NBIA Disorders Association Scientific and Medical Advisory Board (SMAB)
Position statement:  use of experimental drugs and other therapies, including deferiprone
 
The NBIA Disorders Association’s SMAB recognizes that families want to explore a variety of treatment options for NBIA. For some individuals and families, this includes considering experimental drugs or other therapies. Specifically, in recent months the NBIA community has learned of a drug trial in Italy, in which 10 participants are currently enrolled to analyze a drug called deferiprone. Deferiprone is a chelator, meaning it removes iron from the body. Unlike other chelators, it can cross the blood-brain barrier and is suspected, therefore, to be able to remove excess iron from the brain. Although deferiprone is not approved by the FDA for use in the United States, we are aware of a few families who have gained access to the drug through FDA compassionate use applications.

The SMAB cannot recommend or promote the use of any experimental treatment; that is a discussion and decision that you should make with your physician. It is important to recognize that the use of deferiprone in NBIA is experimental and there have been no scientific studies to support the idea that it may be helpful in NBIA by removing iron. Although iron accumulates in some areas of the brain in NBIA and excess iron could be harmful, iron is also essential for normal brain function and development. Therefore, clinical studies are needed to determine whether deferiprone is safe and effective. However, we recognize that some families may pursue experimental treatments and want to emphasize that careful, constant monitoring for harmful side effects must be a top priority. Based on what we have learned about deferiprone, we would recommend the following (at a minimum) for those considering taking it:

1) A detailed plan should be developed with a patient’s physician team prior to starting use of deferiprone and followed carefully.
2) Blood should be drawn weekly to measure the white blood cell count. It is important to monitor white blood cells because a rare, serious side effect is a lowered white cell count, which compromises the ability to fight infections and can be life-threatening.
3) Every three months blood should be drawn to measure serum ferritin (iron level), zinc, and liver function (ALT). 
4) The possible major and minor side effects of deferiprone and potential drug interactions of deferiprone with other drugs should be reviewed periodically with your physician team. Instructions should be given by the physician team regarding what to do if a fever or infection (even a cold or mild flu) or any other adverse events develops in the person taking deferiprone.

Again, the decision to take an experimental treatment or to participate in a clinical trial should be made only after you have thoroughly discussed and understand the potential benefits and risks with your physician.

Appendix

We realize that NBIA families may decide with their physicians to try an experimental new drug or other unproven treatment. Even if this is not done as part of a clinical trial, there is opportunity to collect valuable clinical information that could help lay the foundation for future trials. 

For this reason, we are providing the following rating scales that families can share with their physicians. These can also be used to establish a baseline and follow progression in individuals using more conventional treatments. Two of the three forms can be completed by the individual with NBIA and/or a close family member. The Barry-Albright dystonia scale, which we have modified slightly for NBIA, should be completed by a neurologist and only takes a few minutes to complete. Completing these scales at regular intervals, such as every 6 months, will help to collect the same information across all individuals with NBIA. 
Rating Scales

 
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