Three West Coast-based healthcare organizations are launching a Center for Personalized Medicine in the Intermountain West, designed to bring next-generation care to children by using advanced technologies to understand a child’s unique genetic makeup and offer tailored treatments.
Intermountain Primary Children’s Hospital, University of Utah Health, and Intermountain Precision Genomics are collaborating to develop the program, Primary Children's Center for Personalized Medicine, which officials say “is the first of its kind in the Intermountain West.”
They added, “The center is using cutting-edge genetic technologies and precision treatments to transform clinical care. With these advancements, targeted treatments that were not available a few years ago are helping children with some of the most challenging medical conditions to survive and thrive.”
The collaborative initiative is located at Primary Children’s Hospital and has received start-up funding from the Intermountain Foundation at Primary Children’s Hospital.
There are more than 7,000 known genetic diseases, many of which affect infants and children and can cause life-long disability. There are cures for only a handful of these diseases, and in some cases the diseases can even lead to early death, noted Josh Bonkowsky, M.D., Ph.D., University of Utah Health and Primary Children’s Hospital, who is medical director of the Primary Children’s Center for Personalized Medicine. As such, the goal of the center is to transform this critical need for treatments through research, discovery, and innovative therapies.
Access to a wealth of clinical, genetic, and familial data housed at Intermountain Healthcare and University of Utah Health gives the center a head start and a unique advantage, organization executives noted. By combining these resources with decades of research in Utah, “the center is poised to forge new connections between genetics and human disease and apply those discoveries to benefit children worldwide,” officials attested.
Broadly, the Primary Children’s Center for Personalized Medicine is focused on three core areas: precision diagnosis; gene therapies and novel therapeutics; and stem cell, immunologic and regenerative medicine.
Officials offer a real-world example of Evie Lewis of Ogden, Utah, who was born with a severe genetic disease, Type 1 spinal muscular atrophy (SMA), that is nearly always fatal before the age of 2. But a molecular treatment she receives at Primary Children’s Hospital and University of Utah Health appears to have halted progression of the disease for Evie, who is now two-and-a-half years old. However, a few years ago, before this breakthrough treatment existed, Evie’s older sister, Blakely, died of the same disease. “The work we are doing here and now is transforming pediatric medicine. We will not be done until we have put these diseases out of business,” Dr. Bonkowsky said.
“The Primary Children’s Center for Personalized Medicine is founded on our belief that every child deserves the most advanced care possible,” said Dustin Lipson, Intermountain Primary Children’s Hospital Administrator. “This center has been made possible because of the technologies of Intermountain Precision Genomics, and the synergies created by the open, collaborative environment between Primary Children’s Hospital and University of Utah Health.”
