Faces of Gene Therapy: Meet Kendra Riley

Kendra Riley is a public relations professional, mother, and a strong advocate for gene therapies. In 2020, two of her daughters were diagnosed with an extremely rare genetic disease – Metachromatic Leukodystrophy (MLD) – which damages the central nervous system causing cognitive decline over time. Kendra leveraged her PR expertise and community support to secure a life-saving gene therapy for her youngest daughter who still had time. She has since dedicated herself to advancing awareness, access, and policy changes in the gene therapy space, sharing her family’s story on national stages and working tirelessly to ensure no family face a similar devastating reality.

Kendra spoke with us recently about her journey.

Q: How did you come into the advocacy space?

In 2020, two of our three daughters were diagnosed with MLD – we had no choice but to be advocates for them and find something, anything, that could save their lives. Unfortunately, for our daughter who was symptomatic, there was no option aside from a clinical trial (which ultimately caused more problems) and she is now in hospice at age 7. For our other daughter, who was a newborn at the time and not symptomatic, there was a gene therapy treatment that could potentially save her life. We had one month to raise $500,000 and move to Italy for five months during the pandemic to get her this treatment before it was too late. And we did just that. In part because I own a PR agency and my colleagues helped to share our story in local and national news and in part because of friends and family who rallied in our support. Keira was lucky enough to become the 32nd child in the world to receive this treatment and she is now a normal, symptom-free 5-year-old. Since then, I have made it my personal mission to advocate for families like ours and ensure no other family has to lose one child in order to save another.

I have shared our family's story at global conferences, helped multiple families across the nation get the same life-saving treatment as our daughter, testified in front of the FDA to help support approval for the gene therapy treatment in the U.S., spoke at a Congressional Briefing in Washington D.C. in support of newborn screening for MLD, testified with the ACHDNC in support of newborn screening for MLD, and helped pass Right to Try 2.0 in Arizona (and 11 other states) to ensure patients with rare diseases can access innovative treatments that are potentially life-saving right in their home states.

Q: Why is gene therapy so important for your community specifically?

Every day I see the incredible impact of gene therapy, and the devastating results without it, side by side. I have one daughter who can no longer walk, talk or even hold her head up and is taking several daily medications to stay pain free while her sister who received gene therapy is going to school, running, jumping, singing and shows no signs of this horrible disease. Now that there is an FDA-approved gene therapy for MLD, we must screen for this disease and ensure no other family faces this same fate.

Q: What are some of the major barriers to access you see and how can we address them?

I never knew gene therapy was an option until I spoke with advocacy groups for MLD. Our doctors also had no clue it was available. How will patients and families know an option exists unless we are educating doctors, pediatricians, neurologists and the entire rare disease community? There must be a better process. It shouldn't just come down to parents desperately looking under every rock. Genetic testing should also be highly encouraged for parents-to-be. This could potentially save millions in medical bills and insurance claims for children who will ultimately become symptomatic and be in hospice at age 7, like our daughter.

Q: Why do you believe IGT’s mission of modernizing the policy space for gene therapies is so important? 

Because science is moving MUCH faster than the healthcare system and currently gets buried in government red tape.

There shouldn't be this many hurdles to access life-saving treatments.

Kendra’s story demonstrates the critical need to expand access to gene therapy and remove barriers that keep families from life-saving treatments. She is driving change to help ensure no family fights alone for a chance at hope.