CHAPEL HILL — Meagan Hendren wouldn’t be directly affected by the TREAT Act, but she knows what it’s like to have children with a rare disease and few treatment options.
Her twin sons, Lucas and Logan, are 13 months old and may only live about 11 more. They have infantile Gaucher disease, a metabolic disorder caused by an enzyme deficiency that results in an enlarged spleen, liver, lungs and brain and also disrupts messages the brain sends to the body. There is no cure.
The twins had tracheotomies this week, where a surgeon cut a hole in each of their necks to open an airway; now they breathe with the help of a tube.
The procedure was one of the hardest so far, Hendren said tearfully Tuesday morning as she joined Sen. Kay Hagan in promoting the Transforming the Regulatory Environment to Accelerate Access to Treatments, or TREAT Act, at the N.C. Children’s Hospital in Chapel Hill.
The TREAT Act would speed Food and Drug Administration approval for drugs used to treat people with serious or life-threatening diseases, including rare diseases. Such illnesses are often called “orphan” diseases because they have no drugs made for them; two few people are diagnosed to make it profitable for drug companies to invest in possible treatments.
“For research to be turned into effective treatments, it’s going to expedite development and access,” Hagan said. “We’ve got to be sure all the other orphan diseases we have, have a way to get to the market.”
About 35 patient advocacy groups have endorsed the bill, including the Veterans Health Council, Muscular Dystrophy Association, and National Caregivers Association.
Before a company can sell a drug in the United States, the FDA must test and approve it after clinical trials with patients. The FDA has an accelerated approval process now, but it is complex and hard to navigate, which discourages drug companies from investing in medications for diseases that few people have, said Ken Moch, president and CEO of Chimerix, Inc. a drug development company in Durham.
“There is currently a widespread lack of clarity about which clinical trials can get accelerated approval,” he said. “The time to take a (new) medicine to a patient in need is more than 10 to 15 years. ... The development path is fraught with complexities.”
Hagan’s bill “hopefully will help us in five years, in 10 years and certainly in 20 years not have to say that anymore,” said Dr. Wesley Burks, physician in chief at the N.C. Children’s Hospital.
“There are 30 million Americans living with rare diseases,” he said. “We offer diagnostic evaluations, genetic counseling and ongoing care for the children. Sometimes, though, it’s difficult when we just have to say we don’t really have a treatment.”
The bill has widespread bipartisan support in the U.S. Senate, and there is an accompanying House bill, Hagan said. The legislation should make it to the president’s desk later this year, she said.
Any bill will likely be too late to help the Hendren twins, but Meagan Hendren and her husband, Jarrod, hope the legislation will prevent other parents from watching their child die from a disorder that can’t be treated.
The family has been at the hospital since March. They hope to go home soon and make some memories with the time they have left, she said.
“I never knew there was something out there worse than cancer,” she said. “You just watch your child fade away.”