New therapies for cardiovascular disease reach clinical trial stage
Genomic-based therapies for cardiovascular disease are emerging fast in research and could soon be used regularly in hospitals and practices around the world — changing future treatment, said Ann Marie Schmidt, MD, FAHA.
“The concept of therapeutic, genomic-based therapies for cardiovascular diseases has now reached clinical trials,” said Schmidt, professor of biochemistry, molecular pharmacology and pathology at NYU Langone Health in New York City.
“This area of research and medicine offers landmark, game-changing opportunities for diseases in which genomic profiling in humans (and in animals) has shed light on key mechanisms of disease.”
Several areas of the research will be highlighted in Saturday’s session, “Identifying and Practicing Emerging Genomic-Based Therapies on Cardiovascular Disease.” Dr. Schmidt said the session — co-moderated by Alan Daugherty, PhD, DSc, FAHA, associate vice president for research and physiology chair at the University of Kentucky College of Medicine in Lexington — will focus on several key areas of genomic therapies, including:
- PCSK9 gene for cholesterol metabolism
- TTR gene for amyloidosis and heart disease
- Angiotensinogen for blood pressure regulation
- Hemophilia for bleeding disorders
- ApoC3, Angptl3 for dyslipidemia disorders
The move from animal testing to human subjects represents a significant leap forward for these therapies, Dr. Schmidt said.
“The work in many cases has moved from understanding basic mechanisms in animal model systems to application to human subjects, thereby underscoring the immense value of basic to translational/clinical to population level research,” she said.
Genomic profiling, Dr. Daugherty said, allows researchers to identify the genes in a person or specific cell type and how those genes interact with each other and the environment. This aids in the mission to personalize medicine with respect to a physician’s ability to diagnose, treat and prevent diseases.
“Genomic profiling holds great promise to help us understand why certain people are vulnerable to certain diseases while others are not,” Dr. Daugherty said. “And also to begin to predict which patients may or may not respond to specific therapies. By better understanding how the uniqueness of each person renders them more or less vulnerable to disease and more or less likely to respond to specific therapies, these methods may personalize therapies for cardiovascular diseases.”
Several areas of study will be important in this field in the future, where genomic profiling will have broad reach, according to Dr. Schmidt. These include lipid disorders (including cholesterol) and the impacts for atherosclerosis; renin-angiotensin system targets that could impact treatment of hypertension; and blood disorders and the treatment of hemophilia.
As these areas of research move further into human trials, Dr. Schmidt said the impact on patient health care could be tremendous.
“The realization of some of these new targets for human clinical trials and testing are critical examples of the success of biomedical research and the likelihood for important impact on the heath and lifespans of individual people,” she said.