I’ve lived past my expiration date and I’ve still got questions.
This month marks five years since my ALS diagnosis with a 2-5 year prognosis. I’ve crammed a lifetime of living, loving, learning, and advocating into those few years and I’ve still got questions.
I’ve met hundreds of patients and their families, caregivers, advocates, medical practitioners, researchers, drug developers, regulators, and legislators and I’ve still got questions.
Last week, I was reminded at a meeting of people with ALS and FDA regulators that we are all one ALS community. I would agree that we all have a common goal of finding an effective treatment or cure. However, I would argue that we look at the problem though distinct lenses. We champion our own point of view and often dismiss, distrust, or disparage community members who view it through a different lens. We disagree on how to reach the goal at best and cannibalize at worst. I’ve got some questions about that.
This problem is not new nor unique to the ALS community. But for now, my life and the lives of tens of thousands of other people currently living with ALS depend upon an immediate solution.
The National ALS Association led an effort to create an FDA guidance document to facilitate the clinical trial and drug approval process. It was presented as a collaborative effort, inclusive of all parts of the community. The voice of the conservative neurological science community dominated the conversation and document. Looking through their lens, it was a big win. Looking through my lens, it tragically meant we support the 12+ year, $2.5 billion, status quo drug approval process. That means the research community keeps their version of perfect science while patients like me are guaranteed to die waiting. I understand their position, they are desperately searching for an effective treatment and are unwilling to change because they want a scientifically proven treatment. They truly believe that is what’s best for patients. Drug developers are looking for an effective treatment as well and their lens includes dollar signs and a highly regulatory environment. The FDA would be thrilled to approve a treatment that has been proven effective by passing the gold standard of trial design. I want access to potential treatments now. My lens is urgency and access, period.
Can someone please draw a cartoon of this conundrum?
Here’s the deal. I, along with many others, have tried to gain traction on pressing for bold changes to this process. Today, I was asked by Sandy, a newly diagnosed woman, “Why isn’t something being done? What can I do to create change now?” My heart sank. If I knew what to tell her, I’d be doing it myself. She is one of the lucky ones — participating in a stem cell clinical trial. But, dear God, the study protocols are nothing short of barbaric. Sandy is voluntarily being denied access to currently approved medication for three months to see if she declines rapidly enough to be included in the study. If she progresses enough, she still has a 50% chance of a placebo being surgically injected into her spine. Here’s the real kicker: If Sandy gets the stem cells and shows improvement, she will not have access to future treatments for years. Barbaric.
They’ve got their conservative science that looks promising. Sandy is a desperate, dying woman. She feels it’s her responsibility to endure this trial to benefit patients in the future. Can someone please explain to Sandy’s family how this is okay? A promising treatment exists but your wife and mother is a lab rat and can’t have access to the treatment outside of a barbaric clinical trial.
Nothing is going to change until the patient community demands it. We have to find a solution that creates a win for those of us living with ALS. Let’s take an honest look through each other’s lenses and find a compelling solution. We need a Manhattan project, funding the brain power and advocacy to make it a reality.
Here are my questions:
Who has the leadership, resources, and influence to create a win-win for the entire community?
What barriers are preventing this from happening now?
I’m sitting in my hospital bed, typing with my eyes. Yet, I believe I can make a difference. What if those with resources and influence acted as though their lives depended upon finding a solution now? The answer is out there. Please help me find it.
May 21, 2018 at 11:06 pm
Shelly, “I’m sitting in my hospital bed, typing with my eyes.” Geeessshhhh! Your posts are amazing and this is an extraordinarily amazing one.Your explanation of the lenses with which people are designing and evaluating approaches is well reasoned, well written and well explained. Thank you for helping me understand the barbaric nature of the current study design and the misguided structure of the scientific approach. Reading your post I find myself nodding at your insightful questions “Who has the leadership, influence and resources to create a win-win for the entire community?” I don’t know the answer but I will keep asking and trying to make a difference.
I am glad you continue to advocate and educate on this issue. We all owe you a huge thank you and we all need to help.
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May 22, 2018 at 12:32 am
Love you! Thank you for your continued support and encouragement. It means the world to me.
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September 18, 2018 at 7:40 pm
Shelly, we owe you a great deal of gratitude & I love you❣️ You are my inspiration and the sopport group in general. Your pALS, Erika your pALS buddy 😝
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