Leucovorin prescriptions surged after the White House touted the drug for autism. Parents are still scrambling to find it

By Brenda Goodman, CNN

(CNN) — Meagan Johnson recently spent four days calling dozens of pharmacies across the Austin area, trying to fill a prescription for her son, Jack.

Jack has autism. At age 3, most kids have a vocabulary of 500 to 1,000 words. Jack can say about 20, Johnson said, “and most of those 20, only I can understand.”

Johnson hopes her son could be helped by a prescription drug called leucovorin, which may help some children who have abnormally low levels of the vitamin folate in their brains. During a news conference last year, the White House touted it as a potential treatment for some children with autism. She wondered if it might be right for Jack, too.

Leucovorin is a high-dose vitamin – folinic acid – that until recently was mostly used to treat the toxic side effects of chemotherapy. In some cases, families who have tried it believe it has helped their nonverbal children develop more language and social skills. A few small studies have reported promising results when it is used off-label for children with autism.

In September, it got a boost after federal health officials including US Health and Human Services Secretary Robert F. Kennedy Jr. and US Food and Drug Administration Commissioner Dr. Marty Makary announced that it might help some children with autism and said the FDA had begun the process of approving the drug for a condition called cerebral folate deficiency, which shares features with autism and may affect some children who’ve been diagnosed with it.

Jack’s doctors were skeptical. Both his pediatrician and his developmental pediatrician refused to prescribe the pill, saying there just wasn’t enough research on it. Finally, Johnson found a neurologist willing to prescribe it on a trial basis.

“I know it’s not a magic pill that’s going to make my child speak overnight,” Johnson said, but any improvement, no matter how small, would be welcome. “That is a massive win in my book.”

With a new prescription in hand, Johnson thought the hard part was over – until she tried to fill it.

She estimates that she called as many as 40 pharmacies around Pflugerville, Texas, where she lives, trying to find one that had the pills in stock. She started with the grocery store pharmacy where she usually fills prescriptions but had no luck, and soon she had gotten the same answer from almost all the others in her area, whether they were big chains or small mom-and-pop stores.

She isn’t the only one. In online support groups, parents with new leucovorin prescriptions often seek advice when they can’t find the pills.

“I put literally 100% of my energy into it. I was on the phone from daylight till dark, calling, calling,” said Johnson, who is between jobs, so she was able to dedicate more time.

A new study, published Thursday in the journal The Lancet, is shedding light on why leucovorin has become so hard to find.

Within weeks of the White House announcement, new prescriptions for leucovorin doubled, and they remained high through at least the first week of December, according to the research, which analyzed information on nearly 300 million patients in a large national electronic medical records database.

Study author Dr. Jeremy Faust, an emergency medicine physician at Brigham and Women’s Hospital, said he was concerned – but not surprised – to see the spike in new prescriptions.

“I think that the White House lectern is a very powerful place, and people do listen to our leaders, even though RFK says things like ‘don’t take medical advice from me.’ People increasingly are choosing the kind of care they want based on politics. That’s unfortunate,” he said.

The trend is alarming, Faust said, because the evidence behind leucovorin for autism is thin and doesn’t warrant a shift in practice, at least not without more robust studies.

He said high-quality evidence often takes years to reach medical practice and patients.

“This is just essentially vibes. It’s taking one side of the research that is low-quality and running with that,” Faust said. “I’m concerned about what it means.”

Even without large placebo-controlled trials supporting its use in autism, some parents and doctors see little risk in trying the drug, a water-soluble vitamin that’s easily flushed from the body in urine and has few side effects.

The sudden increase in interest has created what drug supply experts call a demand-side shortage. In demand-driven shortages, a spike in sales cleans off pharmacy shelves. Manufacturers are still making the drug but may not be able to adjust fast enough to keep pace with new orders, said Erin Fox, associate chief pharmacy officer at the University of Utah’s Drug Information Service.

“Generic manufacturers, they plan out their production usually a year, sometimes two years, in advance. So they were probably not counting on this demand,” Fox said. “Even if the White House gave them a heads-up, that doesn’t mean that they can change their production schedule to make more.”

A drug shortage list maintained by the American Society of Health-Systems Pharmacists shows that most manufacturers of leucovorin have the tablets on allocation or backorder. Allocation means they’re rationing existing supplies of the drug and allowing pharmacies order only a limited amount.

Two manufacturers, Hikma and Pfizer, are listed as having the drug in stock.

In December, the FDA issued what’s known as a “Dear Provider” letter to let doctors know that it was taking the rare step of allowing imports of leucovorin tablets from Canada and Spain to help ease the shortfall.

The FDA doesn’t have leucovorin tablets officially listed as being in shortage, however. Such a designation would trigger additional actions to help ease the supply crunch, including allowing certain compounding pharmacies to make more of it.

“I’ve never seen FDA import a product related to a shortfall when it’s not listed as a shortage on their website,” Fox said.

HHS did not answer questions about why leucovorin hasn’t been added to the official list of drug shortages or about the supply in the US.

Alhough the government is allowing imports of the medication, that hasn’t been enough to shore up supplies everywhere.

“We are seeing supply challenges among certain versions of generic leucovorin,” Roslyn Guarino, a spokesperson for CVS Health, said in a statement. “If a local CVS Pharmacy is temporarily out-of-stock of a medication, our pharmacy teams make every effort to ensure patients have access to the medications they need and, if possible, will work with patients and prescribers to identify potential alternatives.”

Laura Bray, who runs the nonprofit Angels for Change, which is focused on preventing drug shortages, said she began working to prevent a shortage of leucovorin last spring, after a news segment highlighted its use for children with autism. Bray knew that cheap generic medications like leucovorin, which has a history of going into shortage, are particularly vulnerable to demand-side shocks.

She worked with entrepreneur Mark Cuban’s CostPlus drugs to get it added to that marketplace, and it still has some strengths of the pills available.

She said that whenever she gets calls from parents who are looking for leucovorin tablets, she sends them to CostPlus. But she said parents shouldn’t have to hunt so hard to find it.

Johnson eventually connected with a helpful pharmacist at CVS who stayed on the phone with her and scoured an internal database to find enough to fill her son’s prescription at a pharmacy nearly an hour’s drive from her home.

Even with a long drive, Johnson was relieved to get it and gave Jack his first dose that night.

Bray says parents shouldn’t have to work so hard to find medications.

“This was completely predictable, that this would happen,” Bray said. “The steps that needed to happen so this wouldn’t happen were also knowable.”

She says that if her small nonprofit predicted it and took steps to alleviate it, the government should have, too.

Johnson agrees. “It shouldn’t be this hard. It just shouldn’t.”

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CNN’s Michal Ruprecht contributed to this report.