Marty Makary
speaker
939 appearances
6 recordings
5 series
first heard Jan 2026
last heard 13 May
Marty Makary’s voice in public audio — every appearance, attributed to the second.
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recordings per month · last 12 monthsRecordings per month over the last 12 months — 6 in all, peaking in Jan 2026 with 4.
Appearances
There's technology now whereby some companies have started to grow a pancreas cell called the beta cell, that is the cell that makes insulin, in a laboratory and then infuse it into an animal or a human to see if it produces insulin in response to the sugar levels inside that individual.
And there's some very promising results.
Now, these cells historically have gotten rejected by the host, but it may be that that barrier is about to be overcome.
And if so, we want to be there as a regulator.
during that process and walk them through the regulatory process.
Another example, baby KJ at Children's Hospital of Pennsylvania was born with a genetic defect that is super rare and you could never do a clinical trial.
There's just not enough babies with this condition.
We directly worked with the family and the doctors and the company that made a gene therapy and enabled that baby
to get a gene therapy in infancy, something that would have been impossible to think about in a regulatory framework in any other setting.
And so we are going to do things that have never been done before because that's the only way we can significantly advance cures and meaningful treatments in certain areas.
We said this is not a silver bullet.
It doesn't help all kids.
But we were at a point now as a group of scientists where we felt like there is sufficient evidence on Lucavorin to the point where we should make it available to doctors and make the information available to parents of children with autism so they can inquire about it.
Leucovorin has been around for nearly a century with an impeccable safety profile.
And what we identified is an indication for Leucovorin whereby it can help some kids with autism significantly.
Let's say a group of children with severe autism, maybe half,
have an antibody blocking the folate receptor at the brain.
And leucovorin can help some of those kids because it bypasses that blocked receptor, delivering methylated folate directly into the brain.
The brain can otherwise be starving of that folate.
So it appears to be powerful.
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