatory authorities around the world is the fastest way to help the largest number of people with a specific disease.
Our Phase 3 placebo-controlled study of tofersen, VALOR, is designed to investigate the safety and efficacy of tofersen in people living with SOD1-ALS. The study has completed enrollment and data are expected in the second half of 2021.
We are working as fast as we can and preparing to open an Early Access Program after VALOR patients are no longer randomized to placebo and if VALOR data show that tofersen is safe and effective.
We are acting as quickly as possible with the interests of all patients in mind within the current regulatory framework, with the hope of bringing new medicines to the ALS community.
source: people.com
