From Wall Street to the search for an autism treatment
by Matt Bielski · The Washington TimesOPINION:
For most of my adult life, I have lived in the world of Wall Street.
I founded Defiance, a leading ETF issuer with approximately $12 billion in assets under management. I built a successful career identifying emerging technologies and connecting them with the power of capital markets.
Then our son Lev was diagnosed with profound autism. Suddenly, the future became personal.
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Lev is 4 years old and struggles to communicate. Like millions of other families affected by autism, my wife, Gabriella, and I entered a world we never expected to know: doctors, researchers, studies and an endless search for answers.
I began reading medical papers instead of analyst reports. What we discovered stunned us.
Autism has been diagnosed in 1 in 31 children in America, yet no medication approved by the U.S. Food and Drug Administration is indicated to treat the core symptoms of autism.
As a father, I could not understand that. As someone who knows capital markets, I could not accept it.
Our search led us to a drug called L1-79, an investigational therapy to treat the core symptoms of autism. It is now preparing to move into Phase 3 clinical development.
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That got our attention. We eventually invested in the company developing L1-79 because we believed that the science behind the drug deserved the opportunity to prove itself.
Still, this was personal, not just an investment.
Gabriella led our family’s successful efforts to pursue access to L1-79 through the FDA’s individual patient expanded access pathway. Through an individual patient expanded-access Investigational New Drug application, Lev received L1-79. The experience has been life-changing.
I do not say that lightly, and I say it cautiously.
For the first time in Lev’s life, we have seen what so many parents of autistic children dream of: meaningful improvements in our son’s communication and eye contact. We have watched him become more engaged with the world around him. We have also seen a significant reduction in agitation.
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For parents of a child who struggles to communicate, these moments of connection mean everything.
I am not a physician. One child’s experience is not a clinical trial. Lev’s experience cannot establish whether L1-79 is effective for another child with autism. That requires clinical evidence.
Still, I am Lev’s father, and I know my son. What Gabriella and I have witnessed has made me determined to help answer that scientific question.
Getting there requires something I understand well: capital.
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Drug development is expensive. A promising medicine does not reach patients simply because scientists believe in it. Researchers conduct trials, regulators need evidence, manufacturing must be scaled, and someone has to finance the enormous cost of finding out whether the medicine actually works.
Wall Street finances enormous risks every day. My firm, Defiance, has directed billions of dollars toward artificial intelligence, semiconductors, quantum computing and space exploration because investors believe that those technologies can change the future.
Why should we not bring that same ambition to autism? That question has become a personal mission for me.
At Defiance, we recently launched the Autism Impact ETF, trading under the ticker ASD, and we plan to donate profits from the fund to support the autism community. Yet the ETF is only part of what must happen.
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The capital markets must be opened to autism drug development. Developing drugs for autism is difficult, risky and expensive, but if investors do not finance those risks, promising treatments may never receive the trials they need.
L1-79 is approaching that moment. It has progressed through early trials. Its next challenge is the most important one yet: moving through Phase 3 trials to determine whether the drug is effective in a larger population.
I do not know whether L1-79 will become the first FDA-approved medicine for the core symptoms of autism — that is what Phase 3 will determine — but our family’s experience makes Gabriella and me want that question answered.
I know how fortunate we are to have had the resources, access and ability to find answers for our son.
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I often think about the parent who does not, such as the single mother trying to hold down a job while raising a child with profound autism. She may not have access to specialists, time to read studies or resources to pursue every option. Her days may simply be about getting her child through school, keeping her family together and making it to work the next morning. She also deserves hope.
That is why this matters.
President Trump and Health and Human Services Secretary Robert F. Kennedy Jr. have committed to making progress on a cure for autism. I share that goal, but in the interim, we should be equally focused on therapies that treat the symptoms.
Our family had the resources, connections and means to pursue the FDA’s individual patient expanded access pathway, but most do not. The FDA must expand access to the program. Mr. Trump’s Right to Try initiative from his first term could help thousands more families access a drug such as this.
Although my focus has been on raising additional capital to help L1-79 prove itself, I am calling on the FDA and HHS to fulfill their roles and expand access.
Additional capital and increased access will hopefully help create a market for autism drug development, encouraging other researchers and investors to pursue new therapeutics. The impact could extend far beyond any single medicine.
If even one more parent gets to experience what I have with Lev — more communication, eye contact and connection — then every bit of this effort will have been worth it.
We started this journey to help our son. Now we want to make sure the next family does not have to search as hard as we did.
• Matt Bielski is an investor and advisory board member at Yamo Pharmaceuticals. His 4-year-old son, Lev, has profound autism and received Yamo’s investigational autism therapy, L1-79, through the FDA’s expanded access program.