What is leucovorin? FDA to relabel drug for autism treatment

WASHINGTON - The U.S. Food and Drug Administration plans to approve a new use for the generic drug leucovorin as a treatment for kids with autism.

Trump, Health and Human Services Secretary Robert F. Kennedy Jr. and other health officials discussed the medication during a press conference on Monday.

Trump administration discusses leucovorian as treatment for autism

What they're saying:

"I'm also here to announce good news today. The FDA is filing a Federal Register notice to change the label on an exciting treatment called prescription leucovorin so that it can be available to children with autism," FDA Commissioner Marty Makary said, adding, "We have thousands of kids, in my opinion, who will benefit."

Following the press conference, the FDA initiated the approval of leucovorin calcium tablets for patients with cerebral folate deficiency (CFD), a neurological condition that affects folate transport into the brain. 

What is leucovorian?

Dig deeper:

According to Mayo Clinic, Leucovorin is a prescription drug that is used to counteract the harmful effects of methotrexate (a common chemotherapy medication). It is also used to prevent or treat certain kinds of anemia. 

Leucovorin acts the same way in the body as folic acid, which may be low in patients using the drug.

Individuals with cerebral folate deficiency (CFD) have been observed to have developmental delays with autistic features (e.g., challenges with social communication, sensory processing, and repetitive behaviors), seizures, and problems with movement and coordination.

Research on leucovorin’s effect on autism

Big picture view:

The FDA said Monday that previous information supports a finding that leucovorin calcium can help individuals suffering from CFD.  

Last year, researchers studied folinic acid in a small group of children and found that those who received the drug had improved scores compared to the placebo group. They also found that the folinic acid supplementation was effective and safe in improving autistic symptoms.

While scientists say leucovorin could be promising for a subset of autism patients, they cautioned that the current data is limited, and the drug needs more research.

According to the Centers for Disease Control and Prevention, one in 31 (3.2%) of children in the U.S. has autism, up from the previous rate of one in 36. 

FDA works with GSK on relabeling effort

What's next:

The FDA said Monday that it is working with GSK, the innovator of Wellcovorin (leucovorin calcium), on a process to include the essential scientific information needed for the safe and effective use of these drug products for adults and pediatric patients with CFD. 

GSK has preliminarily agreed to work with the FDA on this relabeling effort.

"The FDA is collaborating with GSK to broaden the existing Wellcovorin label," said George Tidmarsh the diirector of the FDA’s Center for Drug Evaluation and Research. "This effort reflects the FDA’s commitment to identify opportunities to repurpose drugs to treat chronic diseases. The FDA remains committed to finding and treating the root causes of autism."

Trump’s HHS claims link between autism and tylenol

The backstory:

This news comes on the heels of the Trump administration’s report claiming autism is linked to the use of the painkiller Tylenol, or acetaminophen, during pregnancy.

RELATED: Autism and Tylenol: Trump’s HHS claims link if acetaminophen taken during pregnancy

The White House event was closely watched by scientists and advocates who say there is no single cause for the disorder. This warning also comes even though acetaminophen has long been considered the safest option for managing headaches, fever and other pain during pregnancy.

What is tylenol?

Tylenol is a brand name for the over-the-counter (OTC) medication acetaminophen, which is used to relieve pain and reduce fever. The medication's generic name is acetaminophen in the U.S. and Canada and paracetamol in other countries, according to MedLine Plus.