The objective of Dr. Markov's project is to define whether a nutritional supplement (anapleurotic therapy) capable of promoting energy metabolism can improve brain development and psychomotor function in a mouse of model of Canavan Disease. Anaplerotic diet therapy is based on the concept that an energy deficit in certain diseases is improved by providing an alternative substrate for Krebs cycle and enhanced ATP production.

Unlike other therapeutic approaches which have a relatively long lead time, anapleurotic therapy may represent the shortest route to a disease modifying clinical therapy for Canavan disease. This study will generate safety and efficacy data to support the development of a Phase I study to test anapleurotic therapy in patients affected by Canavan Disease.

According to one member of the review board, "[Dr. Markov's project] addresses a long-standing question of pathophysiology that must be answered."