How Right to Try 2.0 Can Help Patients

“It is July 6th, 2020 and my entire family is fighting like mad to save the life of our 5 month old baby girl, Keira. Who, unlike her sister Olivia (age 2), has a chance to receive a treatment that could potentially give her a normal life and beat the life-wrenching disease known as Metachromatic Leukodystrophy (MLD).

“Keira is eligible for a cutting edge gene therapy treatment only available in Milan, Italy. She would be one of only 30 children in the world to receive this treatment. While it is not a cure, it could give her a chance at a normal life.

“Not only would it require us to move to Italy for 5 months while Keira completes the treatment, but we would also have to transfer Olivia’s clinical trial treatments to a European site. And the cost to us, for treatment, lodging and travel? Hundreds of thousands of dollars.

“The gene therapy treatment Keira is eligible for is not yet considered a cure – it has only been around for a decade – but it is at least a chance at a normal life.” – Kendra Riley

Gene therapy is a cutting-edge treatment that doesn’t fit within the FDA’s outdated clinical trial system, so it’s out of reach for children like Keira. Families shouldn’t have to travel overseas to save their children’s lives. That’s why we need the Right to Try for Individualized Treatments. Read more about the Riley family’s story at rileysroad.com

When Jordan McLinn was three years old, he was diagnosed with a fatal form of muscular dystrophy called Duchenne. His family left the children’s hospital in their hometown of Indianapolis with no options, but then they heard about a new experimental drug that could help Jordan. Unfortunately, Jordan didn’t qualify for the clinical trial that would allow him to use the drug, and his family was left searching for hope.

The FDA tightly regulates clinical trials, and for good reason. Scientific rigor and testing standards are needed to ensure patient safety and provable outcomes. But when a patient is without hope—and when a drug meets safety standards—they should have a right to try potentially life-saving medications.

Fortunately, Jordan was able to participate in a clinical trial, and almost 10 years later, he’s doing great and isn’t showing any signs of decline. He became an advocate for Right to Try so that other children like him who are not so lucky—who can’t get into clinical trials—have a right to try to save their own lives.

There are surely patients like Jordan who are fighting cancer, who are out of options, and who are not able to access new, potentially life-saving individualized treatments because of inhumane and outdated FDA rules.

Matt was a U.S. Navy pilot with a young family, but a diagnosis of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) in 2014 took his life on a different path. Things that come effortlessly to most of us became a daily struggle for him. Before long, Matt was out of treatment options, facing a terminal diagnosis with little hope.

Right to Try became a game changer for Matt and his family. Under Right to Try—pioneered by the Goldwater Institute—terminally ill patients would be able to access investigational treatments yet to receive final approval by the U.S. Food & Drug Administration (FDA).

By the time Right to Try became federal law, Matt didn’t know if he’d ever benefit: He was basically wheelchair-bound and unable to speak, and he needed to use a breathing machine to sleep. But in early 2019, Matt announced that he had begun an investigational treatment under the federal Right to Try law. Within weeks of beginning the treatment, he could pull himself up to standing from his wheelchair. He no longer needed the breathing machine to sleep, and he even saw improvement in his ability to talk.

Matt’s story is an example of how miracles are possible if laws open the door to medical innovation—including individualized treatments—while ensuring patients are safe and under a doctor’s care.