University of California, San Diego (UCSD) Suramin Treatment Trial for Autism

This study is designed to test a new theory of the origin and treatment of ASD. In this
theory, ASD is caused by both genes and environment interacting to produce a persistent cell
danger response (CDR; Naviaux RK, 2014) that interferes with and alters normal child brain
development. Gut microbiome and immune systems are also affected. In this theory, the
pathological persistence of the cell danger response is traceable to mitochondria, and
maintained by purinergic signaling mediated by the release of extracellular nucleotides like
adenosine triphosphate (ATP), adenosine diphosphate (ADP), uridine triphosphate (UTP), and
uridine diphosphate (UDP). Suramin inhibits excess purinergic signaling by acting as a
competitive inhibitor of nucleotide signaling at both ionotropic purinergic (P2X) receptors,
and G-protein coupled, metabotropic purinergic (P2Y) receptors. Suramin has been found to
correct the symptoms, metabolism, and brain synaptic abnormalities in two classical genetic
and environmental mouse models of autism (Naviaux JC, et al. 2015; Naviaux JC, et al. 2014;
Naviaux RK, et al. 2013). This study will test the safety and efficacy of a single dose of
suramin in children with ASD. While it is not anticipated that a single dose will produce
benefits for more than a few weeks, if successful, this study may lead to the development of
newer and safer drugs for autism treatment.

Inclusion Criteria:

- Autism diagnostic observation schedule (ADOS) score of ≥ 7

- Diagnosis of autism spectrum disorder by Diagnostic and Statistical Manual, 5th
edition (DSM-V)

- Stable treatment and diet regimen for ≥ 2 months

- Resident of San Diego region

Exclusion Criteria:

- Any prescription medications

- Hospitalization within the previous 2 months

- Active medical problem such as seizures, heart, liver, kidney, or adrenal disease

- Planning to start a new drug, diet, or behavioral intervention during the study

- Weight under the 5th percentile for age

- Unable to tolerate venipuncture, urine collection, or an indwelling intravenous
catheter for 3-4 hours

- Plasma creatinine ≥ 1.4 mg/dl

- Liver function alanine amino transferase (ALT) or aspartate amino transferase (AST) ≥
1.5-fold above the upper limit of normal

- Known intolerance to suramin or other antipurinergic drugs

- Unable to perform or cooperate with study requirements