Century-old drug offers new hope for autism treatment
Groundbreaking research has identified a potential new treatment for the core symptoms of autism.
A 100-year old drug improves core features of autism in a double-blind placebo trial
Results released from an N of One: Autism Research Foundation supported small clinical trial in boys with autism may represent one of the most dramatic advances in autism yet. The findings offer both a new view of what autism is and the possibility of the first drug to treat its core symptoms.
The encouraging results from the double-blind, placebo-controlled trial that tested the 100-year-old drug, suramin, were published in the Annals of Clinical and Translational Neurology. In the trial, conducted by Dr. Robert Naviaux at the University of California San Diego School of Medicine, 10 boys ages 5-14 years were given either a single dose of the drug suramin or a placebo. Improvements were seen in all three core features of autism: language, social interactions, and restricted or repetitive behaviours across multiple diagnostics in multiple tests in all 5 boys who were given the active treatments. Those improvements were not seen in any of the children who received a placebo.
Suramin was developed in 1916 to treat African trypanosomiasis, also known as sleeping sickness. It is administered intravenously and is currently only available in hospitals for treating sleeping sickness disease.
The only observed side effects in this study was a temporary mild rash, which disappeared without treatment in 2-3 days. The study used a low dose of suramin, which produced blood levels of 5-15 µM. All previous uses of suramin have been at much higher doses – doses used for sleeping sickness produce blood levels of 50-100 µM for 1-3 months. High doses used for cancer chemotherapy produce blood levels of 150-270 µM for 3-6 months.
In order for this drug to become available to the wider autism community, its effects need to be confirmed and optimal dosage and safety protocols established in bigger studies and tested on large numbers of individuals. The researchers are hoping to start the next phase of their research in 2019.
“On a Saturday about a week after the infusion, his dad was making a snack in the kitchen. My son (14 years old, non-verbal) went in and said very clearly the first sentence of his life. He looked at his dad and said, “I want to eat chips.” (parent of a study participant)
“This work is new and this type of clinical trial is expensive,” (Dr Naviaux) said. “We did not have enough funding to do a larger study. And even with the funding we were able to raise, we had to go $500,000 in debt to complete the trial.”
Naviaux R.K, Curtis B., Li K., et al. (2017) Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial. Ann Clin Transl Neurol. doi:10.1002/acn3.424.
Cheng N., Rho J.M., & Masino S.A. (2017) Metabolic Dysfunction Underlying Autism Spectrum Disorder and Potential Treatment Approaches. Front Mol Neurosci. Feb 21;10:34. doi: 10.3389/fnmol.2017.00034.
Naviaux J.C., Schuchbauer M.A., Li K., et al. (2014) Reversal of autism-like behaviors and metabolism in adult mice with single-dose antipurinergic therapy. Transl Psychiatry. Jun 17;4:e400. doi: 10.1038/tp.2014.33.
Naviaux J.C., Wang L., Li K., et al. (2015) Antipurinergic therapy corrects the autism-like features in the Fragile X (Fmr1 knockout) mouse model. Mol Autism. Jan 13;6:1. doi: 10.1186/2040-2392-6-1.
Naviaux R.K. (2014) Metabolic features of the cell danger response. Mitochondrion. May;16:7-17. doi: 10.1016/j.mito.2013.08.006.
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