Scientists share breakthrough that could limit heart valve disease, help patients avoid replacement surgery
After 15 years of research, a cardiologist-led group of scientists has discovered a potential drug candidate for heart valve disease. The team shared its findings in Science, noting that they hope to “avoid tens of thousands of surgical valve replacements every year.”
“The disease is often diagnosed at an early stage and calcification of the heart valves worsens over the patient’s lifetime as they age,” study leader Deepak Srivastava, MD, a pediatric cardiologist and the president of Gladstone Institutes, said in a prepared statement. “If we could intervene early in life with an effective drug, we could potentially prevent the disease from occurring.”
Srivastava first started exploring this topic in 2005, when he encountered a family that had been affected by heart valve disease for five generation. He and his colleagues found that a mutation in the gene NOTCH1 was behind that family’s illness—and that discovery proved to be quite the breakthrough.
“The NOTCH1 mutation provided a foothold for us to figure out what goes wrong in this common disease, but most people won’t have that mutation,” Srivastava explained in the same statement. “However, we found that the process that leads to the calcification of the valve is mostly the same whether individuals have the mutation or not. The valve cells get confused and start thinking they’re bone cells, so they start laying down calcium and that leads to hardening and narrowing of the valves.”
This finding, along with some AI models and a collaboration with the Russian Academy of Sciences, helped Srivastava’s team find a potential treatment for heart valve disease. After successful research with mouse models, they are now ready to launch a clinical trial.
“By using all the knowledge we gathered over a decade and a half, combined with the latest tools, we were able to find a drug candidate that can be taken to clinical trials,” Srivastava added. “Our ultimate goal is always to help patients, so the whole team is very pleased that we found a therapy that could truly improve lives.”
Read the full analysis in Science here.