By Michelle Talsma Everson -
Saturday Jun 27, 2020
$500,000 and five months in Italy. That’s the Riley family’s only hope for saving their 5-month-old daughter Keira, who along with her 2-year-old sister Olivia, suffers from a form of Metachromatic Leukodystrophy (MLD) so rare, they don’t even search for the mutation in today’s genetic testing.
MLD is an extremely rare, fatal brain disease that destroys the protective fatty layer (myelin sheath) surrounding the nerves in the central nervous system. It aggressively takes away motor function and other abilities and when symptoms show early in life, most children are not expected to live past 4 to 6 years old.
Olivia’s siblings were immediately tested while Olivia began the only ongoing clinical trial for MLD patients. The treatment is not curative and cannot reverse damage already done, but seeks to slow the rapid progression of the disease while protecting and preserving quality of life.
“Olivia has lost her vocabulary, cannot pronounce most words and experiences some form of pain from moderate to severe on a daily basis,” said Kendra. “She still has lots of smiles and her big sister Eva and baby sister Keira always make her light up.”
This summer, the Rileys learned Keira also has both mutated copies of the gene and has been diagnosed with MLD.
The Rileys turned to CureMLD.com and The MLD Foundation, and learned of a cutting-edge MLD gene therapy treatment only available in Milan, Italy, and it may not be covered by health insurance. Since beginning the treatment in 2010, the Institute in Italy has treated 29 patients from around the world, 26 of which are still alive.
Currently asymptomatic, Keira is eligible for the therapy but not as part of the clinical trial, which means the Riley family will have to pay for her treatment out of pocket if it is not approved by insurance. The investment for treatment, lodging, travel and living expenses during treatment is estimated at $500,000.
“We only have one hope left. $500,000 sounds impossible. It’s overwhelming, but if 100,000 people who hear our story are compelled to donate just $5 each – we’ll have the opportunity to save our daughter Keira’s life,” Kendra said.