A Phase 1 clinical trial at Cleveland Clinic has shown that a single infusion of gene-editing therapy can significantly and permanently reduce cholesterol and triglyceride levels in patients whose conditions don't respond to standard medications.

The trial results, presented Saturday November 8, 2025 at the American Heart Association's Scientific Sessions 2025 and published simultaneously in the New England Journal of Medicine, showed the experimental CRISPR-Cas9 treatment reduced LDL cholesterol by approximately 50% and triglycerides by about 55% in study participants.

"This treatment is still very early in development but if future trials continue to demonstrate safety and efficacy, the therapy has the potential to change the way we treat lipid disorders," said Cleveland Clinic cardiologist Dr. Luke Laffin, first author of the study. "Rather than a once-daily pill or monthly injection, this therapy would potentially offer a one-time infusion that is safe and durable for patients with high cholesterol."

The 15-patient trial, conducted between June 2024 and August 2025 at six sites in Australia, New Zealand and the United Kingdom, included adults ages 31 to 68 with uncontrolled high triglycerides and elevated LDL cholesterol levels.

Dr. Steven Nissen, Chief Academic Officer of the Heart, Vascular & Thoracic Institute at Cleveland Clinic and senior author of the study, emphasized the revolutionary nature of the therapy. "Once the gene is edited, this is a one and done treatment," Nissen said. "This is really quite revolutionary because the alternative is to take medication every day or inject it every week or two for the rest of your life."

The treatment, called CTX310, uses CRISPR-Cas9 gene-editing technology that Dr. Laffin described as "molecular scissors" that permanently switches off a gene called ANGPTL3 in the liver. People born with a naturally occurring mutation in this gene have lifelong low cholesterol and triglycerides with lower lifetime risk of heart disease and no apparent harmful effects.

The trial reported no serious adverse events related to the treatment. Three participants experienced minor reactions including back pain and nausea that resolved with medication.

The therapy addresses a critical adherence problem. "We know that up to 50% of people by a year after their statin prescription stop taking it," Dr. Laffin noted. Dr. Nissen explained that because high cholesterol doesn't cause symptoms, people tire of taking daily medications.

For many patients, genetics determine 70-75% of their cholesterol levels. "People can eat like rabbits and exercise daily, and it's going to improve their cholesterol," Dr. Laffin explained. "But these are individuals who still can't get to the targets we need them to be to reduce their cardiovascular risk."

Dr. Nissen reflected on the breakthrough: "I used to tell people, we can change everything about you, we can give you drugs, we can change your cholesterol, the only thing we can't change is your parents, cause we can't change your genes. Well, that was wrong, because now for the first time in history, we can change people's genes."

Based on the positive results, Phase 2 studies are planned to begin in 2026. The researchers hope to have the therapy in Phase 3 studies within 2 to 3 years and are working to bring trials to the United States.

Monica Robins is not a medical doctor. All content presented in this program is for informational purposes only. It is not intended to be a substitute for professional medical advice and should not be relied on as health or personal advice. Always seek the guidance of your personal doctor or qualified healthcare professional with any questions regarding your health or medical condition.

Lung cancer remains one of the most prevalent cancers in the United States, affecting more people than breast, ovarian, and colon cancers combined. Yet misconceptions about who can develop the disease persist, according to Dr. Laurie Matt-Amaral, a medical oncologist at Cleveland Clinic Akron General.

"We see a lot of patients who get diagnosed with lung cancer and have never been a smoker," Dr. Matt-Amaral said during a recent interview for Lung Cancer Awareness Month. "They have no exposure, potentially, and the cause is really unknown."

While smoking remains a significant risk factor, Dr. Matt-Amaral explained that secondhand smoke exposure, asbestos, radon, pollution, and other environmental factors may contribute to lung cancer development. The medical community continues researching these potential triggers.

A particularly concerning trend is the increase in younger patients being diagnosed with lung cancer and other cancers. Dr. Matt-Amaral noted that in recent weeks, she has treated patients younger than herself with metastatic cancers of various types.

"There's really been a focus from a researcher's perspective," she said. "We're just seeing so much of it in younger people."

The rise in younger diagnoses may stem from both increased awareness and an actual increase in cases. Dr. Matt-Amaral observed that younger generations are more proactive about seeking medical care when symptoms appear, while researchers work to identify potential causes and preventive measures.

While vaping and marijuana use show evidence of causing lung damage, Dr. Matt-Amaral emphasized that researchers have not yet established a direct causal link to cancer. However, she noted that any changes to lung tissue DNA can increase cancer risk.

One challenge facing younger patients is being taken seriously by healthcare providers. Dr. Matt-Amaral acknowledged this concern but said the oncology community has worked to educate physicians that cancer can affect anyone, regardless of age or exposure history.

"Some people have had to see two, three, four doctors to sort of get their complaints addressed and come to find out it's a real cancer," she said.

Current screening guidelines from organizations like the American College of Physicians and the U.S. Preventive Services Task Force have not changed, partly because researchers still do not fully understand all risk factors. However, Dr. Matt-Amaral suggested that unexplained symptoms warrant investigation.

Warning signs include a persistent cough, unexplained weight loss, coughing up blood, chest discomfort, and new shortness of breath during normal activities. Dr. Matt-Amaral stressed that coughing up blood requires emergency attention.

For those who smoke, Cleveland Clinic Akron General offers a smoking cessation program with specialized nurses who help patients access resources and develop quit plans. Dr. Matt-Amaral recommends a gradual approach rather than quitting cold turkey, such as reducing cigarette use by one or two per day or per week.

Treatment advances, particularly immunotherapy, have improved outcomes for lung cancer patients. Dr. Matt-Amaral said researchers continue developing new drugs and personalized treatment approaches based on each patient's specific cancer characteristics.

"I always look forward to being able to tell someone, hey, your cancer has this molecular abnormality, but good news, I have a drug to go with it," she said.

Clinical trials are available locally through Cleveland Clinic Akron General and Cleveland Clinic's main campus, as well as through other major medical centers in the region and nationwide. Dr. Matt-Amaral encouraged patients to discuss clinical trial options with their oncologists.

Her message for Lung Cancer Awareness Month is clear: lung cancer can happen to anyone. The best defense is open communication with healthcare providers, attention to symptoms, and smoking cessation for those who smoke.

Monica Robins is not a medical doctor. All content presented in this program is for informational purposes only. It is not intended to be a substitute for professional medical advice and should not be relied on as health or personal advice. Always seek the guidance of your personal doctor or qualified healthcare professional with any questions regarding your health or medical condition.