Verve Therapeutics, a biotech company based in Cambridge, recently conducted a trial that produced promising results in the treatment of high cholesterol. The trial tested a gene-editing treatment called VERVE-101 in individuals with hereditary conditions that increase their risk of developing clogged arteries and heart attacks. By using CRISPR techniques to modify liver gene cells, the researchers were able to “turn off” a cholesterol-raising gene called PCSK9, which is found in the liver, in order to decrease LDL-C, or “bad” cholesterol, and prevent plaque buildup in the arteries.
The results of the trial revealed that PCSK9 was lowered by as much as 84 percent in the cohorts that received higher infusion rates of the treatment. Furthermore, the reduction of LDL-C-related proteins lasted as long as 2.5 years, according to previous studies on primates. This groundbreaking gene editing therapy has the potential to revolutionize the current standard treatments for high cholesterol, such as prescription statins and PCSK9 inhibitors, which can have undesired side effects like muscle pain and memory loss.
Despite the promising results, it is important to note that CRISPR gene editing is not yet ready to replace daily medications. The trial reported that two out of the 10 participants experienced a “cardiovascular event” after receiving the infusion, with one event potentially being related to the treatment. This underscores the fact that the use of gene-editing technology carries inherent risks, as edits could occur elsewhere in the genome. Therefore, further research and meticulous observation of the treatment’s long-term effects are necessary before it can be readily available to consumers.
Before a single infusion therapy for high cholesterol can be made accessible to the public, it must undergo extensive evaluation mandated by the FDA, which may last up to 15 years. Verve Therapeutics recently received FDA clearance for an Investigational New Drug Application for VERVE-101, giving the company the green light to conduct trials in the US. The current trials in New Zealand and the United Kingdom are actively seeking clinical trial participants to expand the study and gather more data. These trials are crucial in understanding the long-term safety and efficacy of the gene-editing treatment, ultimately paving the way for its potential approval for widespread use in the future.
Related Post
Although the results are groundbreaking, it is essential to approach this new treatment with caution and continue to monitor its long-term effects. While the potential benefits are significant, rigorous testing and careful consideration of the associated risks are necessary to ensure the safety and effectiveness of this innovative gene-editing therapy. As the research and development of VERVE-101 progress, it offers hope for the future of treating high cholesterol and reducing the risk of cardiovascular diseases.
