Unlocking the Secrets of the Genome to Cure Disease

By exploring the common features of widespread chronic diseases, Salk Institute investigators seek to address some of the biggest questions in medical science today.

In La Jolla, California, a faculty of innovative biological researchers is working to develop a stronger understanding of human health.

The home for their research was built when Dr. Jonas Salk, famed creator of the polio vaccine, set out to form a center for biology scholars to follow their curiosity. Established in 1960 as a vibrant community dedicated to the pursuit of scientific achievement, the Salk Institute for Biological Studies continues today with major areas of study in genetics, neuroscience and metabolic research. Supporting the Salk Institute’s longstanding exploration into the molecular and genetic basis of diseases, the Trust helped create a recent hub at the Institute, the Helmsley Center for Genomic Medicine (HCGM).

The HCGM began by assembling a group of scientists with varied interests, all of whom shared a conviction that there are common genes and shared underlying genetic pathways activated in many different chronic diseases. The Center was designed to capitalize on existing findings that inflammation may be a link in the progression of an assortment of diseases. With a special focus on this role of inflammation, HCGM researchers are tackling today’s most debilitating and expensive chronic health disorders like Alzheimer’s, diabetes and cancer.

“As the aging population of the U.S. grows, the number of those suffering chronic health problems increases along with it," said Dr. Robert Cook, Program Director of the Trust's Basical Medical Research Program. “We know that exciting opportunities exist to develop new treatments at the molecular level, but a fundamental understanding of these diseases is required first and HCGM’s intensive research is the necessary, first step to get there.”

The center is enabling scientists from the fields of genetics, metabolism, cancer, cellular imaging and others to collaborate to more effectively identify potential solutions while gaining a greater understanding of the human genome and chronic inflammation overall.

Dr. Inder Verma, Salk Institute for Biological Studies 

The ultimate goal is to identify therapeutic approaches and facilitate their translation into the clinic. To reach that goal, the Center employs a strategy of joint efforts, coordinating investigations across various disciplines to test new hypotheses about the basic science of chronic diseases. Dr. William R. Brody, president of the Salk Institute, describes it as a model that “provides an unprecedented opportunity to bring together the brightest minds in diverse fields to improve health for generations. By working together, Salk and the Helmsley Charitable Trust are enabling scientists to innovate across disciplines to solve some of the most pressing problems in healthcare.”

To unravel such complexities, these researchers are examining the human genetic material and its products, the proteome and the metabolome. And they are beginning to see initial results.

In the metabolism and diabetes focus of the HCGM, the protein FGF1 has been pinpointed as a promising insulin sensitizer able to regulate blood sugar levels and potentially serve as a new diabetic drug without typical side effects.

Within stem cell research, collaborating Salk experts are screening for molecular pathways that could relieve inflammation in nerve tissue. These and other discoveries are leading to a system for differentiating human stem cells into mini kidney structures, research which could lead to a better understanding of regeneration and aging in tissues.

And in the area of cancer, researchers have already established a powerful new mouse model system that is setting the groundwork to learn how to treat very aggressive forms of cancers of the brain, lung and prostate. Meanwhile, scientists’ protein analyses are beginning to find inspiring links between therapeutic drugs for diabetes and cancer.

Dr. Inder Verma, professor in the Salk Laboratory of Genetics and professor of molecular biology, explained, “The center is enabling scientists from the fields of genetics, metabolism, cancer, cellular imaging and others to collaborate to more effectively identify potential solutions while gaining a greater understanding of the human genome and chronic inflammation overall.” Combining that influence with the latest technology and state-of-the-art equipment, the HCGM is bringing scientific progress closer to defining a genomic approach for the treatment of chronic diseases.

Dr. Inder Verma