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,
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.
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.
children such as Max (pictured L.) are carefully prepared for continuing MRIís,
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
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
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.