OUR RESEARCH

GENE THERAPY
The most recent advances have been especially exciting. The first clinical trial partially funded and approved by the Food and Drug Administration (FDA) and the National Institute of Health (NIH), in which a new and more potent viral vector is being used to transport the genes into the brains of Canavan children, is ongoing at the Robert Wood Johnson Medical Center. Many of the children have been monitored, and the parents of each and every child are reporting clinical improvement, as supported by MRI and MR spectroscopy scans. Lindsay Karlin, now eleven years old and the first child treated in the first trial in 1996, has shown no deterioration since receiving the latest gene vector. Researchers expect to use the findings from this study to apply to research for other, more common diseases, including Parkinson's Disease, Alzheimer's Disease, multiple sclerosis and stroke.

STEM CELL THERAPY
The Canavan Research Foundation is also supporting stem cell therapy, the transplantation of normal neural stem cells into the brain. Stem cells have the potential to develop into any type of cell, and the research teams that we are supporting are currently developing stem cells that will take over for the faulty cells in the brains of Canavan children and produce the enzyme that the children lack. We are focused on identifying and supporting researchers who demonstrate the greatest potential to bring their work to clinical trial in the next two years. Stem cell therapy may also hold the key to curing a host of other diseases, including genetic and degenerative diseases, stroke and traumatic brain and spinal cord injury.

Our objective is to speed up research through private funding so that it may become available as quickly as possible to those who need it - adults and children whose time is running out.

What We've Achieved

  • Donec condimentum enim sit amet felis dignissim laoreet adipiscing dignissim.
  • Vivamus egestas urna sed lorem sagittis, id lacinia erat porta cras.
  • Morbi eu elit in justo auctor dignissim. Sed ultrices eget neque tellus.
  • Sed fermentum eget velit at eleifend. Donec volutpat est a felis pulvinar.
  • Donec condimentum enim sit amet felis dignissim laoreet adipiscing porta. 
  • Vivamus egestas urna sed lorem sagittis, id lacinia erat porta cras.
  • Morbi eu elit in justo auctor dignissim. Sed ultrices eget neque tellus.
  • Sed fermentum eget velit at eleifend. Donec volutpat est a felis pulvinar.

OUR TEAM

Medical Director

Roger H. Karlin, M.D.

Scientific Advisory Board

Roscoe Brady, M.D.
Department of Neurosciences - National Institute of Health (Bethesda, MD)

Ruben Matalon, M.D.
Department of Neurosciences - University of Texas at Galveston (Galveston, TX)

Andrew Freese, M.D.
Department of Neurosurgery - University of Minnesota (Minneapolis, MN)

Christopher Janson, M.D.
Department of Neurosurgery - University of Minnesota (Minneapolis, MN)

Scott W.J. McPhee, Ph.D.
Research Scientist in Department of Surgery in the Division of Neurosurgery - University of Medicine & Dentistry of New Jersey (Camden, NJ)

Jeremy S. Francis, Ph.D.
Research Scientist in Department of Surgery in the Division of Neurosurgery - University of Medicine & Dentistry of New Jersey (Camden, NJ)