I met Dr. Paola Leone in 1996 after my 6 month old son, Jacob, was diagnosed with Canavan, a fatal genetic brain disease.  Doctors told me my son would never reach any milestones, would live trapped in his body, lose his eyesight, develop seizure, lose his ability to swallow and die within the first decade of his life.   There was nothing the medical community could offer and Canavan was so rare no scientist would want to work on it because funding was too difficult to access.  Fortunately, my desperate search to find someone willing to help led to Dr. Leone, who was working on cutting edge gene-therapy research.   Her work was once focused on Parkinson's, but when introduced to 2 Canavan children, she immediately wanted to help and indeed she did.  Within a year, the 2 girls received gene-therapy, and one developed new white matter, the substance that transports impulses from our brain.  Children with Canavan cannot develop white matter because they are missing an essential enzyme.  Dr. Leone’s gene-therapy delivered this enzyme to the brains of the 2 children.

That marked the beginning of Dr. Leone’s career in translational research.  Dr. Leone has become world renowned for her work which led to two FDA approved gene-therapy Canavan trials and pharmaceutical interventions that have stopped the progression of the disease in Canavan children worldwide.  Her passion for working with others and sharing research has led to treatments in more commonly known disorders such as Parkinson’s and ALS.  

Dr. Leone is now leading the way in cutting edge stem-cell research and hopes to begin a Canavan clinical trial early next year. Because of her prominence amongst scientists worldwide and her colleagues at the National Institute of Health and the FDA, a revolutionary partnership was created between Dr. Leone and Geron Biotech, who was eager to share its stem cells with Dr. Leone for her impending Canavan clinical trial.  Geron saw Canavan as an excellent model for the treatment of white matter disease, and they were eager to work with a scientist who had a proven record for getting clinical trials approved, strong relationships with the NIH & FDA and the passion for moving research from the bench to the patients.

I proudly nominate Dr. Paola Leone for dedicating the last 16 years of her life to curing Canavan children worldwide and for being an innovator, a collaborator and a person whose compassion will revolutionize the way Canavan and white matter disease is treated.