Lisa Stockman-Mauriello is dying. She has one last ambition: to live long enough to see her three sons graduate this year — from middle school, high school and college.
She and her doctor believe she needs a new experimental drug to hold on. She’s unlikely to get it.
At a time when the global population is racing to take experimental coronavirus vaccines, it’s business as usual for drug development unrelated to the pandemic. For those whose lives are on the line while they wait for drugs in trials, the incongruity is crushing.
“I just want a fighting chance,” said Stockman-Mauriello, 51, of Summit, N.J. She spoke softly, her voice weakened by amyotrophic lateral sclerosis, the incurable illness also known as Lou Gehrig’s disease. It first attacked the nerves in her face and neck, affecting her ability to speak. From there, it’s been spreading throughout her nervous system.
Stockman-Mauriello’s hope is to somehow get Biogen Inc.’s tofersen, which she believes is the best treatment — or possible treatment — for her condition.
But because the drug is still in trials, the Cambridge, Mass., company, one of the biggest in biotechnology, says its hands are tied. Her struggle to get tofersen, which showed promise in an earlier trial, lies at the heart of a moral and ethical debate that for years has entangled private industry, patients and the federal government.
Patients like Stockman-Mauriello understand that an unproven drug may not work for them and could even hasten their deaths. They’re willing to take that chance. But companies say making exceptions for some could hurt all patients by compromising clinical trials. These battles are likely to intensify as biotech and pharmaceutical companies push deeper into lifesaving drugs. More than a dozen drugs for ALS alone are in various stages of development.
In Stockman-Mauriello’s case, her diagnosis came just after the company stopped accepting applicants to its double-blind trial. Even if she had gotten in, Stockman-Mauriello, who has had a 30-year career as a health-care communications executive, would have had only a 50% chance of receiving the treatment, because half the group received a placebo. That’s been standard practice for decades.
It was for such patients, those who have exhausted all other treatment options, that then-President Donald Trump three years ago signed a law designed to open clinic doors. The law, known as Right to Try, was intended to make it easier for companies to provide experimental drugs by sidestepping regulators.
But Right to Try doesn’t require them to provide experimental drugs to patients who want them. And Biogen, along with other drug companies, have resisted, reasoning that it is both unfair to those in the studies and that it could compromise the trials, which are essential for Food and Drug Administration approval.
PhRMA, the biopharma industry’s lobbying group, has said its members take access to experimental medicines seriously and have asked for FDA guidance on giving treatments outside a clinical trial. PhRMA declined to comment specifically on Stockman-Mauriello’s case and said individuals should consult their doctors to determine the best course of action.
Patient advocate groups, meanwhile, are urging companies to devise their own solutions. “Every company needs to have an expanded access plan, and should be clear about what that plan is,” said Neil Thakur, chief mission officer at the ALS Association.
Neil Shneider, Stockman-Mauriello’s doctor and director of the Eleanor and Lou Gehrig ALS Center at Columbia University in New York, nonetheless asked Biogen for access. Stockman-Mauriello, he said, has the exact genetic form of the disease targeted by the drug, which occurs in only 2% of people with ALS. An earlier study found the drug reduced the defective protein in the spinal fluid, and seemed to slow the progression of the disease and improve muscle strength.
“It’s her best shot for making significant change in the course of her disease,” said Shneider, who is also one of the outside investigators for Biogen’s study.
For Biogen, though, the issue is much larger than Stockman-Mauriello’s case. There are an estimated 15,000 people in the U.S. with ALS, according to the Centers for Disease Control and Prevention. About 5% to 10% of cases are inherited through a mutation, like the one Stockman-Mauriello has, the ALS Association says. There are no drugs approved for that mutation, and getting one on the market as fast as possible is the company’s objective.
“We share the urgent desire to bring new medicines to people suffering from ALS,” the company wrote in a statement. “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.”
Biogen denied Stockman-Mauriello’s request, writing in an email viewed by Bloomberg News that providing the drug to patients outside the ongoing clinical trial would be unfair to people in the trial who received a placebo.
“We cannot overlook these patients when considering questions of broader access, and cannot keep them on placebo while at the same time offering tofersen to those outside of our study,” Alfred Sandrock, Biogen’s executive vice president of research and development wrote in a separate statement.
The company has indicated it plans to create an early-access program before FDA approval if the trial shows favorable results.
That will probably be too late for Stockman-Mauriello, her doctor said. Her body is rapidly deteriorating. She can no longer shout loudly enough to call her dogs inside. She struggles to raise her left arm. Her husband, Bob, helps her talk on the phone and dries her hair.
With her request denied, Stockman-Mauriello found herself in the same situation as thousands of others desperate for experimental medicines who turn to social media in an effort to pressure the company. More than 100,000 people have signed an online petition on Stockman-Mauriello’s behalf. Supporters held a protest outside Biogen’s Cambridge headquarters and one friend even contacted Chief Executive Officer Michel Vounatsos directly. Biogen has remained steadfast.
Even if she doesn’t end up getting tofersen, Stockman-Mauriello may make a difference in the lives of ALS patients coming after her, according to Jinsy Andrews, a neurologist at Columbia’s Irving Medical Center and co-chair of the Northeastern ALS consortium, an international clinical trials group. Her advocacy is making waves and pressuring companies like Biogen to make experimental drugs more available, Andrews said.
For Stockman-Mauriello, it’s now about living long enough to see her sons reach milestones. When she’s not taking on Biogen, Stockman-Mauriello devotes her energy to making albums filled with photos and notes to relatives so they can always keep her close, come what may.