Medical Research

Until recently there was no hope for a child battling Canavan disease. Canavan Research Illinois (CRI) was founded with the mission to fund pioneering medical research to treat and cure Canavan diseases. Although fatal, as a rare disease, Canavan receives limited government funding, and no pharmaceutical industry support.  We rely on private donations and grants to fund life-saving research. 

Because Canavan serves as a model for other neurological diseases, our advancements can also benefit millions suffering from debilitating neurological diseases such as ALS, Parkinson's, Alzheimer's, Multiple Sclerosis, and stroke. Pictured R. Ilyce Randell met with Chairman Regula  to promote bipartisan government awareness of Canavan disease and the need for funding for research to save the children.

We are a major contributor to Dr. Paola Leone's (pictured with Michael and Ilyce Randell) ground-breaking Gene Therapy Trial for Canavan Disease at Cooper Health System, a division of Robert Wood Johnson Hospitals, Camden, N.J. Dr. Leone is the Director of the Cell & Gene Therapy Center and Associate Professor in the Division of Neurosurgery at UMDNJ-Robert Wood Johnson Medical School. Dr. Leone is also the Principal Investigator of the Canavan Gene Therapy protocol using adeno-associated viral vectors. She is an adjunct member of the Coriell Center for Medical Research, and is the leading researcher in the field of gene therapy for Canavan disease (See Canavan Gene Therapy for details)  

Following post-doctoral studies in Montreal, Dr. Leone was an Associate Research Scientist at Yale University from 1996-1998. From 1998-2001, she was Associate Director of the CNS Gene Therapy Center at Jefferson Medical College and an Assistant Professor in the Department of Neurosurgery. Dr. Leone has been responsible for the development and characterization of viral (AAV, adenovirus, retrovirus) and non-viral vectors for the treatment of Canavan Disease and other disorders. At Yale and Thomas Jefferson Universities, she was the IND/FDA sponsor of two separate “Gene Therapy for Canavan Disease” studies.

We are also funding  Dr. Morris Baslow (a renowned expert in NAA and Canavan disease) at The Center For Neurochemistry. Dr. Baslow is collaborating with researchers at Kyoto University in Japan to develop a non-invasive drug therapy to improve the lives of the children battling Canavan disease. 

In addition, we are funding Dr. Brian Popko of the University of Chicago. Dr. Popko is the Director of the Jack Miller Center for Peripheral Neuropathy and Associate Chair for Research, Department of Neurology. This grant is co-sponsored by Canavan Research Illinois and Jacob's Cure of New York.  Dr. Popko and his team will study the ASPA deficiency in Canavan disease and help determine how different mutations of the ASPA gene possibly determine the severity of the disease in children of different genetic backgrounds and all his results will be shared with Dr. Paola Leone.

Evan Snyder, M.D., Ph.D. a leading researcher in the field of stem cells at Harvard Institutes of Medicine is also a recipient of our support. 

Dr. Snyder is a world renowned researcher and co-founder of the stem cell field. His work focuses on understanding the mechanisms underlying Canavan disease and other childhood neurodegenerative diseases. 

This research investigates Canavan at a molecular and cellular basis...particularly as programmed into the Central Nervous System at the stem cell level. 

Dr. Snyder is studying the potential treatment of stem-cells in Canavan disease. His work will focus on studying stem cells in Canavan animal models with the goal of treating afflicted Canavan children.