May 13, 2020 — Autism spectrum disorder (ASD) is a complicated condition with multiple causal factors. At the most basic level, we believe ASD is the result of a multisystem cellular response to environmental and genetic stresses. Called the cell danger response, it results in over-release of ATP, the fundamental carrier of energy. Too much ATP signaling disrupts normal functioning in many types of cells, including how neurons communicate and work together.
Suramin is a century-old drug used to treat African sleeping sickness or trypanosomiasis. It works by inhibiting ATP signaling. In animal studies and in a small Phase I/II clinical trial completed in 2017, we reported that suramin produced dramatic reductions in classic ASD symptoms, such as social abnormalities and learning disabilities. These improvements were temporary, and disappeared in about 8 weeks after the single dose of suramin wore off. We are now in development of a second, larger clinical trial. Progress toward this trial has been slow but steady, no surprise given the complexity of ASD and the need to advance this novel approach carefully, fully supported by empirical evidence and best scientific practices.
Like labs around the world, we have not been immune from the effects of the SARS-CoV-2 pandemic. It has caused some delays and complications. Nonetheless, we expect to have all approvals in place to launch the second trial in spring of 2021.
As additional information becomes available, it will be posted at my lab website - naviauxlab.ucsd.edu - and disseminated via news media.
You cannot push science and the development of new therapies beyond what is safe and smart, especially when the lives and well-being of children are involved. The cell danger response theory and the use of suramin as a remedy for it represent a profound paradigm shift in the culture of medicine and the treatment of many diseases.
My lab, in coordination with other research organizations, pharmaceutical companies, advocacy groups and parents of children with autism around the world, is working diligently toward our common goal: A new, effective treatment for ASD and a better, healthier world.
That takes time.
Robert Naviaux, MD, PhD