PLEASE HeLp!!!!

PLEASE CONSIDER DOING THIS!!!!

Update

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Kulsrud Brothers.

There is a study this summer for the drug Deferiprone that is promising for children with PKAN.  As of now they are considering doing this study with a 1/3 participants receiving a placebo.  This is heart breaking for us since we would like all three boys on the Deferiprone drug.  Please consider filling Dr. Russell Katz's email account with messages of this concern.  

russell.katz@fda.hhs.gov

Russell Katz, M.D. is the director of Division of Neurology Products for FDA Center for Drug Evaluation and Research.  You may also send a letter concerning this to: 10903 New Hampshire Avenue, Silver Spring, MD 20993.  The trial is- Phase 2 Randomized Blinded Safety Trial of Deferiprone in NBIA/PKAN, RFA-FD-11-001 (IND# 104880)

This trial will benefit the Kulsrud family (Jay, Laura, Lane, Tanner and Ty).  In December of 2011 Lane – age 11, was diagnosed with PKAN.  PKAN is a rare genetic disease caused by a mutation of the PANK2 gene.  This leads to neurological degeneration accompanied by a build-up of excess iron on the brain.  Lane is already experiencing side effects as a result of the build-up of iron on his brain.  He has developed slurred speech, loss of balance, frequent falls, loss of reflexes, and dystonia. In March of 2012, the geneticist confirmed that Tanner – age 9 and Ty – age 6, also have PKAN.  Tanner is beginning to show signs of slurred speech.  Ty appears symptom free, for now.  They are hopeful that all three boys will be accepted into this trial.  Deferiprone is their only hope of slowing or stopping the progression of the neurological degeneration caused by PKAN.  

Copy and paste this to his email message:

I am asking you to consider intervention in trial, Phase 2 Randomized Blinded Safety Trial of Deferiprone in NBIA/PKAN, RFA-FD-11-001 (IND# 104880) to eliminate the requirement that 1/3 of the participants receive a placebo.   I understand that there may be trials for other diseases where a placebo is necessary to decipher the results of the trial.  However, currently there is no treatment for PKAN.  In many cases PKAN can take a child from normal to immobile within a few years.  It will be very obvious if the drug is working.  If symptoms subside or quit progressing, they will know that Deferiprone is working.  There is a 1 in a million chance that a child is born with PKAN and none of the children can afford to be on a placebo for 18 months – the potential for progression of the disease and increase in irreparable symptoms is too great.  The FDA has already approved Deferiprone for Thalassemia and has proven that the drug is not harmful in the chelation of iron.  The FDA has also approved Deferiprone in two compassion cases of PKAN, in which positive results were shown. There are no other options for treatment of this disease.  The greatest possibility in fighting the effects of PKAN is getting Deferiprone as soon as possible.  Please, do everything in your power to allow all participants in the Phase 2 Randomized Blinded Safety Trial of Deferiprone in NBIA/PKAN to receive Dereriprone and not a placebo.

Also consider contacting your Senators and Congressmen.  

http://www.senate.gov/pagelayout/senators/f_two_sections_with_teasers/states.htm

http://www.house.gov/representatives/