Costco co-founder Jeff Brotman and Dan Baty met as kids on a playground in Tacoma and remained lifelong friends. Before Brotman’s death in August, the two businessmen and their wives decided to share some of their wealth and pursue a common interest in health care.
The result is a $50 million gift to create the Brotman Baty Institute for Personalized Medicine in Seattle and pursue one of the most sought-after and elusive goals in science: Treatments tailored to individual patients.
The institute will be a collaboration between the University of Washington, The Fred Hutchinson Cancer Research Center and Seattle Children’s, and will work to translate cutting-edge research into improved diagnosis and therapy.
The project mirrors a national Precision Medicine Initiative created by President Barack Obama and supported by President Donald Trump. Among the national initiative’s signature goals is compiling a database of genetic, health and lifestyle information from more than 1 million volunteers to help tease out links between individual variations and disease.
“The typical patient doesn’t really exist, yet for a century medicine has focused on diagnosing and treating typical patients,” said UW geneticist Jay Shendure, leader of the new institute and an adviser to the national initiative.
Precision medicine delves into an individual’s genetic code to uncover characteristics of their illness and the best approach to fighting it. The use of genetic screening to identify people at high risk of some cancers is an early example of precision medicine. Another is the use of sensitivity tests to determine the optimum doses of certain drugs, like the anti-clotting agent warfarin.
Now, the advent of cheap genetic sequencing and the development of new gene-editing tools are allowing researchers to refine those approaches and develop entirely new ones.
“The rate at which we’re learning is tremendous, and accelerating,” Shendure said.
One focus for the new institute is a better understanding of exactly which gene mutations raise women’s risk of breast cancer.
UW scientist Mary-Claire King pioneered the study of mutations in the genes called BRCA1 and BRCA2 that can cause early onset breast and ovarian cancer. But both genes are large and could theoretically mutate in 30,000 different ways, Shendure explained.
Women with poorly understood gene variations often face a disquieting diagnosis: Yes, they have a mutation, but doctors can’t tell them whether it’s harmless or dangerous.
“The dirty secret is that a very substantial proportion of women actually get this result … which causes anxiety and confuses the physicians,” Shendure said.
The new funding will accelerate work already underway at the UW to engineer genes with all possible mutations, then test them in human cell cultures to see if they are likely to increase cancer risk.
Shendure also wants to expand genome testing for children suspected of having genetic disorders.
Screening already exists for some well-known genetic mutations, like those that cause sickle cell anemia or Tay-Sachs disease, which attacks brain cells. But the range of possible genetic disorders is staggering, Shendure said.
“There are hundreds or even thousands of these which are much more challenging to diagnose and that no one has ever heard of,” he said. There’s also growing evidence that some mutations may be linked with autism and intellectual disability, he added.
Rather than testing one gene at a time, Shendure wants to develop protocols for more extensive genetic sequencing and analysis to speed diagnosis and treatment. “We have the technology,” he said. “We have to figure out how to implement it here.”
Some scientists remain skeptical that precision medicine will transform medicine, and accuse advocates of overhyping the approach. Shendure is more optimistic, but he recognizes the challenge of tackling diseases that might involve multiple genes as well as behavioral and environmental factors.
“We need to be as clear as we can that this is a long haul,” he said. “If it takes a few generations to get there, that’s OK.”