Oliver Hurst owes his life to a luckily timed move across the country. His father and mother, who was then three months pregnant with Oliver, arrived in Vilonia from California in March 2020.
At the time, UAMS’ Kapil Arya, M.D., was developing strategies as a UAMS Translational Research Institute Implementation Science Scholar to establish statewide newborn screenings for spinal muscular atrophy (SMA), a rare disease that can disable a newborn within months.
The only effective treatment for SMA is a single-dose $2.1 million gene editing therapy – the most expensive one-time treatment in the world, Arya said. It must be provided within a few weeks of birth to counter the most severe form of SMA, which can be fatal in the first few years of life. Less severe forms can cause a lifetime of disability.
Arya, an associate professor in the College of Medicine Department of Pediatrics, Division of Neurology, is part of the 2020 Implementation Science Scholar class, which is conducted by the UAMS Center for Implementation Research. He saw the program as an opportunity to learn skills that would help him implement the statewide SMA screening.
“Implementation science is the science of teaching you how to do a particular thing in the best way possible,” said Arya, who treats patients at Arkansas Children’s Hospital. “I knew the goals we wanted to achieve with the newborn screening, and implementation science taught me how to achieve those goals.”
The Arkansas SMA screening law was enacted in 2019, but a complex system was still necessary to make the screening routine across the state. Arya helped facilitate the purchase and validation of sophisticated testing equipment, developed training programs and created materials to reach all corners of the state. He also worked with insurers to secure coverage of the treatment cost, and persuaded them to accelerate their approval timeline by several months.
By the time Oliver was born at Conway Regional Medical Center in August, the screenings were being conducted statewide.
The scholars program may have given him the skills to implement the screening, but Arya’s tireless commitment also played a role, said Sara Landes, Ph.D., his primary mentor as a core faculty member at the Center for Implementation Research.
“To go from legislation to full implementation so fast sounds made-up – it just doesn’t usually work that way,” said Landes, an associate professor in the College of Medicine Department of Psychiatry. “He has engaged with stakeholders at all levels – patients, providers, health systems, payers and policymakers. It’s really impressive.”
With about 1 in 10,000 newborns diagnosed with SMA, Arkansas will have an estimated three to four cases each year.
Oliver happened to be one of them in 2020. His mother, Elaine Hurst, said she and her husband Darrell were “wrecked” by the news but hopeful the proposed treatment would work.
Oliver received the single dose infusion of Zolgensma, which uses a common virus to carry the gene-editing tools that should cure him of SMA.
So far so good, Hurst said, and she is thankful for her family’s good fortune. California, they learned later, did not have the SMA screening when Oliver was born.
“Moving to Arkansas saved Ollie’s life,” she said.
