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My son Matt was diagnosed with ALS in April 2014, just a few months before the ice bucket challenge took off on social media.
Many people remember this as a rare moment when our country was united in the hope of solving a terrible problem. For our family, it felt like the universe was rallying to our cause.
But hope only goes so far. Nearly a decade later, people with ALS still have no effective treatment options—only the assurance of a slow, humiliating death.
If FDA doesn’t approve this treatment this year, Matt and many other children will lose their lives. (iStock)
An experimental treatment under FDA review, debamestrocel, could change all that. My son Matt is the only person to receive multiple doses of the treatment through an alternative pathway. It helped him regain the ability to breathe, eat and move his hands.
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Today, he is losing what he gained because he can no longer access the treatment. If FDA doesn’t approve this treatment this year, Matt and many other children will lose their lives.
Over the years, I have grown close with a group of mothers of children with ALS. Our community is small, but through our challenges we have forged a great bond. One mom lost two sons. Others had limited access to debamestrocel through the clinical trial or the company’s expanded access program, but like Matt they can longer get the drug.
We all want the chance to have more time with our children, and we share a belief in this treatment based on what it’s done for Matt.
By 2018, he struggled to swallow, which made choking incidents a frequent and terrifying ordeal. The weight of his lungs made breathing a conscious act that he could only perform while awake, so he slept using equipment to support his respiration. I knew this was the telltale sign that we were on an irreversible trajectory to increasingly invasive breathing assistance, and death.
Desperate, we petitioned the Department of Veterans Affairs to gain access to an experimental drug that uses a patient’s own cells to counteract the neurodegeneration process and help nerve cells survive. The results were staggering.
On the day Matt got his first injection, he nearly choked to death at dinner. But within a month of starting treatment, he stopped choking while eating altogether. He could also breathe without mechanical assistance and was able to sleep without respiratory support. He soon regained the ability to remove his own sunglasses, scratch an itch, and touch his children’s faces.
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Within six months, Matt was able to rise and stand without help. It was nothing short of miraculous. (His medical progress is documented here.)
Matt was able to receive a total of seven injections. However, he started losing ground soon after treatment stopped, and we relived much of the same nightmare all over again. Matt can no longer drive his chair or reach up and touch his children’s faces. It is a small mercy that he can still breathe on this own and sleep without support. But seeing him in this state again when we know what’s possible is breaking this mother’s heart.
Securing FDA approval for debamestrocel is our only hope now. An advisory committee will meet on Sept. 27 to review all available data and advise on the path forward. Everything about Matt’s experience supports the published clinical trial data and reflects what other participants in the clinical trial and the expanded access program have reported. There is no doubt the treatment has had a favorable impact on the progression of this disease.
Mothers of children with ALS are helplessly watching our children wither away under the burden of this disease. For those who have seen the benefits of this treatment, every day that passes without a decision is like a slow death unto itself.
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All we ask is that the FDA exercise regulatory flexibility in the approval of this treatment for all people living with ALS today and for those diagnosed in the future.
This treatment is not cure, but it can certainly allow people living with ALS a chance to survive until another treatment is found. The ALS community deserves to see their disease treated with the same approach as other, now chronic diseases like cancer, HIV and diabetes.
In those diseases patients are receiving therapies that extend their lives while research continues seeking a cure. Through slow incremental innovation these diseases are eminently treatable.
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We know this treatment worked for my son Matt. It would be a travesty to continue to keep it away and the other children who got benefit or could gain benefit. The horror of waiting is only made worse seeing my dear friends also watch their own children decline.
In the case of this treatment, perfect is the enemy of the good. In a few weeks we will know the fate of our families. The FDA holds their lives in their hands.
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