Canavan Gene Therapy

Canavan Research Illinois is a major contributor to the pioneering Canavan AAV Gene Therapy Trial for Canavan Disease at Cooper Health System, a division of Robert Wood Johnson Hospitals, Camden, N.J. 

This trial is led by the world's leading researcher in gene therapy for Canavan disease, Paola Leone, Ph.D. Dr. Leone (pictured with Max) has received the UNESCO lifetime Achievement Award for her devotion to developing a treatment/cure to save the little victims of Canavan disease. 

Twenty-one patients have already been approved for the present phase with more approvals to follow as the trial results continue to show marked improvements in the children. This genetic brain transfer involves the insertion of six catheters into the brain that deliver a liquid virus containing 600 billion to 900 billion viral particles to replace a missing enzyme. The AAV-ASPA virus is deemed a safe virus. 

None of the children who have been treated thus far have experienced any side effects and they are released from the hospital within days of the surgery. Several of the children treated thus far have already experienced documented improvements. Max (pictured) is resting after his gene transfer surgery.

The children such as Max (pictured L.) are carefully prepared for continuing MRI’s, ongoing clinical tests, and evaluations to medically evaluate their progress. All the children treated thus far have experienced improvements in their quality of life and several are already showing the growth of new myelin with less build up of a toxic substance known as NAA. This early success is astounding and has great significance for the treatment of other neurodegenerative diseases.

Due to the promise that this ongoing trial holds for all brain diseases, the NIH has provided partial funding. This is the first time that an NIH grant has ever been offered for gene therapy for a brain disease. Canavan Research Illinois has fully committed this charity to continued fundraising to help to support the costs of this trial that offers hope to so many. Recuperating from the gene transfer surgery Sasha Lyubarsky (pictured R.) is comforted by her mother Elana.

Breakthroughs in gene replacement therapy have not only improved the quality of life for children battling Canavan disease, but offer benefit to millions suffering from debilitating neurological diseases such as ALS, Parkinson's, Tay-Sachs, Alzheimer's, Multiple Sclerosis, and stroke.

Recently an  experimental vector, developed for delivery of corrected genes to the Canavan children, was used in a clinical trial for Parkinson's disease.