What is a chelating agent?

A medication that is used to remove excess iron in the body
The chelating agent most commonly used by NBIA patients is deferiprone (Ferriprox)
- Deferiprone is better at removing iron from the brain than other chelating agents

How effective is it?

We currently don’t know how effective deferiprone is and how much brain iron it can remove
The results of one small phase II pilot trial [Zorzi et al 2011]:
- Deferiprone was used in individuals with PKAN
- It was well tolerated by the 9 individuals who completed the study
- It showed a statistically significant reduction of iron in the pallida (a section of the brain)
- There was no change in individual’s clinical status (symptoms)
- A longer treatment period may be necessary to produce clinical results
An international clinical trial with longer duration is currently underway: http://www.clinicaltrials.gov/ct2/show/NCT01741532

Chelating agents other than deferiprone cause anemia but don’t decrease brain iron levels
The effectiveness of deferiprone is still unknown
- It may be treating the symptoms but not the cause of iron accumulation
- Decreased brain irons levels may not improve clinical outcome or overall health
Side effects of deferiprone include:
- Nausea
- Stomachache
- Neutropenia (a low level of white blood cells) can occur rarely
- A low amount of white blood cells can lead to serious infection and death
  - While taking deferiprone, patients must have their blood drawn weekly in order to monitor their white blood cell count