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Iron chelation drug used to treat rare, devastating neurodegenerative disease in children

January 16, 2013
Children's Hospital & Research Center Oakland
A 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.

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 for more information about enrolling in the trial.

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Materials provided by Children's Hospital & Research Center Oakland. Note: Content may be edited for style and length.

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Children's Hospital & Research Center Oakland. "Iron chelation drug used to treat rare, devastating neurodegenerative disease in children." ScienceDaily. ScienceDaily, 16 January 2013. <>.
Children's Hospital & Research Center Oakland. (2013, January 16). Iron chelation drug used to treat rare, devastating neurodegenerative disease in children. ScienceDaily. Retrieved December 2, 2023 from
Children's Hospital & Research Center Oakland. "Iron chelation drug used to treat rare, devastating neurodegenerative disease in children." ScienceDaily. (accessed December 2, 2023).

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