The connection between autism and leucovorin began with an unexpected detour.
Since President Donald Trump’s press conference on Monday announcing a treatment for autism, research professor Edward Quadros’ phone has been ringing nonstop.
The SUNY Downstate University professor, who has a doctorate in biochemistry and specializes in folate absorption, wasn’t surprised when he heard the news, he told The Epoch Times.
Having leucovorin approved by the U.S. Food and Drug Administration as the first drug to treat autism is the result of more than 20 years of work, Quadros said. Prior to the relabel, it was best known as an adjunct chemotherapy drug given to cancer patients to prevent side effects of methotrexate.
The drug targets a nutritional deficiency in the brain that research estimates affects more than 70 percent of children with autism.
The Discovery of Cerebral Folate Deficiency
The connection between autism and leucovorin began with an unexpected detour.
In 1998, the United States mandated folate, or vitamin B9, fortification in food to prevent neural tube defects in newborns.
Decades of research had suggested that mothers who gave birth to children with neural tube defects were deficient in folate, which affected their babies’ ability to close their spinal cords properly.
Quadros and Belgian neurologist Dr. Vincent Ramaekers were researching why.
Later in 2002, their teams discovered that affected babies had severe folate deficiencies in their brains despite receiving both prenatal and postnatal supplementation. Measuring their spinal fluid showed very low levels of the vitamin’s active form.
It turns out that many of these children with cerebral folate deficiency have autoantibodies to the brain’s main folate receptor. These autoantibodies block the main folate receptors, effectively stopping the brain from absorbing common forms of folate from diet or supplementation.
When these children were treated with folinic acid, or leucovorin—a more readily absorbed form of folate—their symptoms improved.
Of the 25 children they studied with these folate receptor autoantibodies, four had autism.
Leucovorin and Autism
In 2013, a paper led by neurologist Dr. Richard Frye, along with Quadros, found that among autistic children in the clinic, an upward of 75 percent had autoantibodies that can block folate from entering the brain.
By Marina Zhang
