Posted on 10/02/2026 6:27:29 AM PDT by Twotone
Like most 4-and-a-half-year-olds, Lev Bielski loves playing with his big sister, spelling his favorite pastime “pool” on the letter board and cuddling with his parents on movie nights.
But it’s been a long journey for the golden-haired “surfer bro” who loves splashing in the water at surf camp and trying on Dad’s volunteer firefighter gear.
Up until four months ago, Lev was self-harming until he was black and blue, unable to make eye contact and keeping to himself in a corner all day. He was inching dangerously close to being confined by a punishing helmet and sedated with heavy antipsychotic meds to mitigate his severe autism that left him incapable of functioning by himself.
The outlook was bleak for the tormented tyke, who showed symptoms before he was a year old.
His parents, Gabriella and Matthew Bielski, chased every therapeutic intervention and specialist across the country for years, to no avail. The family desperately needed a lifeline, as no medication is approved to address the core symptoms related to autism.
“Half my day was running a business and half was holding him down and trying to keep him from hurting himself — we had no solution,” Matthew Bielski told The Post.
That was until research led the anguished couple to Yamo Pharmaceuticals, a decade-old clinical-stage biopharmaceutical company dedicated to treating the core symptoms of autism spectrum disorder with its “miracle drug” called L1-79.
(Excerpt) Read more at nypost.com ...
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L1-79 is an investigational, twice-daily oral drug developed by Yamo Pharmaceuticals to treat core social communication and interaction deficits in Autism Spectrum Disorder (ASD).
Unlike current medications that only manage behavioral outbursts or irritability, L1-79 is designed to address the core social challenges of autism.
How L1-79 Works
• Target Enzyme: It inhibits tyrosine hydroxylase, which is a rate-limiting enzyme the body uses to produce brain chemicals like dopamine and norepinephrine.
• Calming Brain Signaling: It reduces overactive catecholamine (dopamine and norepinephrine) signaling linked to social and sensory difficulties in autism.
• Broader Effects: By modulating these pathways, it aims to rebalance the neural circuits that govern mood, attention, motor skills, and social behavior.
Clinical Trial Status
• Phase 2 Results: Completed Phase 2 trials showed statistically significant and clinically meaningful improvements in socialization scores (such as an average 8-point improvement on the Vineland-3 scale) for adolescents and young adults.
• Regulatory Status: The U.S. Food and Drug Administration (FDA) has granted L1-79 Fast Track Designation.
• Next Steps: Yamo Pharmaceuticals is preparing to advance L1-79 into Phase 3 clinical trials.
Why This Matters
• Unmet Need: There are currently no FDA-approved medications that treat the core social and communication symptoms of autism.
• Existing Options: Legacy drugs like risperidone are only approved to treat secondary symptoms like irritability and aggression, and they carry significant side effects.
• Safety: Clinical studies to date have reported a favorable safety profile and good tolerability in patients
Wonder if this little tyke “visits” his friends “on the hill”.
Wonder if this little tyke has had all of the recommended childhood vaccines.
I hope the news is real. Looks promising. Phase two trial completed last year under FDA was positive. That was undertaken before the gutting of the FDA, so at this point hard to know if there’s more progress.
My grandson is profoundly autistic. I am calling his parents immediately.
Names of tyrosine hydroxylase inhibitors include:
Demser
Metyrosine
https://www.rxlist.com/how_do_tyrosine_hydroxylase_inhibitors_work/drug-class.htm
Another under reported accomplishment of Trump and his passing the “Right to Try” Act.......
Perhaps, because the FDA was “gutted”, this miracle will move forward rapidly.
By the way, I was not aware of the FDA being “gutted”. I will research that.
Generic Name: Metyrosine
Brand Name: Demser
one drug, two names
Every time I read about a “miracle drug”for mental problems it brings to mind the movie “Awakenings”.
Hope it’s not the case in this situation.
The evidence is starting to become more and more clear that the action most significantly correlated with autism is the mother taking lots of tylenol during pregnancy. That’s what Kennedy and Oz say their preliminary investigation is pointing to. I can say that my wife’s cousin took a lot of tylenol during her pregnancy for her autistic son.
Nothing should be ruled out until it can be ruled out. But that’s where the evidence seems to be pointing right now.
“AMPT was used in the 1960s for preoperative pharmacological control of catecholamine overexpression that causes hypertension and other arterial and cardiac abnormalities. The use of AMPT to treat Pheochromocytoma prior to surgery was discontinued due to its extensive side effects.”
https://en.wikipedia.org/wiki/%CE%91-Methyl-p-tyrosine
Same.
My 8-y/o daughter is Level III. That's real autism, not Reddit "autism is my superpower" autism.
Watching her struggle and considering her future is challenging and heartbreaking.
Acetaminophen:
As father of a son with developmental disabilities (Down syndrome,) I’m pretty clued into the community of parents.
The hope of a magic bullet is ever-present.
The fast-tracking of Yuma’s therapy does not mean that it is approved. Joseph Coyle, director of McLean Hospital’s Laboratory for Psychiatric and Molecular Neuroscience (Belmont, MA,) said “The decision blows me away. The drug affects several distinct neuronal systems most of which have nothing to do with autism.”
Maybe 20 years ago, the New York Times published a frontpage story about a cancer cure which a couple months later had to be retracted. I am hopeful but skeptical of this kind of story.
We are very early in the verification of the drug. Anecdotes don’t cut it. Hopefully the science pans out.
[Separately, I’m familiar with the story of the Bielski partisans in the Second World War. More people should know about them.]
Thanks for that summary. Potentially the best and most wonderful news that has been posted in a very long time.
I’m on the Spectrum and my mother was chasing a sedative with a cocktail while smoking a cigarette while pregnant with me.
Dad says autism ‘miracle drug’ drastically and immediately improved his son’s severe symptoms.
How can he tell
AMPT has a different chemical structure than L1-79, close, but not the same. And we know that for a such low molecular weight compound it matters. It also has two chiral centers which indicates the presence of possibly four stereoisomers in the drug substance. It would be interesting to know if only one is responsible for the favorable therapeutic effect (remember the thalidomide story?...).
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