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Sandy Morris does a lot. And this would be true even if she wasn’t living with ALS – but the fact that she was diagnosed with the disease in 2018 at the age of 51 makes her tireless work to advocate for people living with ALS all the more impressive. Her efforts have reverberated throughout the ALS world – from her work to support the ACT for ALS to her drive to make sure that pharmaceutical companies consider participants’ needs when designing clinical trials to her drive to convince the federal government to get more involved in ALS research.
First Symptoms to Her ALS Diagnosis
Sandy’s ALS story began in Truckee, a town in Northern California near Lake Tahoe where she lived with her husband and three children (they all still live together, up the road, in nearby Sierraville). The family spent much of their time enjoying all the Sierra mountains had to offer, skiing, hiking, and riding horses. One day, on a horse ride with her husband and daughter, Sandy realized that she couldn’t get her foot under her horse in order to signal it to gallop. Soon, she found she was tripping when she walked. Then she was falling.
“Just like everybody else that has ever been diagnosed with ALS, you ignore it for a long time,” she remembers. “Because when I looked up, 'why won't my flip-flop flop?' the only thing I could find was ‘foot drop.’ I was like, ‘that cannot be true. I have a pulled muscle. I've hurt myself. I haven't been sleeping well.’ But you never, ever think that it is a neuro disease.”
While at first, she had trouble accepting that something was seriously wrong, she continued to research her symptoms. By the time she finally decided to go to the University of California, San Francisco to pursue a diagnosis, she was convinced that she was experiencing the early stages of ALS. While her doctors at first expressed skepticism because of her young age, healthy lifestyle, and athleticism, she was firm in advocating for testing to be sure. In January of 2018, Sandy’s conviction was validated when she received her confirmed ALS diagnosis. When her doctors told her she had ALS, she replied “I know.”
Getting Involved in Clinical Trials
By the time Sandy received her diagnosis, she was already well aware through her research that there were few options available to treat her disease. It’s a reality that has fueled her as she threw herself into activism and advocacy for better access to investigational treatments over the past four years.
“I didn’t cry,” she remembers about receiving her diagnosis. “I’ve never cried about this disease. When I do cry, it’s because I realize this disease has not been a priority the way that it should be, because of its brutality, and because it’s 100% fatal. I just wish that there was more of a focus on trying to save us.”
Considering her options, Sandy and her family began searching for a clinical trial. They decided they wanted to explore stem cell treatments. Sandy chose to participate in BrainStorm’s phase 3 clinical trial for NurOwn in which participants would receive either stem cells harvested and grown from their own bone marrow, or a placebo, injected into their spine three times. After her participation in the trial was complete, Sandy would eventually fly all the way to South Korea for another stem cell treatment, CoreStem’s NeuroNata-R. However, after she had received four treatments in South Korea, in March 2020 much of the world shut down because of the emergence of the COVID-19 pandemic, and travel became impossible.
When her stem cell treatments were interrupted, Sandy says she began to experience rapid progression – losing much of her upper body strength and breathing ability. It was a tough time, but her family rallied around her, jerry-rigging their home to be more accessible, and working around the clock to provide her with care. In 2021, NurOwn was made available to a limited number of previous trial participants, including Sandy, through an expanded access program. Over the course of three more spinal injections, she felt that she saw her condition stabilizing for a time. While she says her progression has since begun to advance again, she recently was able to travel to San Francisco for one more injection.
Leaving the ALS Community in a Better Place
Inspired by the lack of options and her own experience as a clinical trial participant, Sandy also dedicated herself to fighting for expanded access to investigational therapies for people with ALS during this time.
“What I decided in that hospital room, when I was up against absolutely zero choices, was that I would leave this disease in a better place than I found it,” she says, “that the next 51-year-old mother of three would not be up against this hopelessness.”
One of Sandy’s first big advocacy efforts was to join the call to encourage the FDA to issue updated guidelines for designing trials for ALS treatments that would better address the needs of people with ALS who were enrolling as participants. She fought to make sure the patient voice was heard in the creation of these guidelines – advocating for, as she puts it, “trials that were designed for people, not zebrafish.”
In September of 2019, the FDA released the document “Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry.” The guidance encouraged clinical trial sponsors to adopt patient-centric practices such as making sure their participants could still access the standard of care treatments while participating in a trial, limiting exposure to placebos as much as possible, and taking advantage of FDA programs for expedited development and approval.
To Sandy and her collaborators, this felt like a major victory – she says she wanted to “run through the streets like Charlie with the golden ticket in Willy Wonka.” The next step was making sure that pharmaceutical companies would be held accountable to these standards – and that people in the ALS community would know which trials might best cater to their needs. To this end, Sandy has served as the leader the I AM ALS Clinical Trials Team for much of the past four years. Collaborating with other members of I AM ALS, including Nadia Sethi, who has since joined the ALS Therapy Development Institute (ALS TDI) as our Director of Community Outreach and Engagement, she created a universal rating system for ALS Clinical Trials. Called Patient Centric Trial Design (PaCTD), this system assigns trials a rating on a 5-star scale, based on how well they believe they adhered to the FDA’s new guidelines in the areas of humanity and efficiency . They also worked with over 30 clinical trial sponsors to make sure that they understood these new criteria – and that the ALS community would be paying attention to whether they adhered to them.
Passing The ACT for ALS
The next big issue Sandy decided to tackle was funding. Sandy and her family made several trips to Washington, DC, meeting with members of Congress to discuss the urgent need to fund expanded access programs (EAP) that could increase access to therapies for people with ALS.
At first, she says they were met with skepticism by many. Eventually, however, the momentum behind what would become the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) grew and grew thanks to Sandy and many other dedicated activists. Despite delays related to the Covid-19 pandemic that derailed the ACT’s initial momentum, it soon became a rare bipartisan issue in Washington, with Republican Senator Lisa Murkowski and Representative Jeff Fortenberry joining Democratic Senator Chris Coons and Representative Mike Quigley to sponsor the bill.
On December 16th, 2021 the Act for ALS passed the Senate with an unanimous vote, and was signed into law by President Biden on December 23rd. The new law calls for the establishment of a grant program to help drug sponsors conduct expanded access programs (EAP) for investigational ALS treatments. For Sandy, the bill’s passage was one of the proudest moments of her time in advocacy.
“Now we have the money there for [clinical trial sponsors] to study those of us who can’t get into the trials,” Sandy says. “When you really think about it, only 10% of us can get into the trials. But there are those of us who were diagnosed late, and those of us who would have given away everything we owned to be in a trial, but we didn’t make it. So, ACT for ALS says, here is door number two. Join an EAP, and we’ll take some of your data, to help advance the science. Because so many of us don’t just want the therapies to save our own lives, but we are here to help pay it forward”
Continuing to Fight
Today, Sandy is still fighting for better conditions of people with ALS as fiercely as when she began. She currently meets every Friday with the ALS Problem Solvers, a group of people with ALS, caregivers, and activists that discuss solutions and strategies for ALS’ biggest issues. Their focus is on encouraging the National Institutes of Health to adopt strategies for ALS research that have benefited other once poorly understood diseases such as HIV/AIDS. She also worked to help create the Morris ALS/MND principles, a set of guidelines that calls on policy, research, and healthcare leaders to make sure that people with ALS have a seat at the table when decisions are made about research, treatments, and funding for the disease.
Right now, one of her most visible projects, having received national media coverage, is working to reform California’s medical aid in dying (MAID) laws. Right now, the law states that any medications taken for this purpose must be self-administered – they cannot receive any help in taking the MAID drugs, which are usually taken orally. This is an issue for people with diseases that cause paralysis such as ALS, as they must decide between either choosing to commit to MAID before they are fully paralyzed – and possibly before they are truly ready – or sacrificing it as an option entirely. Along with a group of collaborators, Sandy has filed a lawsuit against the state of California alleging this violates the Americans with Disabilities Act.
Through her activism, Sandy has amassed what would be a long list of accomplishments for anyone, much less someone also navigating a terminal, debilitating disease. To Sandy, it’s all about using the time that she has left to make the biggest impact possible.
“I want my children to see a fighter,” she says. “I want them to know I gave this everything I had when I make that call to my beloved death doctor. I want them to know that, if I could do all this with ALS, anything is possible for them with their incredible minds and their beautiful bodies. That nothing is out of their reach.”
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