L1-79, a novel therapy for the treatment of autism, is being developed by Yamo Pharmaceuticals and has demonstrated promising clinical results in early testing. This agent has been developed that can produce reductions in the core symptoms of autism as measured by the commonly accepted and validated psychometric scales used to diagnose autism (e.g. ADOS, etc.). Preliminary data shows that within days of starting L1-79 therapy, there was a reduction in some patients’ irritability, lethargy, hyperactivity and anxiety, and improvements in speech, communication, socialization and awareness of themselves and others, resulting in improved behavior and better manageability of the patients.
L1-79 has been well tolerated in all patients. This agent is a modified formulation of an agent that was approved by the U.S. Food and Drug Administration (FDA) and which has an extensive body of associated clinical experience that should serve to expedite the regulatory approval process.
Underlying the safety and efficacy of L1-79 is a novel mechanistic theory of action that integrates extensive bodies of pre-existing medical research into a more unified theory of autism than has been previously possible. This theory includes the ability to modulate the functions of the following:
- Mechanisms in the central nervous system (CNS) which regulate mood, attention, reward, irritability, attention, motor function and other elements in the brain which appear to be affected by autism;
- Different systems in the CNS which serve to modify brain function through inhibition and which are believed to be deficient in the hyperactive CNS state that characterizes autism;
- Mechanisms in the gut which have been implicated as underlying causes of autism;
- Mechanisms in the liver and pancreas that are altered in the presence of autism, and;
- Importantly, a novel system of integrated neurologic and metabolic signaling that unites the gut, the mesenteric organs, the central nervous system and the autonomic nervous system.
The concepts that comprise this theory have found application in a single agent that has been found to be well-tolerated to-date and with the ability to reduce the core symptoms of autism in many of the first group of patients treated with L1-79. Improvements in the core symptoms of autism may occur quickly and continue over time with repeated treatment.