Recently, Meagan Johnson contacted dozens of pharmacies in search of leucovorin for her 3-year-old son, Jack, who has autism. Courtesy of Meagan Johnson
As highlighted by CNN
Megan Johnson recently spent four days calling dozens of pharmacies in the Austin area, trying to obtain a prescription for her son Jack.
Jack has autism. At age three, most children’s vocabulary ranges from 500 to 1,000 words, while Jack speaks about 20. According to Johnson, most of these words would be understood only by her.
Johnson hopes that the prescription drug leucovorin may help children with abnormally low folate levels in the brain. During a press conference last year, the White House named it a potential autism treatment, and she became interested in whether it might be suitable for Jack as well.
Leucovorin is a high-dose folic acid, previously used mainly to mitigate toxic side effects of chemotherapy. In a few small studies, there were hopeful results using the drug off-label for children with autism.
In September it gained momentum after statements by federal health officials, including U.S. Health Secretary Robert F. Kennedy Jr. and FDA Commissioner Dr. Marty Makary, who said the drug could help some children with autism, and noted that the FDA had begun the process of approval for a condition called cerebral folate deficiency, which has features similar to autism.
Doctors of Jack were skeptical. Both his pediatrician and a developmental pediatrician refused to prescribe the tablet, noting that the studies were not convincing enough. Finally Johnson found a neurologist who agreed to prescribe the drug on a trial basis.
“I know this isn’t a magic pill that will instantly teach my son to speak, but any improvement, no matter how small, would be a big win for me.”
With the new prescription in hand, Johnson thought the hardest part was behind her – until the battle for the actual pills began.
She estimated that she had called at least 40 pharmacies in Pflugerville, Texas, where she lives, trying to find one that had the medication in stock. She first contacted the grocery store pharmacy where she had previously filled prescriptions, but to no avail – and in the following days received the same response from almost all other pharmacies in the region, whether large chains or small family-run stores.
She wasn’t alone: in online support groups, parents with new leucovorin prescriptions often seek advice when they can’t find the pills.
«I poured 100 percent of my energy into this. I was on the phone from morning until dusk, calling and calling again,» said Johnson, who is currently not employed full-time but can devote more time to this.
A new study, published Thursday in The Lancet, highlights the reasons leucovorin has become so hard to obtain.
Research and expert assessment of the supply situation
A few weeks after the White House announcement, leucovorin prescriptions doubled and stayed high at least through the first week of December, according to a study analyzing data from nearly 300 million patients in a large national medical records database.
The study’s author, Dr. Jeremy Foster, an emergency physician at Brigham and Women’s Hospital, said he was concerned but not surprised by the surge in prescriptions. “I think the White House podium is a very powerful place, and people listen to our leaders, even when RFK Jr. says things like ‘don’t listen to my medical advice.’ People are increasingly choosing the care they want based on politics. It’s sad,” he said.
“I think the White House podium is a very powerful place, and people listen to our leaders, even if Robert Kennedy Jr. says things like ‘don’t listen to medical advice from me.’ People are increasingly choosing care based on politics. It’s sad.”
According to Foster, such a trend is troubling because evidence for leucovorin for autism is weak and does not justify changes in practice without further, higher-quality studies. “This is more a feeling. You take one piece of low-quality research and anchor it to the conclusions,” he noted. “I worry about what that means.”
Even without large placebo-controlled trials, some parents and doctors do not see significant risk in trying the drug – a water-soluble vitamin that is easily excreted in urine and has few serious side effects.
The surge in interest created what drug-supply experts call a demand shortage. In such a shortage, sales pull stocks off shelves. Manufacturers continue to produce the drug, but may not keep pace with new orders, said Erin Fox, Deputy Chief Pharmacy Officer at the University of Utah’s Drug Information Service.
“Generic manufacturers usually plan production about a year, and sometimes two years ahead. So they probably did not anticipate such demand,” Fox said. “Even if the White House warned them, that does not mean they can change the production schedule to make more.”
The drug shortage list maintained by the American Pharmacists Association for Health Care Systems shows that most leucovorin manufacturers have tablets in distribution or on backorder. Two manufacturers – Hikma and Pfizer – are listed as having the drug in stock.
In December the FDA issued a Dear Provider letter to alert doctors who import leucovorin from Canada and Spain to ease the shortage. However, the FDA has not officially listed leucovorin as a drug in shortage, and such designation could trigger additional steps to ease supply, including authorizing certain pharmacies to manufacture medicines for distribution.
«I’ve never seen the FDA import a product in shortage if it’s not on their shortage list,» Fox noted.
Although the government allows importation of the drug, that is not enough to ensure full stocks across regions.
“We are seeing supply issues among certain versions of generic leucovorin,” – said Roslyn Guarino, CVS Health spokesperson. “If a local CVS pharmacy is temporarily out of stock, our pharmacists do everything they can to ensure patients have access to the medications they need, and when possible – work with patients and prescribing doctors to identify potential alternatives.”
Laura Bray from the nonprofit Angels for Change, which works to prevent drug shortages, said she began taking action last spring after news stories highlighted the use of leucovorin for children with autism. She knew that inexpensive generics like leucovorin, which historically fall into shortages, are especially sensitive to sudden swings in demand.
She collaborated with Mark Cuban’s CostPlus Drugs to add the drug to that online platform, where fragments of tablets are still kept. She added that when she receives calls from parents seeking leucovorin, she directs them there, but emphasizes that parents should not search for medicines that way.
Finally Johnson managed to find a grateful pharmacist at CVS who stayed on the line and checked an internal database to locate a sufficient quantity of tablets for her son’s prescription at a pharmacy nearly an hour from home. Even after a long journey she felt relief, obtaining the drug and giving Jack the first dose that same evening.
Bray noted that parents shouldn’t have to go through so much trouble to find medicines.
“It was entirely predictable – this would happen,” Bray said. “The steps that could have been taken to avoid it were also known.”
She also added that if her nonprofit anticipated this and acted, the government should have done the same.
Johnson agrees: “It shouldn’t be this hard. It just shouldn’t.”
The CNN correspondent Michal Ruprecht contributed to this report.