International Clinical Trial Uses Iron Chelation Drug to Treat Rare, Devastating Neurodegenerative Disease in Children at Children’s Hospital & Research Center Oakland
Children’s Hospital & Research Center Oakland’s promising pilot study of deferiprone for the treatment of the neurodegenerative disorder, PKAN, leads to an international trial with potential implications for Parkinson’s and other neurodegenerative diseases.

January 16, 2013

Oakland, Calif. – A groundbreaking, international clinical trial of an iron chelation drug, deferiprone, to treat the rare and devastating neurodegenerative disease, pantothenate kinase-associated neurodegeneration (PKAN), began last month at the trial’s only North American site, Children’s Hospital & Research Center Oakland. Children’s Oakland is one of six clinical centers participating in an international consortium called Treat Iron-Related Childhood-Onset Neurodegeneration (TIRCON). The international consortium is studying the effect of deferiprone on patients with this rare, inherited nervous system disorder characterized by iron build-up in the brain and progressive difficulty with movement, speech and vision, which eventually proves fatal in children and teens.

Elliott Vichinsky, MD, Children’s Oakland’s Director of Hematology/Oncology and an expert in iron disorders, obtained FDA Investigational New Drug (IND) approval for pilot treatment of PKAN patients with deferiprone, the only oral iron chelator that crosses the blood-brain barrier. In 2007, Dr. Vichinsky’s patient, Brent, then 13, was one of the first patients to receive deferiprone for PKAN. News of Brent’s and others success with the drug spread throughout the tight-knit global PKAN community and Dr. Vichinsky’s team has been fielding phone calls from eager parents hoping to get their children on a clinical trial of the drug.  

Dr. Vichinsky, the trial’s Principal Investigator, has been trying to secure funding for a clinical trial of deferiprone for PKAN for the last three years. Dr. Vichinsky and international neurodegeneration with brain iron accumulation (NBIA) leaders Susan Hayflick, MD, and Penny Hogarth, MD, at Oregon Health & Science University, and Professor Thomas Klopstock, MD, at Klinikum der Universitat Munchen in Germany, were able to initiate the TIRCON study, a phase III international trial designed to obtain FDA approval.

The funding finally came in early 2012 when TIRCON was awarded a € 5.2 million (approximately $6,692,240) research grant by the European Union’s (EU) European Commission. In September 2012, the FDA awarded Children’s Oakland another $1.59+ million to fund the study. ApoPharma, the Toronto-based pharmaceutical company that produces deferiprone, joined this international effort and is helping support the clinical trial.

Each of the 130 patients enrolled in the study will participate in the double-blind, placebo-controlled trial for 18 months. Children’s Oakland is the only site outside of Europe and will enroll the largest cohort of 40 patients.

”Families with children affected by NBIA have been an inspiration to me. This study offers hope and the potential to decrease their suffering,” said Dr. Vichinsky.

Neurodegeneration with Brain Iron Accumulation (NBIA) is a hereditary disease characterized by brain iron accumulation in the basal ganglia causing progressive dystonia, spasticity, and early death. There is no effective therapy to halt or reverse the disease.  Pantothenate kinase mutation (PKAN), is the most common cause of NBIA. It is a rare disease that only affects an estimated one to three cases per million births; however the implications of this research extend into other neurodegenerative disorders involving brain iron accumulation including Parkinson’s disease, Alzheimer's disease, and Friedreich's ataxia.

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

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About Children’s Hospital & Research Center Oakland
Children’s Hospital & Research Center Oakland is Northern California’s only independent not-for-profit regional medical center for children. Children’s Hospital Oakland is a national leader in many pediatric specialties and sub-specialties including hematology/oncology, neonatology, cardiology, orthopedics, sports medicine, and neurosurgery. The hospital is one of only two solely designated California Level 1 pediatric trauma centers with the largest pediatric inpatient critical care unit in the region. Children’s Hospital has 190 licensed beds, 201 hospital-based physicians in 30 specialties, more than 2,700 employees, and an annual operating budget of more than $350 million. Children’s is also a premier teaching hospital with an outstanding pediatric residency program and a number of unique pediatric subspecialty fellowship programs. To learn more about Children’s Hospital & Research Center Oakland first 100 years, go to www.100amazingyears.org.

Children’s research program, Children’s Hospital Oakland Research Institute (CHORI), is known internationally for state-of-the-art basic and clinical research and translating it into interventions for treating and preventing human diseases. CHORI has 300 members of its investigative staff, a budget of about $50 million, and is ranked among the nation’s top 10 research centers in National Institutes of Health funding to children’s hospitals. For more information, go to www.childrenshospitaloakland.org and www.chori.org.

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