The thought of watching your child fight a rare, potentially fatal disease is a terrible one; but what would you do if all three of your kids were suffering -- and there was next to nothing you could do about it?

That horrifying situation is a reality for the Kulsrud family of Grace City, North Dakota.

All three of Jay and Laura Kulsrud's boys -- Lane, 13, Tanner (the middle son; age unspecified), and Ty, 9 -- have the rare neurodegenerative disorder PKAN.

The condition is characterized by the body's inability to metabolize vitamin B5, which results in life-threatening deposits of iron in the brain.

These iron deposits gradually wear away the brain's -- and therefore, the body's -- ability to function properly; left untreated, it typically claims the sufferer's life within 10 to 12 years after symptoms appear.

Enter the experimental drug RE-204 and Senate Bill 2259.

RE-204 is a derivative of B5, produced by U.S. pharmaceutical company Retrophin, that has not yet been approved by the FDA. Despite pleas by both the family and public officials, the administration has refused to grant the Kulsruds a "compassionate use" exemption to try RE-204.

SB2259, also known as the "Right to Try" bill, was introduced by Sen. Tim Mathern (D-Fargo) and aims to open up access to the drug for patients with no other treatment options.

The bill would allow terminal patients who had exhausted all other options to use experimental drugs for treatment, given an official recommendation by the treating physician, and that the drug had passed the first stage -- that which assess safety -- of FDA review.

Insurance providers would not be forced to pay for the treatments; however, access to experimental drugs in clinical trials is often provided free by the makers of the drug.

SB2259 has passed the Senate and had its first House committee hearing Tuesday (Mar. 10).

[SOURCE] [SOURCE] - Bismarck/Minot/Williston/Dickinson-KXNEWS,ND