Fuel a New Treatment for Autism Spectrum Disorder and Beyond
One in 68 children in the United States is on the autism spectrum. Approximately 1 million Americans are battling chronic fatigue syndrome (CFS). Each year, close to 6,000 people are diagnosed with ALS and given a two- to five-year life expectancy. These and many other chronic diseases can be traced to mitochondrial dysfunction. The personal and public health cost of these complex conditions exceed $2 trillion annually, and many have no viable treatments, let alone a cure. However, Robert K. Naviaux, MD, PhD, and his colleagues at The Naviaux Lab are exploring new perspectives in chronic diseases in order to enact revolutionary change for patients.
At his lab at the University of California, San Diego, Dr. Naviaux bases his work in his Cell Danger Response (CDR) Theory, which hypothesizes that a large percentage of chronic diseases may be the result of a reversible metabolic syndrome that, when activated by genetic mutations or environmental triggers, causes cells to “stick” in the midst of the healing cycle. When this occurs, cells behave as though they are still in distress, even after injury or illness has passed. CDR Theory states it is this block to the normal healing cycle that creates the disruption to normal function, leading to chronic disease.
Applying this theory, the team has made a number of promising research forays. Dr. Naviaux’s most notable accomplishment is his discovery that a single dose of suramin, a century‐old drug originally used for African sleeping sickness, provides signiﬁcant reduction in the core symptoms of Autism Spectrum Disorder (ASD). When given in a single dose, suramin was found to be safe and to dramatically improve the core symptoms of autism, including enhancing language and social interaction and reducing repetitive and restricted behaviors.
Additional trials are in development to test if continued treatment can lead to even further improvements. Dr. Naviaux also believes this therapy may prove eﬀective for CFS and other chronic diseases.
The Naviaux Lab relies almost exclusively on funding from philanthropic partners. In joining forces with our community, we seek to bring clinical trials for a number of chronic diseases, like ASD and CFS, from concept to reality.
As Dr. Naviaux and his team negotiate and formalize logistics, we call upon partners like you to fuel hope and excitement and fortify the laboratory infrastructure that is critical to the clinical trial process. With your generous support, we can position the Naviaux Lab to advance the trials needed for Food and Drug Administration approval of suramin to treat ASD. You can be a part of accelerating the possibility of bringing this unparalleled treatment to patients worldwide. Thank you for being a source of inspiration as we continue this life-changing work.
Suramin and Autism Spectrum Disorders
For the last 20 years, Dr. Naviaux has studied the role of mitochondria in health, disease and healing. In 2008, he formulated the CDR Theory. The CDR is a natural and universal cellular reaction to injury or stress, and consists of about 30 metabolic pathways that work together to defend the cell. When cells are distressed, the response jumpstarts the healing process by hardening cellular membranes, ceasing interactions with neighboring cells and withdrawing until the threat of illness or injury has passed. During CDR, cellular mitochondria produce adenosine triphosphate (ATP), which is released from the cell to indicate danger. Although CDR to some degree is necessary for healthy function, it becomes problematic when cells continue to release ATP after the body has healed. Accordingly, Dr. Naviaux scoured literature to find any therapeutic that could inhibit extra ATP signaling and help restore normal cellular function. Only one was available for human use — suramin.
Suramin works by inhibiting the signaling function of ATP, essentially telling cells they are no longer threatened and can return to normal neurodevelopment, growth and healing. Dr. Naviaux and his team investigated suramin as a solution for ASD, and found in animal studies that the drug effectively moderated ATP and ASD-consistent symptoms. From that juncture, the team recruited children with ASD to participate in a small human clinical trial, which wrapped in 2016 (see link below for published article). Participants who received suramin experienced significantly more powerful benefits from their usual therapies and enrichment programs, showed more interest in and aptitude with social interactions, and some even spoke a full sentence for the first time in their lives. These results did not occur in any participants on a placebo drug.
Dr. Naviaux aims to embark on larger and more diversified clinical trials to further assess the safety and efficacy of the drug, particularly when used to treat children. If results replicate the initial trial, suramin would become the very first and only available pharmaceutical treatment for ASD.
For published data regarding Dr. Naviaux’s first clinical trial using suramin for ASD, please reference the Annals of Clinical and Translational Neurology.
For more details, including the parent testimonials of the five children who received suramin, please refer to UC San Diego's suramin media package.
For more information about Dr. Robert K. Naviaux and the Naviaux Lab, visit naviauxlab.ucsd.edu.