Treating ‘Fragile X’

Diwen Shi | New University

Through isolation and the utilization of naturally occurring marijuana-like chemicals in the brain, French national research agency INSERM, in collaboration with UC Irvine professor Daniele Piomelli, have successful established a treatment for behavioral issues attributed to the neurological disorder, Fragile X Syndrome (FXS).

FXS, the most commonly known genetic cause of autism, is caused by the mutation of the FRM1 gene that is located on the X chromosome. The mutation causes the loss of fragile X mental retardation protein, which regulates signaling at key receptors in the brain. Inhibited signaling of this kind has been known to cause behavioral issues and other neurological issues, attributed as traits common to autism.

The study performed by Piomelli involved mice genetically altered with FMR1 mutations exhibiting traits of FXS Treated with novel compounds, that boost natural endocannibanoid signaling, mice with FXS showed dramatic behavioral improvements in maze tests.

“Experimental data suggest that boosting endocannabinoid signaling might be useful in a variety of medical conditions, including tobacco addiction and schizophrenia,” Piomelli said in regards to potential utilization of endocannibanoids for other disorders with similar behavioral issues.

Still, with so many benefits of endocanniboids and their enhancers, questions of potential treatment with naturally occurring cannibanoids has already arisen.

Some researchers have already begun experimentation with marijuana and other cannibanoids to test the therapeutic effects on behavioral issues, due to their similar effects as endocannibanoids.

“THC in marijuana is chemically different from the endocannabinoids, but produce similar effects because it combines with the same receptor on the surface of brain cells,” Piomelli said, commenting on the use of marijuana in other studies to treat disorders like autism.

However, the question of negative feedback loops that eventually inhibit positive effects is a concern that has many researchers questioning the trade-offs.

“There is a risk for the cannabinoid receptor to become insensitive to the drug over time,” Piomelli said. “That’s a concern.”

And with such stigma surrounding use of narcotics like marijuana in medical treatments, research utilizing cannibanoids seeking for treatments must work around ethical concerns as treatments progress to the testable human level after such success in other testing species.