As it stands now, Lisa Stockman Mauriello doesn’t have much time left, but a new drug could prolong her life long enough for her to see each of her three children graduate this spring.
But the drug company has denied her access stating they are “working as fast as we can and preparing to open an Early Access Program” for patients like herself.
Lisa, 51, of Summit, New Jersey, was diagnosed with a rare form of ALS in January 2021, and her condition has only continued to get worse.
“A week ago, she could type with both hands, and now she can only really type with her right hand,” her husband Bob told WRAL in the beginning of April.
“I am doing her hair and makeup. I am feeding her by tube everyday,” he said. “Within months, if she is still with us, she is not going to be able to do anything for herself really. That’s what we are trying to prevent.”
Her doctor, Dr. Neil Schneider believes a drug, Tofersen, that’s currently in clinical trials would greatly benefit her because it’s designed to treat patients just like her.
But Lisa’s diagnosis came several weeks after enrollment for Tofersen’s phase three trial closed, and the drug’s company, Biogen, is refusing to give her access despite a federal law, The Right To Try Act, which gives patients who have been diagnosed with life-threatening conditions and have tried all available treatments and who were not able to participate in a clinical trial access to unapproved treatments.
The law states that Biogen can make Tofersen available to Lisa under “compassionate use,” but it is not required. And in this case, Biogen is refusing access stating:
“Providing individual access to Tofersen at this time could jeopardize access to Tofersen for hundreds of SOD1-ALS patients by impeding our ability to complete the study and seek subsequent regulatory approvals.”
“If we offered the certainty of access to Tofersen to patients outside of our study, we could not in good faith ask patients in our study to continue to receive placebo, without offering them the same opportunity for access to Tofersen,” the company said in a statement.
Lisa just wants more time
Lisa and her family understand that Tofersen will not cure her of ALS, but they hope it will give her more time to accomplish a few goals.
“My three sons and my husband, Bob, are my life. My goals are modest and, I believe, realistic,” she wrote on a Change.org petition.
I want to survive long enough to be here for three milestones: to see my oldest son, Scott, graduate from college in May, my youngest, Dean, graduate from the 8th grade, and my middle son, Luke leave for college in September. We believe Tofersen gives us that hope.
“It’s a bit of a cowardly move on part of the company,” former FDA Associate Commissioner Peter Pitts told ABC7.
“We have to cut right the chase,” said Pitts. “These patients need help and they should get help and companies should strive to provide their products, even under extraordinary circumstances and not make excuses.”
For now, Lisa and her family are hoping that someone at Biogen will hear their pleas and give her access to a potentially life-changing treatment.
So far more than 100,000 people have signed an online petition asking Biogen to give Lisa access to Tofersen.
It’s despicable that drug companies are able to deny patients, especially those who are nearing the end of their life, access to potentially life-changing treatments.
Surely there has to be some way to give Lisa and others like her the treatment while not compromising their clinical trial.
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