Five-year-old Olivia Riley of Arizona suffers from an extremely rare and fatal genetic brain disorder called metachromatic leukodystrophy (MLD). She is tragically in hospice care, unlikely to survive her childhood.
That her younger sister Keira, who has the very same disease, is a thriving 4-year-old is a testament to the power of an innovative gene therapy that saved Keira’s life as an infant, before she started showing symptoms.
Had the Riley family waited for the Food and Drug Administration (FDA) to approve that personalized treatment, Lenmeldy (OTL-200), which finally happened last week, it would have been too late for Keira. The Rileys had to move to Italy in 2020 so that Keira could get the care she needed.
Thanks to that treatment, Keira gets to live a normal childhood. But the Riley family’s story of hope and heartbreak highlights a troubling reality: The U.S. is falling behind the world when it comes to individualized medicine. It doesn’t have to be this way.
Instead of learning to walk, run, talk, play and sing, young children with MLD (the disease is most common among infants and toddlers) fade away physically and mentally until death, which usually occurs within five years. But Lenmeldy provides an almost miraculous ability to slow or halt the disease progression, like it did with Keira.
By 2020, Lenmeldy had received approval in the European Union, the U.K., Iceland, Lichtenstein and Norway. 2020 is the same year the Riley family learned that Olivia and Keira had MLD. First, 1-year-old Olivia was diagnosed with MLD in March 2020. Within 90 days of symptom onset, she lost the ability to walk and talk.
Her baby sister, Keira, was diagnosed three months later. Since she was pre-symptomatic, Lenmeldy could help. But despite its history of success in treating patients with MLD, outdated federal regulations meant it was not available in the U.S. Because of the disease’s rapid progression, the Riley family almost immediately raised the vast resources to relocate to Italy —during the height of the pandemic — in order to try to save Keira.
It worked. According to Keira’s mother, Kendra, “She’s doing things that we never got to see Livvy do. She’s running, she’s climbing, she’s having full conversations with us … These are really great, everyday things for normal parents, but for us, they’re huge, huge milestones that we didn’t get to see with Livvy. So it’s really, really heartwarming to see that happen for Keira. And it’s all because of this treatment.”
Americans desperate for hope shouldn’t have had to move across the globe for a chance at a life-saving treatment. The plight of the Riley family inspired the Goldwater Institute, where I am a senior fellow, to craft the Right to Try for Individualized Treatments, often referred to as Right to Try 2.0.
The original Right to Try protected terminally ill patients’ right to access investigational treatments slowing moving through the FDA approval process, and it’s already saving lives as pharmaceutical companies use Right to Try to treat entire groups of patients in promising new ways. But today, the latest innovations in medicine are made specifically for each patient, based on their genetics, and by definition cannot go through the FDA’s outdated regulatory processes in a timely manner.
About 30 million Americans suffer from rare diseases for which there are no treatments or cures, and half of those are children. Right to Try 2.0 allows those desperate patients to access the latest innovative personalized treatments without begging the government for permission. It brings the hope of 21st-century medicine to patients who need it the most. Already adopted in Arizona and Nevada, this legislation is currently being considered in states like California, Maryland and Mississippi.
It’s not enough to marvel and celebrate the scientific breakthroughs that treatments like Lenmeldy bring. We must also ensure that Americans have access to innovative, potentially lifesaving treatments that are being pioneered in other countries and might otherwise be pioneered here. It’s time for U.S. policymakers to put patients first, ensuring that the right patient may access the right treatment at the right time.
Naomi Lopez is a senior fellow at the Goldwater Institute.