Leslie Leinwand is researching the genetic mutations that cause the most common inherited heart disease, hypertrophic cardiomyopathy, which, as she says, is a longwinded way of saying “big, sick heart.” The heart’s walls thicken and contract more strongly than healthy hearts. Unfortunately, the disease often goes undiagnosed until young athletes in their late teens or early 20s die suddenly.
Leinwand and her colleagues are working to understand how numerous mutations influence the disease, as well as develop small molecule therapies to fix heart muscle proteins, so they contract normally. She’ll discuss both at the MDC lecture on June 17.
What inspired you to pursue this area of research and enter the biotech sphere?
Leslie Leinwand: I started my scientific career in human genetics and ever since then I’ve had a great desire to determine whether we could develop something that might help people with genetic diseases of heart and skeletal muscle. When I first started, I wasn’t saying, “Ten years from now, I want to start a biotech company.” But I always had the idea that if we study these diseases and the gene mutations that cause them, it might ultimately lead to a benefit for people. It is particularly compelling if you feel like you can help somebody who is very, very young and who has potential for a long and full life, but because of a mutation is at risk for sudden death.
What are you most excited about right now?
Our company is in clinical trials and making some great progress towards developing therapeutics. But the disease is a lot more complicated that we initially thought. We are trying to develop new tools that can allow us to understand this disease better.
What other questions or challenges are you grappling with?
In hypertrophic cardiomyopathy, if you have mutations in the motor protein, myosin, and you are a younger woman, you’re going to have much later diagnosis and later onset of symptoms than if you are a younger man. Nobody understands this. And it is not simply tied to estrogen and testosterone. I want to try to get people to understand that heart disease in men and women can be fundamentally quite different. Treating women the same as men, just at lower doses, is unbelievably short-sighted.
What have you learned from your experience launching three biotech companies, including MyoKardia Inc. in 2012, which has raised more than $789 million USD in financing through venture capital, stock market and banks?
If you don’t have a track record, and you walk into a venture capital firm and ask them for a whole pile of money, their initial response is “OK, why should I do this for you? What do you have that 50 other people don’t have?” You have to grow a thick skin if you are going to try to do this.
What do you hope attendees will take away from your lecture?
If you have a desire to see your findings in the laboratory ultimately translate into something that might be of therapeutic benefit — it’s within reach. It does take a long time and it is really hard. It needs to be a passion. I find it to be the most rewarding outcome of the decades of basic academic research.
The questions were asked by Laura Petersen.
No registration required.