An experimental gene-editing treatment has been shown to dramatically reduce cholesterol levels in a small, preliminary study. Cientistas indicated that the therapy may offer a possibly permanent solution after just one infusion. The results were released this Monday, raising expectations about the future of heart disease prevention.
Esta preliminary discovery, if confirmed in larger studies, could revolutionize the prevention of cardiovascular diseases, the leading cause of death in many countries. Most gene therapies currently focus on rare diseases, but application to a condition as widespread as heart disease represents a significant advance. Cardiologistas see the potential single-dose “cure” as a milestone.
LDL cholesterol Redução by 62% after single infusion
The study, published in *New England Journal of Medicine*, presented an interim analysis of 35 patients. Todos participants had genetically elevated levels of LDL cholesterol, known as “bad cholesterol,” or were already facing established heart disease. Esta clinical trial phase is expected to include up to 85 participants.
A single infusion of the highest dose of the experimental treatment resulted in a reduction in LDL levels by up to 62% among the 35 patients analyzed. The drop in cholesterol levels remained stable and lasting in a subgroup of patients who were followed for 18 months. Tal consistency is crucial to validating the long-term efficacy of a single-dose therapy.
Impacto in the prevention of cardiovascular diseases
The research raises the possibility of a “one-and-done” way to prevent heart disease on a large scale. Cardiovascular diseases are responsible for almost 800 thousand deaths annually in Estados Unidos alone. Early and effective intervention is a constant debate in cardiology.
John H. P. Alexander, a cardiologist at Universidade Duke who was not involved in the study, emphasized the importance of the discovery. “We have these debates and new guidelines saying we should treat people sooner,” Alexander said. Ele added that “a curative therapy would change the game” in the current scenario.
Segurança and next research steps
The publication of such preliminary results in *New England Journal of Medicine* is considered unusual. Eric Rubin, editor-in-chief of the magazine, commented that the essay “seems to work very well.” Ele noted that the attempt to apply advanced gene therapy to the leading cause of death in Estados Unidos is ambitious.
Contudo, much more safety data is needed before final approval of an innovative treatment.
- The next step in the research will be a larger study, which will include 200 patients to confirm efficacy and safety.
- The Food and Drug Administration (FDA) requires that all patients in gene therapy studies be followed for a period of 15 years.
- Essa Close monitoring aims to ensure that no long-term adverse effects occur.
J. Michael Gaziano, director of preventive cardiology at the Departamento of Assuntos of Veteranos and Boston health system, who was not involved in the study, reinforced the need for more information. “We need much more safety data,” declared the expert. Caution is a fundamental element in high-impact experimental treatments.
Desafios and adherence to current treatments
Atualmente, high LDL levels are largely controllable with several medications available on the market. Entre the most common treatments are the traditional, widely prescribed daily statin tablets. More recent Avanços include injections that act by blocking the protein produced by the PCSK9 gene, generating an effect similar to that of direct gene editing.
Apesar of the effectiveness of existing therapies, many people cannot or simply do not want to follow ongoing medication regimens. Estudos show that between a third and half of patients stop taking cholesterol medications within a year of starting treatment. Este adherence problem persists even among individuals who have already suffered a heart attack. The convenience of a single dose could therefore overcome this significant barrier to disease prevention.