Genetic research in the past few years has revealed that autism unexpectedly shares common roots with cancer. Based on these intriguing findings, some researchers are turning to rapamycin, a proven cancer drug, as a potential treatment for autism.
Of the 18 candidate genes for autism uncovered so far, 3 genes ― PTEN, TSC1 and TSC2 ― are part of a biochemical pathway with a long-established role in cancer.
Knocking out these genes in mouse brains causes enlarged neurons, seizures and behaviors similar to autism.
“[This] brings about an enormously optimistic idea that, for some small subset of kids with autism, treatment of this pathway with a drug might reverse some symptoms,” says microbiologist Arnold Levine of the Institute for Advanced Studies at Princeton University.
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