Ali Mackanic

Excited to see this new paper describing our real world experience of the first 50 patients with hypertrophic cardiomyopathy prescribed a cardiac myosin inhibitor at Stanford come out last week. Authors include the entire Stanford Center for Inherited Cardiovascular Disease team (it takes a village!). The cardiac myosin inhibitor story really is remarkable. Here's a disease we've known about for 300 years, the molecular basis of which we've known since the early 1990s, but for which until very recently we had no precision therapy. Our mainstay treatment, beta blockers, gave most patients side effects and did very little for their symptoms and literally nothing vs the underlying disease. But then, enter science! Science from the labs of Jim Spudich at Stanford and Leslie Leinwand in Boulder led to a small molecule screen to identify a cardiac myosin inhibitor to try to treat the underlying cause of the disease. Rapid progress through Phase I-III trials led to Myokardia's acquisition by BMS for $12bn and now the drug is literally changing patients' lives in the real world. Almost every patient we have prescribed the drug expresses moderate or significant benefit. In some cases, they describe that it has transformed their lives. In other cases, we have seen clear evidence of the disease reversing. This is what the application of science and the careful execution of a novel precision therapy within the health care system can do. True teamwork! frontiersin.org/journals/car… Figure: Evidence of disease reversal in hypertrophic cardiomyopathy after one year of treatment with a cardiac myosin inhibitor