Featured Research

from universities, journals, and other organizations

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

Date:
January 16, 2013
Source:
Children's Hospital & Research Center Oakland
Summary:
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.


Story Source:

The above story is based on materials provided by Children's Hospital & Research Center Oakland. Note: Materials may be edited for content and length.


Cite This Page:

Children's Hospital & Research Center Oakland. "Iron chelation drug used to treat rare, devastating neurodegenerative disease in children." ScienceDaily. ScienceDaily, 16 January 2013. <www.sciencedaily.com/releases/2013/01/130116163538.htm>.
Children's Hospital & Research Center Oakland. (2013, January 16). Iron chelation drug used to treat rare, devastating neurodegenerative disease in children. ScienceDaily. Retrieved September 21, 2014 from www.sciencedaily.com/releases/2013/01/130116163538.htm
Children's Hospital & Research Center Oakland. "Iron chelation drug used to treat rare, devastating neurodegenerative disease in children." ScienceDaily. www.sciencedaily.com/releases/2013/01/130116163538.htm (accessed September 21, 2014).

Share This



More Mind & Brain News

Sunday, September 21, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Could Grief Affect The Immune Systems Of Senior Citizens?

Could Grief Affect The Immune Systems Of Senior Citizens?

Newsy (Sep. 19, 2014) The study found elderly people are much more likely to become susceptible to infection than younger adults going though a similar situation. Video provided by Newsy
Powered by NewsLook.com
Food Addiction Might Be Caused By PTSD

Food Addiction Might Be Caused By PTSD

Newsy (Sep. 18, 2014) New research shows that women who suffer from PTSD are three times more likely to develop a food addiction. Video provided by Newsy
Powered by NewsLook.com
Corporal Punishment on Decline, Debate Renews

Corporal Punishment on Decline, Debate Renews

AP (Sep. 16, 2014) Corporal punishment in the United States is on the decline, but there is renewed debate over its use after Minnesota Vikings running back Adrian Peterson was charged with child abuse. (Sept. 16) Video provided by AP
Powered by NewsLook.com
FDA Eyes Skin Shocks Used at Mass. School

FDA Eyes Skin Shocks Used at Mass. School

AP (Sep. 15, 2014) The FDA is considering whether to ban devices used by the Judge Rotenberg Educational Center in Canton, Massachusetts, the only place in the country known to use electrical skin shocks as aversive conditioning for aggressive patients. (Sept. 15) Video provided by AP
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins