Shortages of leucovorin drug affect autism treatment for children
Recent reports highlight difficulties in obtaining leucovorin, a drug sought by parents of children with autism, amid rising demand following increased prescriptions. Meagan Johnson spent four days contacting numerous pharmacies in the Austin area trying to fill a prescription for her son, Jack, who has autism and reportedly has limited verbal communication skills, reports BritPanorama.
Johnson believes leucovorin could aid Jack, given its potential to address low folate levels in the brain, as noted during a White House news conference last year where it was described as a possible treatment option for some children with autism.
The drug, a high-dose folinic acid primarily used to mitigate chemotherapy side effects, has received attention after some families reported improvements in language and social skills in their nonverbal children. Small studies suggest promising results for its off-label use in autism cases, further raising its profile.
In September, US Health and Human Services Secretary Robert F. Kennedy Jr. and FDA Commissioner Dr. Marty Makary indicated that leucovorin might assist children with autism, announcing a procedural initiation for FDA approval for its use in cases of cerebral folate deficiency—a condition that could overlap with autism diagnostics.
Despite her hope, Jack’s pediatrician and developmental specialist were hesitant to prescribe the medication due to the limited research backing its use. Ultimately, Johnson located a neurologist willing to prescribe leucovorin on a trial basis.
“I know it’s not a magic pill that’s going to make my child speak overnight,” she remarked, expressing her desire for any improvement, no matter how minimal.
With the prescription acquired, Johnson thought her challenges were behind her. However, her search to fill it proved arduous; she called approximately 40 pharmacies in Pflugerville, Texas, encountering stock shortages, whether at large chains or smaller stores.
Johnson’s experience mirrors numerous other parents in online support groups, who have reported similar difficulties in procuring leucovorin prescriptions. “I put literally 100% of my energy into it. I was on the phone from daylight till dark,” she noted, acknowledging her capability to dedicate significant time due to her current job status.
A recent study published in the journal The Lancet reveals that the rise in leucovorin prescriptions, which doubled within weeks of the White House announcement, contributed significantly to its scarcity, with the high demand seen through at least the first week of December.
Dr. Jeremy Faust, an emergency medicine physician, expressed concern about the spike in prescribing such a medication without solid evidence to justify its extensive use. He highlighted that leveraging political influence in medical decisions could lead to unfortunate outcomes.
Without robust trials to support leucovorin’s efficacy for autism, Faust worries the medical community is relying on low-quality research, undermining more rigorous scientific methodologies.
Nevertheless, some parents and healthcare providers see minimal risks associated with trying leucovorin, as it is a water-soluble vitamin with few adverse effects, which contributes to its increasing demand.
This surge has induced a demand-side shortage, with experts indicating that while drug manufacturers are producing leucovorin, they are struggling to meet the accelerated order rates resulting from the public interest spurred by recent announcements.
The American Society of Health-Systems Pharmacists indicates most leucovorin manufacturers have their supply on allocation or backorder, though two—Hikma and Pfizer—report having stock available.
In December, the FDA issued an uncommon “Dear Provider” letter to inform healthcare professionals of plans to permit imports of leucovorin from Canada and Spain to help alleviate the shortfall, despite the FDA not officially listing the drug as being in shortage.
Roslyn Guarino, a spokesperson for CVS Health, confirmed supply challenges for certain generic versions of leucovorin, stressing that their pharmacy teams work diligently to ensure patient access to needed medications.
Laura Bray, who manages a nonprofit focused on combating drug shortages, expressed her proactive efforts to prevent a lack of leucovorin supply, aware of the medication’s vulnerability to sudden demand shocks. She has collaborated with Mark Cuban’s CostPlus Drugs to improve the availability of this medication.
Ultimately, Johnson’s persistence led her to a supportive CVS pharmacist who located enough stock to fulfill her son’s prescription, despite the long drive involved in obtaining it. Yet, Bray argues that parents should not face such hurdles in accessing essential treatments for their children.
Johnson’s sentiment reflects broader frustrations, emphasizing, “It shouldn’t be this hard. It just shouldn’t.” As experts and advocates call for more systematic approaches to prevent similar drug shortages, the conversation around leucovorin and its role in autism treatment continues to evolve.
