March 15, 2022

By Joe Setyon

After finding out their infant daughter had a fatal illness, Keira Riley’s family learned there was only one option available if they wanted to save her life: personalized gene therapy. Now, the Riley family is advocating for the Goldwater Institute’s Right to Try for Individualized Treatments reform so that other patients with serious illnesses have a chance at hope.  

The Rileys’ lives were forever changed in March 2020 when their 1-year-old daughter Olivia was diagnosed 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. Three months later, Olivia’s younger sister Keira was diagnosed with the same disease.

“There were no options available” for Olivia, who is now in hospice care, the sisters’ mom, Kendra Riley, told lawmakers on the Arizona House Health and Human Services Committee this week.

But Keira had a chance at survival. “Thankfully, there was one option available, and that was gene therapy treatment that was only available in Milan, Italy,” Kendra said Tuesday. Facing Food and Drug Administration (FDA) red tape in the U.S., as well as a global pandemic, the Riley family had just one month to raise hundreds of thousands of dollars so they could uproot their lives and move to Italy for five months to get Keira the treatment she needed.

Keira was ultimately able to access the life-saving care, and the now-2-year-old is doing much better today. But her mother knows this is the exception rather than the rule. “We were lucky that things happened and fell into place for us the way they did, because so many families don’t have that option,” she told lawmakers.

The problem, as Goldwater Director of Healthcare Policy Naomi Lopez testified Tuesday, is an outdated clinical trial system with federal regulations that aren’t keeping pace with modern medical advances. “Many of the medical innovations being pioneered today have made it possible to take an individual’s genetic information and create a treatment for that individual person,” Lopez said. However, “individualized treatment is subject to the same clinical trial process as single treatments that are intended for hundreds or even thousands of patients,” she added.

But there’s solution: Goldwater’s Right to Try for Individualized Treatments reform.
Sponsored by Arizona state Sen. Nancy Barto, Senate Bill 1163 protects patients’ right to try to save their own lives by seeking treatments tailor-made for them that have not yet been approved by the FDA. This common-sense reform—which passed the Arizona Senate and the House Health and Human Services Committee with bipartisan support, and which will soon be on the House floor for a final vote—accounts for new innovations in medicine, and helps get those innovations to the patients who need them the most, Lopez said.

“The fact that there was a chance, and there was one little slice of hope that we had for Keira, was amazing,” Kendra told lawmakers. “That’s the impact that this could have on so many families, and on so many—not just children, but adults facing these very rare illnesses. They deserve that chance at life, that chance at hope.”

Now, Arizona lawmakers have an opportunity to continue to lead the nation in empowering patients with serious illnesses to access potentially life-saving treatments. As Kendra put it: “It doesn’t make sense to not give someone [a] chance when the science is there.”

The science is there. Now it’s up to lawmakers to unleash the potential of today’s medical innovation to save lives.

Joe Setyon is a Digital Communications Associate at the Goldwater Institute.

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