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Together we can make a big difference |
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This is not a call for help, but an invitation –to join us on our journey that began with saving the life of a single child. A journey that takes us towards a treatment that could give life to many children affected by currently incurable brain diseases. Reading our story costs nothing – just two minutes of your time.
In July 2008 we were faced with every parent’s nightmare - our third child, Lavi, was diagnosed with a fatal and terminal disease called Canavan Disease. The prognosis of children diagnosed with this rare brain disease is a tragically short life in a vegetative state. Naturally we were  devastated by this diagnosis; for days we searched for anyone who would be willing to even attempt to reverse the course of this disease. We found a scientist in the United States who has been the leader in groundbreaking research on Canavan Disease since 1995 - Dr. Paola Leone, who has developed innovative medical treatments specifically for Canavan patients. One of these is a pharmacological therapy that Lavi began at the age of three months and succeeded in delaying his brain deterioration. Today, Lavi is a medical miracle – he can see and hear, and is slowly developing neurologically rather than regressing into vegetative state as predicted in the diagnosis.
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About Dr. Paola Leone and the CGTC |
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Paola Leone, Ph.D.
Dr. Paola Leone is an Associate Professor of Neurosurgery and Director of the Cell & Gene Therapy Center (CGTC). As Principal Investigator at UMDNJ-Robert Wood Johnson Medical School for Gene Therapy for Canavan Disease, she leads all research activities at UMDNJ and the affiliated centers. Her research interests have included in vivo neurochemistry of epilepsy and gene transfer approaches for the treatment of neurological disorders. She has published extensively in the field of CNS gene therapy and conducted the first direct gene therapy trial using a liposome-based vector for a pediatric neurodegenerative disorder. In addition to applications of viral vectors and stem cells, Dr. Leone is studying pharmacological approaches in humans and in animal models of Canavan Disease, Amyotrophic Lateral Sclerosis, Tay Sach’s and other neurological disorders.
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