CHORI Staff Directory
CHORI Intranet

Making Hope A Reality
CHORI Senior Scientist Spearheads Novel Treatment of Rare Neurogenerative Disease

January, 2013 – CHORI Senior Scientist and Director of Hematology/Oncology at Children's Hospital & Research Center Oakland, Elliott Vichinsky, MD, has been pioneering the use of the iron chelator, deferiprone, in the treatment of pantothenate kinase-associated neurodegneration (PKAN), a form of neurodegeneration with brain iron accumulation (NBIA). Dr. Vichinsky’s efforts culminated in the December, 2012 launching of an international clinical trial in collaboration with other researchers in the United States and Europe to study desferiprone in patients with PKAN. Researchers hope the trial will eventually result in the United States’ Food and Drug Administration (FDA) approval for use of the drug in PKAN patients.

“Families with children affected by NBIA have been an inspiration to me,” says Dr. Vichinsky. “This drug offers hope for the first time of decreasing their suffering, and with this study, we have the potential to make that hope a reality.”
“This drug offers hope for the first time of decreasing their suffering, and with this study, we have the potential to make that hope a reality.”

NBIA is a rare, inherited, neurological disorder characterized by iron accumulation in the brain that causes progressive difficulty with movement, speech and vision, and is eventually fatal in children and adolescents. Deferiprone is most commonly used to treat iron overload in patients with blood disorders who require regular transfusion therapy. It is also the only oral iron chelator that can cross the blood-brain barrier. In 2007, Dr. Vichinsky obtained Investigational New Drug (IND) approval from the FDA to study the drug on a compassionate use basis in a patient with PKAN.

The results of that pilot treatment were so successful that Dr. Vichinsky has been working for the past three years to secure funding for a clinical trial in a larger population. Those efforts culminated at the end of 2012 in a phase III international trial designed to win FDA approval for deferiprone use in PKAN patients.

The trial, in collaboration with Susan Hayflick, MD and Penny Hogarth, MD at Oregon Health & Science University and Professor Thomas Klopstock, MD, at Klinikum der Universitat Munchen in Germany, is being led by the international consortium, Treat Iron-Related Childhood-Onset Neurodegeneration (TIRCON), with funding from the European Commission, the FDA, and ApoPharma, the Toronto-based pharmaceutical company that produces deferiprone.

TIRCON will enroll 130 patients in the double-blind, placebo-controlled trial for 18 months. Children's is the only site outside of Europe and will enroll the largest cohort of 40 patients.

“While PKAN only affects a very small number of patients, it is a devastating condition and we hope to alleviate the suffering of patients with PKAN and their families.”

"In addition, however, the results of this study may also be applicable to other neurodegenerative disorders such as Parkinson's disease, Alzheimer's disease, and Friedreich's Ataxia," says Dr. Vichinksy.

Children's is currently recruiting patients for the clinical trial. Please contact Nancy Sweeters, RN, PNP at (510) 428-3885, ext. 4151 for more information about enrolling in the trial.


Friday, April 12, 2013 7:38 AM

© 2005 Children's Hospital Oakland Research Institute
5700 Martin Luther King Jr Way • Oakland, California 94609
Phone 510-450-7600 • Fax 510-450-7910
Site MapDisclaimerCHORI Intranet