“Of all tyrannies, a tyranny sincerely exercised for the good of its victims may be the most oppressive. It would be better to live under robber barons than under omnipotent moral busybodies. The robber baron’s cruelty may sometimes sleep, his cupidity may at some point be satiated; but those who torment us for our own good will torment us without end for they do so with the approval of their own conscience.”C.S. Lewis
By Matt Bellina |
Matt Bellina is a father, Navy Veteran, and patient advocate who is receiving an experimental treatment under the new Federal Right to Try law.
|Caitlin Bellina and Jim Worthington stand with Matt Bellina for the National Anthem before The 2017 Rock ‘n’ Roll Philadelphia Half Marathon | Photo by Helga Proudfoot|
Over ten years ago, I was training to become a Naval Aviator when my body started slowly failing. By the time I earned my wings of gold, one physician estimated about a third of my motor neurons had already died.
ALS is a spectrum of diseases that result in the degeneration of upper and lower motor neurons, causing dementia in some cases and eventual death in all patients. The literature varies, but roughly ten percent of cases are genetically inherited, and even within that subgroup, there are at least forty separate genetic mutations.
When I was formally diagnosed in the spring of 2014, I had already been symptomatic too long to qualify for a single FDA-registered clinical trial. There were a few new treatments that I thought looked especially promising, but I knew they were likely too early in the development pipeline to be available in my lifetime.
So I did what any rational person would do: I started the process of begging companies to let me try their experimental treatments outside of the trial.
One treatment that excited me was Copper ATSM. Another was the widely-known NurOwn from Brainstorm Therapeutics. These therapies were particularly interesting to me because both sought to address broad issues in the pathology of the disease.
I was also very interested in a precision medicine program that was being conducted by a nonprofit with proceeds from the historic Ice Bucket Challenge. I strongly encourage you to read about the ALS Therapy Development Institute’s Precision Medicine Program at ALS.net. It is a groundbreaking initiative that allows researchers to interface with patients while studying their actual cells and DNA profile all while employing cutting edge digital techniques for measuring disease progression. This is the absolute tip of the spear in individualized medicine.
|Yiding Yan tests a sample at the ALS Therapy Development Lab in Cambridge, MA | Photo by ALSTDI|
Unfortunately, these therapies could not be made available under the FDA’s so-called “expanded access” pathway.
Thus began my involvement in patient advocacy. I went looking for answers and I found them from a woman in Wisconsin and a small boy in Indiana.
In Wisconsin, an ALS patient named Trickett Wendler had already keyed in on the same issues and was meeting with Senator Ron Johnson to create a solution for patients with life-threatening illnesses. It would be called the Right to Try Act and would give patients and physicians the ability to work with drug companies without having to petition the FDA. Trickett passed away shortly after beginning her lobbying efforts.
Her death deeply impacted my family, so much that we traveled to Washington DC to see if we could help further the cause. It was there that I met a young boy named Jordan McLinn who was battling Duchenne Muscular Dystrophy. Jordan had already succeeded in convincing then-Governor Mike Pence to sign the state version of Right to Try in Indiana in 2015.
At about the same time, my friend and leader in the fitness industry, Jim Worthington, encouraged me to make sure that Right to Try would become a federal law. With the leadership already being provided by the Goldwater Institute and patients across the nation, we knew the stars were aligned.
On May 30th, 2018 we were present when President Trump signed Right to Try into federal law, officially amending the Food, Drugs and Cosmetics Act. There are so many people that should be given credit for this tremendous achievement, but there is not enough space here to name them all. Thankfully everyone involved in this movement is more interested in results than getting credit and they all know who they are.
|Patients, caretakers, and advocates gather around Jordan McLinn after the federal Right To Try signing ceremony | Photo by The White House|
I will say I was glad to have my strong wife Caitlin by my side because none of it would have happened without her. After the passage of Right to Try, I knew the work was just beginning since most companies are cautious about changes in the drug development process, but Caitlin gave me confidence that we could achieve anything.
I will always be grateful that Brainstorm had the courage to step up and set the example for how the law can be successful.
On the morning of December 27th, 2018, American Airlines flight 1776 flew from Boston to Philadelphia with a small, temperature-controlled box containing my personal mesenchymal stem cells in the form of the treatment known as Nurown. Jordan McLinn and his mother Laura flew in from Indiana for the occasion. I was thrilled to see he is still doing well on the experimental treatment he receives for his disease. My infusion was benign and I was sent home twenty four hours later.
Within two weeks, I felt the overwhelming urge to stand up out of my wheelchair, so my family propped me up against the kitchen counter and I stood!
Since then I have had six injections and I have regained the ability to stand on my own without assistance. My lung capacity is 37% higher than it was before my first injection, so I no longer need the assistance of a breathing machine. I have gained enough mobility in my arms to scratch my face and even take my glasses off. All of these are improvements from where I was before the treatment.
I am extremely grateful and excited about my treatment, but I am heartbroken that I am the only person with ALS to receive access outside of the trial at this point.
Our work is just beginning. I feel like my gains are a small but significant victory in the war against heterogeneous diseases. People are receiving treatments for other life-threatening illnesses through the Right to Try law, but many companies have not been educated on how the law works and most are still timid about using it.
I see our role now as fighting misinformation being spread by people who stood to profit off the old paradigm. We need to help researchers and manufacturers to see opportunity in the bodily autonomy of well-informed people battling terminal illnesses.
For me, Right to Try is as much about moving the needle towards civil liberties as streamlining medical research. Today I am a living example of how both are beneficial, and I want so many more people to share in the experience.
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