About the Event
From Bench to Bedside: The Role of Science in Treating a Deadly Disease.
Join us for an educational evening with Distinguished Professor Leslie Leinwand, who will present her journey in finding and developing treatments for genetic diseases where none existed, with a focus on familial hypertrophic cardiomyopathy and the first FDA-approved drug in 2021 for this disease, developed by MyoKardia (a company co-founded by Professor Leinwand in 2012 and since been acquired by Bristol Myers Squibb).
I was fascinated by human genetics by the time I went to college, and have been passionate for decades about unraveling genetic contributions to disease. A long path that took a few twists and turns ultimately led me to a longstanding goal: to develop treatments for genetic diseases where none existed. It started with mapping genes to human chromosomes. The next phase led me to be an academic scientist. Here at the University of Colorado, my lab studied how certain muscle mutations lead to disease. In other words, how a small DNA change leads to an often fatal crisis in the heart. It is this kind of work, sometimes called basic research, that lays the foundation for the bridge from bench to bedside.
The disease that we continue to study is called familial hypertrophic cardiomyopathy, a long way of saying a big sick heart that runs in a family. This disease is most notable for killing young athletes, sometimes with no prior symptoms. In fact, familial hypertrophic cardiomyopathy is the leading cause of sudden death in young people. Geneticists identified what genes, if mutated, cause this disease. Once tools became available, it was possible to determine which members of a family carried that mutant gene, frequently after someone in the family suffered a cardiac arrest. Having the knowledge of your genotype is an important advance. But, what do you do with that information when the only effective treatment for this disease was a heart transplant? Many were told to stop athletic activity; others were followed by a cardiologist, but this was a very anxious way of life. My talk will tell the story of finding a treatment for a fatal genetic disease of the heart.
About the Speaker
Leslie Leinwand, PhD is a Molecular, Cellular, and Developmental Biology (MCDB) Distinguished Professor and the Chief Scientific Officer of the BioFrontiers Institute at the University of Colorado Boulder. She was recruited to be Chair of MCDB in 1995. She received her Bachelor’s degree from Cornell University, her PhD from Yale University and did post-doctoral training at Rockefeller University. She joined the faculty at Albert Einstein College of Medicine in New York in 1981 and remained there until moving to Colorado in 1995. She co-founded Myogen, Inc. which was sold to Gilead Pharmaceuticals. She was also a co-founder of Hiberna, Inc, and more recently of MyoKardia, Inc, a publicly traded company founded to develop therapeutics for inherited cardiomyopathies. She is a Fellow of the AAAS, former MERIT Awardee of the NIH, Established Investigator of the American Heart Association and was recently elected to the American Academy of Arts and Sciences and the National Academy of Inventors. The interests of Dr. Leinwand’s laboratory are the genetics and molecular physiology of inherited diseases of the heart and how gender and diet modify the heart. The study of these diseases has required multidisciplinary approaches, involving molecular biology, mouse genetics, mouse cardiac physiology, and the analysis of human tissues. Her teaching was recognized by funding from the Howard Hughes Medical Institute’s Professor Program.
(Biography sourced from the website of the BioFrontiers Institute)
About the Series
The CU Boulder Retired Faculty Association (UCBRFA) presents the distinguished professors of the University of Colorado, a lecture and presentation series featuring some of our finest professors and their extraordinary research and scholarly work.