Innovative gene-editing trial paused as patient experiences unusual side effects
Verve Therapeutics, a pioneering biotech start-up, has temporarily halted its inaugural human gene-editing trial known as the "Heart-1" trial. The decision was made following unexpected side effects experienced by a participant, according to Bloomberg. The trial is integral to Verve's mission of using gene-editing to reduce cholesterol levels that cause heart attacks in patients with familial hypercholesterolemia, a genetic disorder leading to excessive LDL cholesterol buildup.
Verve's innovative approach to lowering cholesterol
Verve's proposed solution involves injecting a serum named "VERVE-101" into the liver of patients. This serum is designed to reduce fatty LDL molecules by genetically modifying the PCSK9 gene, which plays a pivotal role in cholesterol management. The approach was developed after promising tests on monkeys, which led to the initiation of human trials. However, these trials have encountered challenges due to unexpected side effects in participants.
What exactly prompted the trial suspension
A participant in the Heart-1 trial suffered a heart attack last year leading to his death, although experts agreed that VERVE-101 was not to blame, attributing it instead to the patient's severe heart disease. Now, another participant experienced serious side effects, raising enough safety concerns to warrant a pause in the trial. Among the initial six patients who participated in the Heart-1 trial, one developed "abnormal liver enzymes" and thrombocytopenia - a condition characterized by a low blood platelet count.
5 participants witnessed a reduction in their cholesterol levels
Thrombocytopenia presents a severe condition which, in severe instances, may result in fatal internal bleeding. The symptoms of the patient reportedly vanished after a few days without the medication. Notably, the other five participants witnessed a decrease in their cholesterol levels without any side effects.
Verve shifts focus to new gene-editing drug
In response to the unusual side effects, Verve is now focusing on VERVE-102, a new version of the gene-editing drug suspended in a different lipid serum. The company suggests that the irregularities were caused by fatty lipids present in the serum used to administer VERVE-101 into patients' livers. According to Verve's website, the new delivery system was "well tolerated" by patients in an independent clinical trial. The upcoming Heart-2 trial is set to begin in second quarter of this year.
