For Suffering Families, New Right-to-Try Legislation Offers a Glimmer of Hope
When Kendra Riley’s daughters were diagnosed with a rare genetic disease, doctors told her that a novel gene therapy, only available in Milan, Italy, represented their only chance for survival.
Riley’s daughters, Olivia and Keira, were diagnosed with metachromatic leukodystrophy months after they were born in 2020. Olivia’s condition began to deteriorate rapidly, leaving her unable to walk or talk.
Keira, however, remained asymptomatic, which made her eligible for a novel gene therapy that offered an “extraordinary opportunity to save her life.” But the therapy has not yet received FDA approval, so the grieving Riley family was forced to pack up and move away from home to save their daughter.
“Our community rallied around us to help raise the half-million dollars we needed to make the move,” Riley told National Review.
But now, thanks to new legislation that was just introduced in the House and Senate, there’s hope that families faced with similar tragic circumstances won’t be forced away from their homes
The mother of three has spent years advocating for novel gene therapies and right-to-try for individualized treatments laws after her family was forced to leave the U.S. to save her daughter.
An initial right-to-try bill passed in 2018, which allows certain patients to access treatments that have undergone the phase-one testing but have not yet received FDA approval. However, conditions such as Olivia and Keira’s, which require experimental treatments tailored to their DNA, still cannot be accessed in the U.S. without formal approval.
In Milan, the Rileys arrived at San Raffaele Telethon Institute for Gene Therapy and sought experimental care. Keira underwent stem cell treatments, chemotherapy, and stayed in a “sealing room” for weeks while her body healed.
“That was the hardest part of the process,” said Riley. Keira was just months old.
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When the Riley’s returned from Italy, they started raising awareness of rare genetic illnesses, and for more pathways to treat them locally. The Rileys began to share their story, and to connect with other families dealing with metachromatic leukodystrophy.
Their journey brought them into contact with Arizona state legislators and the Goldwater Institute, which has been an advocate for right-to-try laws and helped to pass the first federal right-to-try bill in 2018. Since then, almost twenty states have updated that legislation with a second right-to-try bill that allows patients to seek individualized treatments that are still in the experimental phase.
With the strong support for right-to-try at the state level, the Goldwater Institute is looking to expand nationwide with the introduction of the bill into both the House and the Senate in June.
“The law was created with the idea that [for individualized treatments] … you’re not going to be able to find the interest financially for taking something into trial,” said Naomi Lopez, a Senior Fellow at the Goldwater Institute who researches healthcare issues.
The new legislation could affect many more patients like the Riley sisters, who require care that is personalized to their DNA.
“There’s a much stronger growing demand, you know, with the influence of genetic testing, and gene editing, and gene therapies,” said Lopez.
Lopez reports that the current administration has been supportive of individualized treatments legislation, after Trump enthusiastically signed the initial right-to-try bill during his first term.
The Rileys, and many other families with rare diseases, are hopeful.
“This legislation is the lifeline that rare disease families have been desperately praying for,” said Kendra Riley.
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