Skepticism surrounds autism fast traked

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26 Jul 2018, 2:01 am

Skepticism surrounds austism drug given ‘fast track’ for approval

Quote:
As in the young woman’s case, results from the study — and a subsequent 41-person trial — hint that the drug, L1-79, improves social behaviors in adolescents and young adults with autism, according to J. Thomas Megerian, chief medical officer at Yamo.

The company has yet to publish its results or post them on Clinicaltrials.gov — a federal repository of clinical trial results — so they are difficult to confirm, however.

Despite the dearth of data, in May the U.S. Food and Drug Administration (FDA) granted the drug ‘fast track’ status, according to Megerian. (FDA officials declined to confirm this, citing agency rules.)

Fast-track status is not an endorsement of a drug’s potential: A candidate drug can gain fast-track status if it has the potential to fulfill an unmet need for a serious medical condition; the agency can grant this status at any point during a drug’s review. The drug’s makers can also meet frequently with FDA officials about their study design.

Many scientists say they are surprised by the FDA’s decision because of the lack of published data on the drug’s effectiveness.

“[The decision] blows me away,” says Joseph Coyle, director of McLean Hospital’s Laboratory for Psychiatric and Molecular Neuroscience in Belmont, Massachusetts. The drug affects several distinct neuronal systems, he says, “most of which have nothing to do with autism.”

It’s unclear how blocking signaling by dopamine or other catecholamines would improve social behavior in people with autism — the claim the company makes in the white paper it made available to Spectrum.

In its description of the drug’s proposed mechanism, Yamo “was not only selective, but it was also to the point of being less than scholarly,” says George Anderson, senior research scientist at the Yale Child Study Center. “To think this is in any way improving a fundamental alteration of systems in autism is, I think, misguided.”

In Yamo’s phase II clinical trial, launched in 2016, 31 young men with autism, aged 13 to 21 years, took the drug for 28 days; another 10 took a placebo.

“Nobody thought this was going to show anything,” says Megerian. But to his surprise, he says, most participants taking the drug improved on several scales that assess social behavior.

Parents of children in the trials have given video testimonials that the company shared with the FDA about the drug’s benefits. In one video, a mother tells a story about her teenage son, who sometimes bangs his head on walls and head-butts his brothers. While taking the drug, she says, her son went for weeks without hurting himself; his sleep improved and he accepted his parents’ affection.

However, poignant anecdotes are no replacement for well-designed clinical trials, scientists note. Given the lack of published data, they are concerned about raising families’ hopes of a treatment.

“Parents are desperate for treatment for their kids,” Coyle says. “I can tell you, in terms of the autism community, there are a number of unfortunately fringe, charismatic people offering treatments that have no biomedical validity.”

Yamo officials say they intend to enroll 250 people with autism in a second phase II trial in early 2019.


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Professionally Identified and joined WP August 26, 2013
DSM 5: Autism Spectrum Disorder, DSM IV: Aspergers Moderate Severity

My autism is not a superpower. It also isn’t some kind of god-forsaken, endless fountain of suffering inflicted on my family. It’s just part of who I am as a person. - Sara Luterman