Cutting-Edge Autism Therapy & Treatment Options

In the United States, 1 in 59 children now are diagnosed with autism spectrum disorder (ASD). Most recently, noteworthy advances have been made ranging from research into the cause and biology of ASD to the evaluation of new techniques for earlier identification and intervention.

Read on for some of the most recent and advanced research findings related to autism treatment.

Reversing autism with experimental cancer drugs

7% of ASD cases have been tied to chromosomal defects, suggesting that difficulties in social interaction, communication, behavior, and movement are tied to genetics. Specifically, some people with autism are missing a small portion of the chromosome 16. Known as 16p11.2 deletion syndrome, a defect in this chromosome often leads to impairment in language skills and neurodevelopmental disability. Now, using experimental cancer drugs, researchers may have found a way to reverse this genetic form of autism. Prof. Riccardo Brambilla of Cardiff University in the UK and fellow colleagues were able to reverse the condition in mice.

“The human 16p11.2 microdeletion is one of the most common gene copy number variations 56 linked to autism,” explained Prof. Brambilla and his team. Yet, the mechanisms of this chromosomal defect and its ties with autism are poorly understood. For this reason, the research team decided to design a mouse model of the chromosomal defect in order to examine its physiopathology.
The researchers witnessed that the mice displayed behavioral and molecular abnormalities, including dysfunctions in their maternal behavior, hyperactivity, and problems with their olfactory perception. Moreover, they found that the mice with 16p11.2 deletion syndrome had higher levels of the ERK2 protein. Since ERK2 has been emerging as a target in cancer treatment, the researchers decided to examine the effect of experimental cancer drugs on mice with 16p11.2 deletion.

They found that the cancer drugs stopped ERK2 from reaching the mice's brains, which reversed the autism-like symptoms. “Importantly, we show that treatment with a novel ERK pathway inhibitor during a critical period of brain development rescues the molecular, anatomical, and behavioral deficits in the 16p11.2 deletion mice,” wrote the researchers. The experimental cancer drugs not only provided relief of symptoms for the adult mice, but also prevented mice genetically predisposed to the condition from being born with a form of ASD. This occurred when the drugs were administered in the mother mouse during pregnancy.

Prof. Brambilla noted that while it would not be possible to treat pregnant women who have been screened for the genetic defect, it may be possible to permanently prevent the condition by treating the infant as early as possible following birth. “In the case of adults with the condition, ongoing medication would probably be required to treat symptoms” he added.

In the near future, the researchers hope to be able to test the experimental cancer drugs in human clinical trials and to replicate their findings.

Treating autism with cancer drugs is revolutionary enough, but there are even more advancements being made. Read on to learn more.