Bill provides access to customized gene therapies, medicines

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(The Center Square) - A new congressional bill would give patients with life-threatening diseases access to customized gene therapies and medicines.

The Right to Try for Individualized Treatments Act, whose sponsors include U.S. House members from Arizona and Tennessee, would allow patients a pathway to access individualized investigational treatments when no other approved options are available. The bill defines “individualized investigational treatments” as “a drug or biological product for the patient based on an analysis of the patient’s unique genomic profile.” It would allow for medicines and gene therapies, both tailored specifically for the patient.

Treatment must be administered by an eligible healthcare facility “operating under federal assurance for protection of human subjects,” according to the bill.

Doctors or healthcare facilities are not required to offer these treatments, the bill noted.

The legislation has support from people such as a mother who has seen one daughter end up in hospice care and another daughter, who received special treatment in Italy, now thriving in the classroom. The young girls had the same disease. The mother spoke to The Center Square, and more about the family is reported later in this story.

U.S. Reps. Andy Biggs, R-Gilbert, Ariz., and Diana Harshbarger, R-Morristown, Tenn., are introducing the Right to Try for Individualized Treatments Act in the House. U.S. Sen. Ron Johnson, R-Wisconsin, is introducing the bill in the Senate.

“We are entering a new era of medicine where breakthroughs in genomics and precision therapies can create treatments designed specifically for an individual patient," said Harshbarger, a licensed pharmacist. "But our regulatory system was built for a different time and simply hasn't kept up.

“This legislation makes sure patients have a clear, durable path to pursue individualized treatments when all other options have failed,” she said.

Biggs noted Congress passed the first Right to Try bill in 2018, which allowed terminally ill people to obtain access to investigational drugs that were not completely approved by the U.S. Food and Drug Administration.

“Our coalition was unwilling to let one more American die without this chance, and we are motivated to build on this original bill with the Right to Try for Individualized Treatments Act,” Biggs said.

Johnson said the bill builds upon the success of the first Right to Try bill. He added that the bill is “about medical freedom and putting doctors and patients at the top of the treatment pyramid.”

Kendra Riley, an Arizona mother of three, told The Center Square this week that her family is “living proof of what happens when you have access to a treatment you need and what happens when you don’t.”

For patients dealing with rare diseases, “timing is everything,” according to Riley.

Kendra Riley’s daughters, Olivia Riley and Keira Riley, were both diagnosed with a rare and aggressive genetic disorder called metachromatic leukodystrophy (MLD) in 2020.

According to the Goldwater Institute, Olivia Riley started to show signs of MLD, such as losing her ability to walk and talk. Doctors told her family that Olivia Riley’s MLD would cause her to deteriorate and that she would need hospice care.

Keira Riley had not yet started showing symptoms even after being diagnosed, the Goldwater Institute said.

To get Keira Riley’s treatment, Kendra Riley told The Center Square that her family needed to raise $500,000 and move to Italy to get the life-saving treatment.

Keira Riley received the treatment in Italy, whereas her older sister was unable to.

Kendra Riley said Olivia Riley, who is 8 years old, is currently in hospice, and Keira Riley is a healthy 6-year-old with zero symptoms.

"Under the legislation, my family would not have needed to move to Italy to get treatment," Kendra Riley told The Center Square.

The Right to Try for Individualized Treatments Act would allow a patient to get a treatment in the U.S. if a doctor and facility are “willing and able to offer it,” she said.

The bill would give "rare-disease patients a chance before an illness becomes an irreversible loss,” Kendra Riley noted.

The mother of three said she thinks the FDA “is behind science and technology that’s moving at the speed of light.”

“No one should have to wait on government red tape to try and save their life,” she added.

The bill is an attempt to give “families a chance before a rare disease becomes an irreversible loss,” Kendra Riley said.

“We see right in front of us every day with our two girls what happens if you have it and what happens if you don’t,” she explained. “It’s the difference between a child in hospice and a child in the classroom.”