Drug touted by Trump admin. for autism shows promise for some symptoms
On Monday, President Donald Trump and several members of his administration announced the FDA will approve the drug Leucovorin for treatment of autistic children. The announcement comes as officials announced more research into autism.
FDA approval
“Peer reviewed literature has documented that up to 60% of folate deficient children with [autistic spectrum disorders] have improved verbal communications if given Leucovorin,” Robert F. Kennedy Jr., Secretary of Health and Human Services, said during a press conference covered by Forbes.
Those studies date back more than twenty years and have shown promise amongst health workers.
“I’ve been treating autism using Leucovorin for almost, probably 19-20 years,” Dr. Dan Rossignol, a family physician who’s conducted one of those studies, told Straight Arrow News. “And not every kid responds to it, but the improvements that we tend to see are things like speech and social, which is kind of big because we don’t really have any treatments that are approved to treat speech and social and autism.”
Those studies reportedly showed exactly what Kennedy and Rossignol said.
“There have been at least two clinical trials that are published that were done double blind, placebo controlled and showed significant benefit in terms of language, speech and social interaction in these kids who already are in the autism spectrum,” Edward Quadros, a researcher, Ph.D., and professor at Suny Downstate, told Straight Arrow News.
The administration plans to ramp up those studies.
“[Centers for Medicare and Medicaid Services] is also issuing guidance to convene states and existing research networks to support NIH and FDA in their research efforts of prescription Leucovorin, while also maintaining the privacy of all patients, all health information of our beneficiaries, collecting real world data, which is what many of you have been asking us to do in the community,” Dr. Mehmet Oz, Administrator for the Centers for Medicare and Medicaid Services, said. “It’s fast. It is lifesaving, and we’ll get answers based on what happens over the next few months.”
Having FDA approval will also likely get this drug to more kids.
“If you’ve never used it, you’re kind of nervous, like, I don’t know if I want to do this, right?” Rossignol said. “So of course, if the FDA says okay, then I think more doctors would use it.”
The American Academy of Pediatrics issued a statement to Straight Arrow News.
“There is no single, root cause of autism, and there is no single medication that will give every autistic child or adult what they need,” a spokesperson said. “Individualized plans, often involving a combination of developmental, behavioral, educational and social-relational strategies, can help improve outcomes that are meaningful to individuals and families.”
What is Leucovorin?
Leucovorin is a prescription version of folinic acid. Along with its more common form folic acid, it can be taken as a supplement but the over-the-counter forms come in much smaller doses.
“The dose is going to be so high that you couldn’t really do so well with it as a supplement,” Dr. David Danish, a double board-certified psychiatrist who’s been prescribing Leucovorin for autistic children for years, told Straight Arrow News. “You’d be taking dozens and dozens of supplements of the pills each day.”
The drug has been around for decades and is most-commonly used to help cancer patients undergoing certain chemotherapies. Some forms of chemo deplete patients of folate and Leucovorin helps replenish those levels.
“It’s not a chemotherapy med, it’s just a vitamin that replaces your folate level when it gets depleted by the medication,” Rossignol said.
Because it’s a well-known drug and has been used for decades, doctors are also very aware of the side effects, or the lack thereof.
“There is no long-term issues there with taking like folinic acid over months and years,” Danish said. “You’re doing three months and stopping, and they’re not really seeing any long-term negatives with that. You might see irritability or insomnia in some cases, but you simply stop people if it’s too bad, and they go back to where they were relatively quickly. It’s not a long-term problem.”
How does it impact autism
The doctors said many autistic children have an antibody in their blood that blocks normal folic acid receptors. While researchers cannot totally quantify that just yet, they can see the lower folate levels.
That’s where folinic acid comes in because it can get around that block.
“It can be absorbed by the brain even in the presence of that antibody and folinic acid is sort of a step further down the road in terms of the type of folic acid that we want to use in our cells anyway,” Danish said. “So, it’s getting up into the brain a lot more easily than the normal folic acid when we do the folinic acid as a medication, aka, Leucovorin.
The research found the earlier kids get diagnosed with autism and begin this treatment, the better the results are.
“If you can identify kids before the age of three or even before the age of two, and rigorously treat them, you may prevent a lot of the autistic features that sort of appear later on in life,” Quadros said.
Doctors said they’ve seen real improvements in speech and social skills while using this drug.
“The most important and most common improvement we’re seeing is improved communication, so their speech and language improves,” Danish said. “We have someone, a mom who just wrote in the other day about their child attempting so many new words, which is just out of the blue for them.”
The doctors also made sure to point out this is not a cure-all treatment for autism and won’t work for every child.
So, if you’re a parent of an autistic child, is this for you?
“Families who have questions about their child’s medications, autism care plans or other health care should consult with their pediatrician or health care provider,” The American Academy of Pediatrics said. “The AAP remains committed to providing accurate information to guide pediatricians and parents in making decisions that are family centered and individualized to meet every child’s needs and goals.”
What about the cost?
Drugs rarely come cheap in the U.S. but the Trump administration hopes to help with that piece as well.
“Over half of American children are covered by Medicaid and CHIP government health insurance, over half,” Oz said. “Therefore, because they’re covered in Medicaid, and CHIP upon this label change state Medicaid programs, in partnership with CMS, will cover prescription Leucovorin around the country. It’s yours.”
While that will help children on Medicaid, many of those kids will lose that insurance due to the administration’s recent budget bill.
Despite that, the doctors SAN spoke with believe most insurers will still cover the medication, especially with the FDA approval.
“Most insurance will pay for medication even considered off-label use,” Rossignol said. “Like some people get a seizure med to treat their migraines, right? And the insurance will pay for the seizure med even though they don’t have seizures.”
There also could be some other issues preventing children from acquiring the medication. Supply issues from expanded use since it will now be used more to treat autism along with helping chemotherapy patients.
“Hopefully, what the government does there will help,” Danish said. “That, as well as this news getting out there so other generic drug manufacturers, or some of the drug manufacturers that currently make it, will increase resources to increase that production.”
Despite any challenges, overall, doctors were encouraged by today’s news when it comes to Leucovorin.
“I think it’s generally going to be very positive,” Danish said. “You want to be careful about fast tracking anything, but I will say, in this particular case, I think it actually might be okay.”
“It has a good safety track record,” Rossignol said. “It’s been around for decades. People know the side effects. It may be beneficial for some kids. It’s certainly not the cure all, but I think it’s worth a trial.”
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Ella Rae Greene, Editor In Chief
