CARDIOVASCULAR JOURNAL OF AFRICA • Volume 35, No 1, January – April 2024 34 AFRICA myxomas – a 30-year clinical experience. Ann Thorac Surg 1995; 59(4): 851–855. 33. Siminelakis S, Kakourou A, Batistatou A, et al. Thirteen years followup of heart myxoma operated patients what is the appropriate surgical technique? J Thorac Dis 2014; 6(Suppl 1): S32–S38. 34. Lad VS, Jain J, Agarwala S, et al. Right atrial trans-septal approach for left atrial myxomas nine-year experience. Heart Lung Circ 2006; 15(1): 38–43. 35. Hosain N, Quaium Chowdhury MA, Maruf MF, et al. Surgical treatment of atrial myxomas: outstanding outcome of a treacherous tumor. CJC Open 2020; 3(3): 354–360. 36. Elbardissi AW, Dearani JA, Daly RC. Survival after resection of primary cardiac tumors: a 48-year experience. Circulation 2008; 118(14 suppl): S7–15. 37. Jiang CX, Wang JG, Qi RD, et al. Long-term outcome of patients with atrial myxoma after surgical intervention: analysis of 403 cases. J Geriatr Cardiol 2019; 16: 338–343. 38. Vroomen M, Houthuizen P, Khamooshian A, et al. Long-term follow up of 82 patients after surgical excision of atrial myxomas. Interact Cardiovasc Thorac Surg 2015; 21: 183–188. 39. Shah IK, Dearani JA, Daly RC, et al. Cardiac myxomas: a 50-year experience with resection and analysis of risk factors for recurrence. Ann Thorac Surg 2015; 100: 495–500. 40. Cianciulli TF, Cozzarin A, Soumoulou JB, et al. Twenty years of clinical experience with cardiac myxomas: diagnosis, treatment, and follow up. J Cardiovasc Imaging 2019; 27: 37–47. … continued from page 11 The treatment ‘is almost like science fiction’, said Dr Martha Gulati, director of preventive cardiology at the Smidt Heart Institute of Cedars-Sinai Medical Centre in Los Angeles and president of the American Society for Preventive Cardiology, who was not involved in the trial. The geneediting tool acts like a pencil and an eraser. The eraser wipes out one letter of the target gene, and the pencil writes in a new one, turning off PCSK9. The goal: a single cholesterollowering treatment that results in lifelong protection from heart disease. Patients received varying doses. LDL cholesterol levels in the three who received the highest doses fell by 39 to 55% – enough to get them towards their cholesterol goal. In the study, those who received the higher doses had flu-like symptoms for a few hours. Two patients had serious adverse events that the study’s independent data safety and monitoring board deemed a result of their underlying severe heart disease. The board advised the researchers not to stop the study. One had a fatal cardiac arrest five weeks after receiving the infusion. An autopsy showed that several of his coronary arteries were blocked. The other patient had a heart attack the day after the infusion. It turned out that he had been having chest pain before receiving the infusion but had not reported it. If the investigators had known, he would not have received the treatment. In a way, the treatment is a culmination of studies that began a decade ago when researchers discovered rare but healthy individuals with cholesterol levels that seemed impossibly low. The reason was that their PCSK9 gene was mutated and no longer functioned. As a result, these people were protected from heart disease. That led to the development of antibodies to block the gene. Patients inject themselves with the antibodies once a week. Then came a twice-yearly RNA injection that prevents PCSK9 from being made. It seemed possible that those treatments, as well as statins for those whose cholesterol is more easily controlled, could help solve the heart disease problem. But heart disease persists as a killer. Even after people are diagnosed with heart disease, less than 60% of all patients take a statin. Only a quarter take one of the more effective, high-intensity statins, Gulati said. ‘Patients take it initially, and then they forget or decide they don’t need it,’ she said. ‘That happens more than you’d think.’ Dr Michelle O’Donoghue, a cardiologist at Brigham and Women’s Hospital, said that because patients so often do not take their pills or injections, ‘there is a lot of interest, through gene editing, of a one and done – a single treatment and a lifetime response’. Family history was the inspiration for Kathiresan at Verve Therapeutics. His uncle and grandmother died of heart attacks. His father had a heart attack at 54 years. And then, on 12 September 2012, his 42-year-old brother returned from a run dizzy and sweaty. He was having a heart attack. He died nine days later. At that moment, Kathiresan said, he vowed to find a way to prevent what had happened to his brother from happening to anyone else. Of course, even if gene editing works, applying it to young people with heart risk is well into the future. But, Gulati said, early gene editing of younger patients with genetically high cholesterol levels might prevent arteries from hardening. ‘It could be an incredible medicine,’ she said. This all depends on success and safety of the gene editing and on its effects lasting. The first patient was treated just six months ago. But a previous study in monkeys lasted two and a half years, and the results of the gene editing persisted. Urnov, who said he has a genetic risk for heart disease, is optimistic for himself and his six-year-old daughter. ‘I honestly cannot wait for this medicine to become available for heart disease prevention,’ he said. ‘I love the idea of having gene editing as a vaccine for the prevention of heart disease.’ Source: MedicalBrief 2023
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