Jacob's Cure founder Jordana Holovach met Dr. Paola Leone in 1996 after her 6 month old son, Jacob, was diagnosed with
Fortunately, Jordana’s desperate search to find someone willing to help led her to
That marked the beginning of Dr. Leone’s career in translational research - bringing research directly to the patient. Dr. Leone has become world renowned for her work which led to two FDA approved gene therapy trials for Canavan disease 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. She has also lent her expertise in gene therapy to colleagues working on another childhood brain disorder called Battens. It was her consulting that helped get the FDA approvals the Battens team needed to begin their trial.
Dr. Leone is now leading the way in cutting edge stem cell research and hopes to begin a Canavan disease clinical trial early next year. Because of her prominence amongst scientists worldwide and her colleagues at the National Institutes of Health and the FDA, a revolutionary partnership was created between Dr. Leone and Geron Biotech (leaders in embryonic stem cells) who were eager to share their 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.
Dr. Paola Leone has dedicated the last 16 years of her life to curing Canavan children worldwide. She is an innovator, a collaborator and a person whose compassion will revolutionize the way Canavan disease and other white matter disorders, such as Parkinson's, MS, and ALS, are treated.
Learn More about Dr. Leone and Canavan Research >
Canavan disease, a rare genetic neurological disorder.
Doctors told Jordana that her 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. Equally devastating was when doctors explained to her that there was nothing the medical community could offer and Canavan was such a rare disorder that no scientist would want to work on it because funding was too difficult to access.Fortunately, Jordana’s desperate search to find someone willing to help led her to
Dr. Leone, who was working on cutting edge gene therapy research.
Her work was once focused on Parkinson's, but when introduced to two children with Canavan disease, she immediately wanted to help and indeed she did. Within a year, the two girls received gene therapy, and one developed new white matter, also known as myelin, the substance that transports impulses from our brain. Children with Canavan disease cannot develop white matter because they are missing an essential enzyme. Dr. Leone's gene therapy delivered functioning genes to the brains of the two children.That marked the beginning of Dr. Leone’s career in translational research - bringing research directly to the patient. Dr. Leone has become world renowned for her work which led to two FDA approved gene therapy trials for Canavan disease 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. She has also lent her expertise in gene therapy to colleagues working on another childhood brain disorder called Battens. It was her consulting that helped get the FDA approvals the Battens team needed to begin their trial.
Dr. Leone is now leading the way in cutting edge stem cell research and hopes to begin a Canavan disease clinical trial early next year. Because of her prominence amongst scientists worldwide and her colleagues at the National Institutes of Health and the FDA, a revolutionary partnership was created between Dr. Leone and Geron Biotech (leaders in embryonic stem cells) who were eager to share their 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.
Dr. Paola Leone has dedicated the last 16 years of her life to curing Canavan children worldwide. She is an innovator, a collaborator and a person whose compassion will revolutionize the way Canavan disease and other white matter disorders, such as Parkinson's, MS, and ALS, are treated.
Learn More about Dr. Leone and Canavan Research >