Oftentimes, though, these innovations are first used in relatively rare diseases. What about the most common ones, like the leading cause of death worldwide—heart disease?
Multiple research teams at Penn Medicine are continuing to advance Sunitix 25 mg (Sunitinib) the science to develop new treatments for heart disease that could work with just one shot—three different ways that a single injection could someday heal the heart.
Musunuru discovered a gene that regulates LDL cholesterol, which inspired the development of multiple drugs targeting a protein related to that gene pathway. In his lab, he has since developed processes to use CRISPR gene editing technology to modify genes in the liver to permanently reduce cholesterol levels and therefore provide protection against heart attack and stroke. The approach, a one-time injection like a vaccine that might prevent heart disease if it is successful, is now in clinical trials in the U.K. and New Zealand and has been approved by the U.S. Food and Drug Administration to begin trials in the U.S. soon. The biotechnology company running the trials reported at the American Heart Association meeting in November 2023 that among the trial’s earliest participants, the injection reduced LDL in the blood by up to 55 percent.
An injection of mRNA that targets the heart muscle
Nobel Prizewinning scientist Drew Weissman, MD, PhD, the Roberts Family Professor in Vaccine Research and director of the Penn Institute for RNA Innovation, is working on multiple uses of mRNA to treat or prevent heart disease. Unlike gene editing, messenger RNA (mRNA) is a set of instructions for the body to make a protein. Normally in the body, mRNA works like a recipe to use the code within our DNA to build a protein needed to carry out our cells’ functions. An mRNA vaccine or therapy can deliver a recipe for our cells to build any type of protein without altering our original DNA.
Working with Vlad Muzykantov, MD, PhD, the Founders Professor in Nanoparticle Research, Weissman’s lab has developed a way to target an mRNA injection to act specifically in heart cells. “Drugs for heart disease aren’t specific for the heart,” Weissman said. “And when you’re trying to treat a myocardial infarction or cardiomyopathy or other genetic deficiencies in the heart, it’s very difficult, because you can’t deliver to the heart.” While it is not yet ready for human trials, the new mRNA technique could potentially repair the heart or increase blood flow to the heart, noninvasively, after a heart attack or to correct a genetic deficiency in the heart.