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In April 2014, my son Matt was diagnosed with ALS, right before the ice bucket challenge gained popularity on social media.
For many, the ice bucket challenge represented a moment of unity and hope in our country’s fight against a devastating problem. For our family, it felt like the universe was rallying behind us.
However, hope can only take us so far. Even after almost a decade, individuals with ALS still don’t have effective treatment options, only the prospect of a slow and degrading death.
If FDA doesn’t approve this treatment this year, Matt and many other children will lose their lives. (iStock)
An experimental treatment called debamestrocel, currently under FDA review, could change everything. Matt is the only person who has received multiple doses of this treatment through an alternative pathway. It has helped him regain his ability to breathe, eat, and move his hands.
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Unfortunately, he is now losing the progress he made because he no longer has access to the treatment. If the FDA doesn’t approve this treatment soon, Matt and many other children will lose their lives.
Throughout the years, I have formed close bonds with other mothers of children with ALS. Our community is small, but our shared struggles have brought us together. Some of these mothers have tragically lost more than one child to ALS. Others had limited access to debamestrocel through clinical trials or the company’s expanded access program, but like Matt, they can no longer obtain the drug.
We all yearn for more time with our children, and we strongly believe in the effectiveness of this treatment based on what it has done for Matt.
By 2018, Matt was struggling to swallow, leading to frequent and terrifying choking incidents. Breathing became a conscious effort for him, and he could only do it while awake. He had to rely on respiratory support when sleeping. Seeing these signs, I knew that his condition was progressively worsening, leading to more invasive breathing assistance and ultimately death.
Desperate for a solution, we appealed to the Department of Veterans Affairs to grant us access to an experimental drug that uses a patient’s own cells to counteract neurodegeneration and promote nerve cell survival. The results were astonishing.
On the day Matt received his first injection, he almost choked to death during dinner. However, within a month of starting the treatment, he stopped choking while eating entirely. He regained the ability to breathe without mechanical assistance and could sleep without respiratory support. He even regained the ability to do simple tasks like removing his own sunglasses and scratching an itch, as well as touching 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. You can read about his medical progress here.
Matt received a total of seven injections, but his progress started to decline once the treatment stopped. We are experiencing the same nightmare all over again. Matt can no longer operate his wheelchair or touch his children’s faces. The fact that he can still breathe and sleep without assistance is a small comfort, but it breaks this mother’s heart to see him in this state knowing what is possible.
Our only hope now is to secure FDA approval for debamestrocel. An advisory committee will convene on September 27th to review all available data and provide guidance on the way forward. Matt’s experience aligns with the published clinical trial data and echoes the experiences of other participants in the trial and the expanded access program. There is no doubt that this treatment has had a positive impact on the progression of ALS.
Mothers of children with ALS are helplessly witnessing their children wither away under the weight of this disease. For those who have experienced the benefits of this treatment, every day without a decision feels like a slow death in itself.
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All we ask is that the FDA demonstrates regulatory flexibility in approving this treatment for all individuals living with ALS today and in the future.
This treatment may not be a cure, but it can provide ALS patients with a chance to survive until a definitive solution is discovered. The ALS community deserves to have their disease treated with the same urgency as other chronic ailments like cancer, HIV, and diabetes.
In these diseases, patients receive therapies that extend their lives while research continues to find a cure. Through incremental innovation, these diseases have become manageable.
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We know this treatment has worked for my son Matt. It would be a tragedy to deny it to him and other children who have benefited or could potentially benefit. The agony of waiting is intensified when I see my dear friends also witnessing their children’s decline.
In the case of this treatment, perfection should not be the enemy of progress. Soon, we will know the fate of our families. The lives of our loved ones are in the hands of the FDA.
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