March 29, 2022
For the Riley family, life would never be the same after March 2020. That’s when their 1-year-old daughter Olivia was diagnosed with an extremely rare and fatal brain disease. Then, just three months later, Olivia’s younger sister, Keira, received the same diagnosis. Thankfully, a treatment was available for Keira — but the family would have to raise hundreds of thousands of dollars and travel to Italy to access it.
Now the family is advocating for the Goldwater Institute’s Right to Try for Individualized Treatments (Right to Try 2.0), a law that would ensure that families can access potentially lifesaving treatments in the United States. You can learn about their story in this new video from the Goldwater Institute.
It was an unthinkable situation for the Rileys. Their daughters were both stricken with Metachromatic Leukodystrophy (MLD), a highly progressive and rare genetic brain disease that robs patients of their ability to walk and talk, and eventually their life.
Tragically, following her diagnosis, Olivia’s disease progressed quickly. “Within 90 days of symptom onset, our daughter Olivia lost the ability to walk, she lost the ability to talk,” Kendra Riley says in the video. (Olivia, now 3, is currently in hospice care, unlikely to survive her childhood.) But Keira, a newborn at the time of her diagnosis, had not yet experienced any symptoms. That meant that while there were no additional treatment options available for Olivia, there was one open to Keira: personalized gene therapy, which could modify her DNA so her body could function as it was meant to.
However, since this specialized treatment had not been approved by the U.S. Food and Drug Administration, Keira couldn’t access it in the United States. Instead, her family had to raise hundreds of thousands of dollars to move to Italy amid a global pandemic to get Keira the care she needed. “It is unconscionable that an American patient has to travel to another country, to Europe, in order to be able to get access to a treatment that could save their lives,” Goldwater Executive Vice President Christina Sandefur explains in the video.
Thanks to the cutting-edge, personalized treatment she received, 2-year-old Keira is developing normally today. “She’s doing things that we never got to see Livvy do,” Kendra said. “She’s running, she’s climbing, she’s having full conversations with us. She’s enrolled in preschool for the fall. 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.”
The Riley family’s story of heartbreak and hope demonstrates the urgent need for Goldwater’s Right to Try for Individualized Treatments reform, which would offer other patients with serious illnesses a chance at hope. This reform is the natural next step following Goldwater’s work to make Right to Try the law of the land. In 2018, Goldwater passed a federal Right to Try law that protects terminally ill patients’ right to access treatments that could save their lives but aren’t yet approved by the federal government. While this reform has empowered countless patients to access potentially life-saving care, it wasn’t an option for the Rileys. “The Riley family wasn’t able to use the original Right to Try because they were seeking a treatment that was individualized for their daughter,” Sandefur says.
Now, the Riley family is urging lawmakers in their home state of Arizona to pass Goldwater’s Right to Try for Individualized Treatments reform, which builds on the original Right to Try law and protects patients’ right to try to save their own lives by seeking treatments tailor-made for them that are not yet FDA-approved. This common-sense measure accounts for new innovations in medicine and helps get those innovations to the patients who need them the most. The reform passed the Arizona Senate and the House Health and Human Services Committee with bipartisan support, and it will soon be on the House floor for a final vote.
“Now it’s time for Right to Try 2.0, which will give hope to a new generation of families—families for whom the original, traditional treatments just won’t work, families who are out of options and need the best, most cutting-edge medicines out there,” Sandefur says. “That’s why we need Right to Try 2.0.”
You can watch the full video above. For more information on Right to Try 2.0, click here.