March 17, 2022
By Kendra Riley
In March of 2020, my family got the news that would forever change all of our lives: My daughter Olivia, who was almost 2 years old, was first diagnosed with a highly progressive and rare genetic brain disease. Her sister, Keira, would be diagnosed with the same disease three months later. Incredibly, though a treatment was available, we would have to travel halfway around the world to access it, all because of outdated rules and red tape in the United States.
We had never heard of the disease—Metachromatic Leukodystrophy (MLD)—and neither have most doctors. But what we did know was that something was very wrong as Olivia, a previously thriving baby, was losing her ability to walk and talk.
Keira had not yet shown any symptoms, and there was a very short window where a promising gene therapy could dramatically halt or slow the progression of this unimaginably cruel disease. The treatment couldn’t help Olivia because she was already symptomatic, but we did have a chance to help Keira.
We embarked on a desperate race against time to raise hundreds of thousands of dollars to relocate our family to Italy for five months—amid a global pandemic no less—so our sweet Keira could obtain a gene therapy treatment that doesn’t fit within the Food and Drug Administration’s (FDA) outdated clinical trial system. While it has recently been approved in the European Union, families shouldn’t have to travel overseas to save their children’s lives. Nor should they be sent home to watch their children die, even though promising treatment options exist. That’s why we need the Right to Try for Individualized Treatments—a new law that protects patient’s right to access potentially lifesaving treatments.
Had this reform been in place when Keira needed it (and if the doctors and technology had been available in the U.S., and if the drug company was offering its product on a compassionate-use basis—as it is now), we could have had her treatment completed in the U.S. without having to fundraise hundreds of thousands of dollars and move our family halfway around the world for five months.
The proposed legislation, Senate Bill 1163, adheres to the strictest patient protections and physician involvement, ensuring that these treatments work in tandem with the highest standards of care.
Should this pass in Arizona, as the original Right to Try bill did, other states will likely follow suit, and then the federal government to follow.
It brings tears to my eyes thinking of all the other special needs families out there who have always held on to hope for a chance like this. A chance at healing. A chance at a normal life—something every single one of us deserves.
Kendra Riley is a mother in Arizona whose family has firsthand experience with cutting-edge, individualized medical treatments. She’s urging Arizona lawmakers to pass Goldwater’s Right to Try for Individualized Treatments reform, SB 1163, which is sponsored by Sen. Nancy Barto. You can read more about the Riley family’s story at Kendra’s blog, The Riley’s Road to Italy.