A four-year old US boy’s life – and that of his family – has been completely transformed since he started treatment on a “miracle drug” for his severe autism four months ago, reports New York Post.
Previously, Lev Bielski was self-harming until he was black and blue, unable to make eye contact, and sat 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 little boy, 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.
Then research led the 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.
They managed to get Lev the therapy before FDA approval, thanks to the FDA’s expanded-access programme – and the results, they said, were immediate.
“It was the next day,” Bielski said. “It was right away …it changed everything for us.”
He has predicted that the drug will be the “Ozempic of autism”.
What separates this investigational oral therapy from other treatments is its focus on brain signalling involved in social interaction, rather than just the behaviours surrounding autism.
With FDA fast-track designation, Yamo said L1-79 is poised to become the first FDA-approved therapy for the core symptoms of autism, with the potential to transform the lives of millions. And as the drug moves into crucial Phase 3 trials, Bielski is determined to see that transformation happen.
“We hope that the FDA works closely with Yamo because right now, there are zero solutions, and this has the best shot of being that,” he said.
With about one out of every 31 children diagnosed with autism, the “ground-breaking” drug could change millions of lives, said Dr Tom Megarian, a veteran paediatric neurologist and CMO & EVP of Clinical Development at Yamo.
Phase 2 results from a 12-week study presented last year showed what Yamo described as “unprecedented” gains in socialisation, alongside critical strides in communication skills and speech patterns, with parents describing an overnight transformation.
“As a clinician practising in this space for 30 years, I’ve never seen that level of improvement in that short a period. I haven’t even seen that level of improvement in that many children over years of therapy,” said Megarian.
Yamo boasted a highly favourable safety profile – a key distinction from the potentially serious side effects associated with antipsychotic medications.
“The benefit has been almost miraculous, and there have been no serious side effects,” Dr Eric Bartky, a child, adolescent and adult psychiatrist who treats Lev, told The Post, noting the positive outcomes for mild to severe cases.
Taking the oral formula administered twice a day is a highlight for Lev. “He’s excited to take it because he kind of knows it’s helping him,” said his father.
But even with the all-important FDA fast track designation, Yamo still needs $75m to move to the pivotal phase 3 trials with enough meds to support the full study.
Bielski said he is betting big on the revolutionary drug. The Wall Street broker and his wife are personally investing in Yamo.
As founder and CEO of a leading exchange-traded fund issuer, Defiance ETFs, he said his company would launch the first-of-its-kind Autism Impact ETF in which all net advisory profits for the first two years are donated to autism-related causes.
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