Century Old Drug Provides Promising Furture In Autism Treatment

A new study conducted by researchers at the University of California, San Diego (UCSD) School of Medicine was recently published in Translational Psychiatry. Researchers found that a century old therapy originally meant to treat sleeping sickness may actually be used to reverse autism symptoms. The drug appeared to restore normal cellular signaling in a mouse model of autism by reversing the symptoms of autism in animals that were the human biological age of 30 years old.

Dr. Robert K. Naviaux, co-director of the Mitochondrial and Metabolic Disease Center at UC San Diego explains, “Cells behave like countries at war. When a threat begins, they harden their borders. They don’t trust their neighbors. But without constant communication with the outside, cells begin to function differently. In the case of neurons, it might be making fewer or too many connections. One way to look at this related to autism is this: When cells stop talking to each other, children stop talking.”

The mice groups used in the study are engineered to exhibit symptoms of autism spectrum disorder and so the researchers gave them the drug suramin, which was created in 1916 to treat African sleeping sickness, hoping that it would block the signal pathway that would stimulate the “cell danger response.” They found that the drug did work to this end and the cells and metabolism in the mice actually began behaving normally. Though this trial was successful, the drug is not permanent or preventative as a single dosage was only effective for about 5 weeks. Also long the drug cannot be used for long term since it can have serious side effects of anemia and adrenal gland dysfunction.

However the study was promising in that they were able to create normal cell behavior in the mice. Later this year they will be conducting a clinical trial that will assess the treatment using children with ASD.

Dr Naviaux suggests that “the treatment, rather than being used as an autism ‘cure,’ may be used effectively to complement non-drug behavioral and developmental therapies.” He says that this new drug provides a new perspective on the way we thing and address the challenges of autism. 

ICare4Autism’s International Autism Conference, less than two weeks away, will be talking about new drug developments and biomedical perspectives on Day 2 of the conference. Martha Herbert, director of the TRANSCEND research program and pediatric neurologist at Mass General Hospital,  will be giving a presentation called “Taking a Fresh Look at Autism: Chronic Dynamic State—Not Fixed Trait.” To see her presentation and others, CLICK HERE!