Time is running out for brothers Lane, Tanner and Ty Kulsrud.
All three boys, who range in age from 13 to 9, have a rare metabolic disorder that is progressively damaging their brains and robbing them of physical abilities.
Untreated, their disease is a death sentence. Their best hope is an experimental drug – a treatment so far barred by the Food and Drug Administration because it has not entered clinical trials.
They suffer from PKAN, a neurodegenerative disorder that afflicts an estimated 1,000 in the United States and 6,000 around the world.
The disease is caused by the body’s inability to metabolize vitamin B5, resulting in debilitating iron deposits in the brain.
The Kulsrud brothers’ plight is the impetus behind a so-called “Right to Try” bill before the North Dakota Legislature.
Supporters hope Senate Bill 2259, which passed the Senate and had its first House committee hearing Tuesday, can help unlock the door to the experimental drug the Jay and Laura Kulsrud family is desperate to try for their sons.
The Kulsruds are from Grace City, about 65 miles north of Jamestown.
So far, five states have passed “Right to Try” laws and North Dakota is among 20 states considering measures to help desperate patients obtain access to treatments undergoing FDA review.
If enough states pass “Right to Try” laws, supporters believe the FDA could relent and allow easier access to experimental therapies for patients with terminal diseases and no proven treatments.
“We have nothing else to try, and we’ve tried everything else,” Laura Kulsrud said in an interview after she gave testimony before the House Human Services Committee supporting the bill.
Lane, who will turn 14 in May, accompanied his mother but did testify because he has difficulty speaking, especially when he is excited, as he tends to be when speaking of his situation, Laura Kulsrud said.
A few years ago, he played basketball and bolted around like any other energetic boy. Then symptoms appeared around age 10, a pattern repeated by his brothers. Now Lane walks with a cane and assistance.
“He is so frustrated that they will not do anything for him when there is nothing else,” Laura Kulsrud said.
Despite pleas from members of the North Dakota congressional delegation, the FDA has refused to grant a “compassionate use” exemption to enable the Kulsrud brothers to take an experimental drug that has shown promise in animal studies and in European trials.
The bill, introduced at the Kulsrud family’s request by Sen. Tim Mathern, D-Fargo, applies to patients with a terminal illness who have exhausted all other treatment options.
The patient’s treating physician must recommend the treatment, which must have passed the first phase of clinical trials, which assesses safety.
Insurers are not obligated to pay for the experimental treatments; pharmaceutical companies often provide access to experimental drugs in clinical trials at no cost.
“Facing the death of a family member and knowing something might help but ‘that something’ is just out of reach is a painful place to be,” Mathern said in remarks to the House Human Service Committee.
“I trust we can help this family and others to take a well-supervised and contained risk when no other options are available,” Mathern said.
The Kulsrud family’s struggles began in 2011 when their oldest son, Lane, began showing symptoms, including slurred speech and balance problems causing him to stumble and fall often.
Tanner, the middle son, last year started exhibiting similar symptoms. Ty, the youngest, so far hasn’t shown signs of PKAN but has been diagnosed with the disease.
Brain scans reveal that Ty has iron deposits in his brain, similar to those of his older brothers, a characteristic pattern called the “Eye of the Tiger.”
Death typically comes to PKAN sufferers within 10 or 12 years of the onset of symptoms.
The family’s hope rests with a new drug called RE-204, a derivative of vitamin B5 that is made by Retrophin, a U.S. pharmaceutical company.
Because none of the Kulsrud brothers are on the “edge of death,” they have been denied access to the drug by the FDA.
Only about 1,000 people a year surmount the FDA’s cumbersome and expensive “compassionate use” application process, which requires the treating doctor to complete paperwork estimated to take 100 hours to complete, according to Craig Handzlik, state policy coordinator for the Goldwater Institute, which supports the bill.
“ ‘Right to Try’ would essentially give them another arrow in the quiver in their battle,” Handzlik said of the bill.
In urging support of the bill, which overcame an early defeat to pass 32-15 in the Senate, Sen. Terry Wanzek, R-Jamestown, one of the bill’s sponsors, said, “Let’s send a message to the FDA on how we feel. … I can’t understand why they wouldn’t help a family that is as desperate as this one is.”
The Kulsrud brothers take a form of B5 in the hope of slowing the progression of PKAN – and wait for the day they can try the experimental drug.
“I don’t know if it’s going to help in this case,” Laura Kulsrud said of the North Dakota “Right to Try” bill. “It can’t hurt. There’s so many people out there with cancer. I’m not just fighting for my kids.”