Kendra Riley has been practicing public relations here in the Valley for over 20 years and has owned her agency, Dawning Public Relations, for 10 years. She’s a founding partner for Scottsdale.com, and in her spare time is a fierce advocate within the rare disease community, for which she was just recently featured in the Wall Street Journal, CNN, NewsNation, USA Today and The Epoch Times.
In addition to being a board member of Armer Foundation for Kids and on the committee for the Phoenix Heart Ball benefiting the American Heart Association, it was her daughters who inspired her advocacy within the rare disease space after two of the three were diagnosed with an extremely rare disease called Metachromatic Leukodystrophy (MLD) in 2020. These devastating diagnoses caused their family to move to Italy during the pandemic so their youngest daughter could become the 32nd child in the world to receive a life-saving gene therapy treatment.
First, let’s talk about your sweet children. Can you tell us a little about them?
Dave and I have three beautiful girls, Eva (8), Olivia or Livvy as we call her (who is 5), and Keira (4).
In 2020, at the height of the global pandemic, Livvy was diagnosed with MLD. What is MLD?
MLD is short for Metachromatic Leukodystrophy. It is a dreadful disease that causes the body to lack the creation of an enzyme that is needed to breakdown sulfatides in the brain, the build up of which becomes toxic to the nerves and causes a progessive loss of all development. Within 90 days of diagnosis Livvy lost the ability to walk, then talk, and then eat on her own. She is now entirely reliant on us for everything, is tube fed, and takes 10 medications a day to stay pain-free.
Keira was diagnosed with the same condition, as well …
After learning this disease was genetic, we had to get our other daughters tested and in June 2020 the results came back that Keira (who was only 6 months old at the time) also had the two gene mutations that caused MLD. We assumed we would have to put her in the same clinical trial Livvy was in to stall the disease (as there is no cure for symptomatic children) but we were thankfully speaking with CureMLD.com Founder Maria Kefalas who informed us of a curative gene therapy treatment in Italy that was working wonders on non-symptomatic babies.
You traveled all the way to Italy for help …
Once we learned of the treatment in Italy, Cure MLD connected us with the doctors in Milan who then reviewed all of Keira’s diagnostic paperwork and confirmed she was a candidate. However, the clinical trial for this treatment had ended. So they were able to offer us the drug on a compassionate use basis but every other part of the treatment at the hospital (in addition to the travel, housing, food, etc.) would have to be paid for out of pocket. It would also require us to live in Italy for five months, as we could not leave the country due to the pandemic.
In mid-July they confirmed we were a “go,” with the caveat that we needed to get her there in one month’s time to receive the treatment and ensure its efficacy. And we needed $500,000 to make it a reality. So the fundraising and PR campaign began, and on Aug. 15, 2020 we boarded the plane to Italy.
Since your children’s diagnoses, you’ve become a crusader. This past month, there was a treatment approved by the FDA to treat MLD.
This was a monumental day for the MLD community. It means the gene therapy treatment that Keira received in Italy—which has allowed her to live a completely normal life, symptom-free of MLD—is now FDA-approved for non-symptomatic MLD patients to receive the treatment here in the U.S.
You were instrumental in this happening and have done so much since returning from Italy to help other families like yours. Tell us more.
After returning from Italy, I knew I wanted to do whatever I could to help other families going through similar situations. I immediately joined the board of Armer Foundation for Kids, who helped us fundraise, and helps relieve the financial burden for families whose children are facing life-threatening diagnoses; myself and many other MLD families testified with the FDA to share our family’s stories and explain how life-changing this treatment is not just for the patient but the entire family; I’ve shared our family’s story on national and international stages; and the Goldwater Institute, along with Senator Nancy Barto (who we reached out to prior to going to Italy in regards to Right to Try and if that would allow us to get Keira the treatment here at home), informed us that because the original Right to Try law did not allow for individualized treatments, like the gene therapy Keira received in Italy, our story inspired them to update the law to include just that. So, I testified at the Arizona state capitol in support of this reform, which passed in 2022, and has since passed in many other states throughout the nation.
How will this gene therapy treatment revolutionize care for this disease?
It will completely change the lives of babies diagnosed before symptom onset. Instead of being in hospice by age 5 (like our daughter Livvy), children will be able to live a completely normal life; going to school, running, talking, playing sports. Normal things to the everyday family but true miracles for families like ours who have seen what this disease does to a child (and a family).
What’s next for help?
The primary way this disease is even diagnosed is for a child to become symptomatic, and by then it’s too late for treatment, but any younger siblings could be spared. So the next step to ensure no family will have to lose one child to MLD in order to save another is to get MLD added to the RUSP (Recommended Uniform Screening Panel, which is a list of disorders that the Secretary of the Department of Health and Human Services (HHS) recommends for states to screen as part of their state newborn screening (NBS) programs.)—also more widely known to parents as the foot prick test each baby gets as a newborn.
I just testified in support of this happening last fall and am very hopeful that it will soon be added now that there is this FDA approved treatment that is providing so much hope for families and a chance at a normal life for kids like Keira.
Can you and your husband, Dave, share a few words about each of your children?
Eva’s our oldest. She’s 8 years old, loves to swim, play guitar, golf, and is super into Pokemon right now. Livvy is about to turn 6 and, like her sisters, is a major Swiftie. She loves being outside, swinging on her Make-A-Wish swing set, and having us read books to her. Keira is 4 years old and is our girly girl; she loves to wear dresses, put on makeup, do gymnastics, and sing along to Taylor Swift.
When you’re not with your children, husband, working, or crusading for this, we’d find you …
Enjoying the occasional spa day with girlfriends at Joya Spa, meeting with fellow business owners in the Valley at weekly breakfast meetings for the Executives’ Association of Greater Phoenix, and going for walks around the neighborhood.
How can people help your family or the fight to diagnose and cure MLD?
By making a donation to CureMLD.com or the MLD Foundation.