Ancient Drug Has Capabilities of Being a New Approach to Autism

A recent study reveals that, can be safely administered to children with autism spectrum disorder (ASD).

In a small, randomized phase I/II medical trial (SAT1), researchers at University of California San Diego School of Medicine say a 100-year historic drug referred to as suramin, originally developed to deal with African sleeping sickness, can be safely administered to children with ASD, who had displayed measurable, however transient, development in core symptoms of autism.

ASD encompasses a group of developmental issues, typically characterized by communication and language difficulties, repetitive behaviors and inability to socialize. The Centers for Disease Control and Prevention estimate that ASD occurs in 1 in 68 children, with the medical condition four times common in boys than girls.

ASD has no single recognized cause, but may just involve both genetic problems and environmental reasons, akin to viral infections, pollution or problems in the course of pregnancy. One of the pursuits of the SAT1 study is to experiment the cell danger hypothesis as a possible unifying conception that contributes to the pathogenesis of ASD.

Writing in the Annals of Clinical and Translational Neurology, author Robert K. Naviaux, MD, PhD, professor of medicine, pediatrics and pathology at UC San Diego School of Medicine and the other researchers describe a novel double-blind, placebo-controlled safety study involving 10 boys, aged 5 to 14 years, all identified with ASD.

Around 5 of the 10 boys received a single, intravenous infusion of suramin, a drug at first developed in 1916 to deal with trypanosomiasis (sleeping sickness) and river blindness, both precipitated by parasites. The other 5 boys obtained a placebo. The trial followed prior testing in a mouse model of autism wherein a single dose of suramin briefly reversed signs of the neurological disease.

The outcome in humans had been remarkable, though the rationale of the SAT1 trial was basically to scan the researchers’ underlying theory about a unifying rationale for autism and to assess the safety of suramin, which isn’t an accepted treatment option for ASD. Currently, there are no approved drugs to treat the core symptoms of ASD.

All 5 boys who used the suramin infusion displayed improvements in language and social behavior, restricted or repetitive behaviors and coping skills. Evaluation of improvements was established upon observational examinations and interviews utilising standardized tests and questionnaires, such as the Autism Diagnostics Observation Schedule, 2nd version (ADOS-2), the Expressive One Word Picture Vocabulary Testing (EOWPWT), the Aberrant Behavior Checklist (ABC), the Autism Treatment Evaluation Checklist (ATEC), the Repetitive Behavior Questionnaire (RBQ) and the Clinical globalImpression (CGI) questionnaire. To decrease misinterpretation of typical everyday variations in signs, the parents were asked to mark a symptom as changed within the 6-week CGI only if the symptom lasted for a minimum of one week.

The researchers found that ADOS-2 ratings were expanded within the suramin treatment group at six weeks, but not in the placebo group. Mainly, ADOS-2 ratings improved by -1.6 points in the suramin group, but did not change within the placebo. Children who have a score of 6 or lower in ADOS-2 will have milder symptoms but not meet the formal diagnostic criteria for ASD. A rating of 7-8 suggests that the child is on the autism spectrum. Nine and above classifies the little one as autistic.

Suramin treatment was associated with improvements in the ABC, ATEC and CGI measurements, but not RBQ. Essentially the most transformed behaviors, the authors said, have been social communication and play, speech and language, calm and focus, repetitive behaviors and coping capabilities.