Gene Therapy Causes Patient to Grow Alarming Tumor

"In a new study published in The New England Journal of Medicine, researchers concluded that a young Hurler syndrome patient who received an adeno-associated virus (AAV) - used to deliver a therapeutic gene to the patient's DNA - later developed a tumor due to the therapy. The patient's illness, Hurler syndrome, stemmed from a mutation that thwarted an enzyme in his body, impacting brain development. A bone marrow transplant was unable to resolve the issue, which ultimately led to the gene therapy."

"After receiving the treatment, the boy's brain began to develop appropriately for his age, but by age five, doctors spotted a walnut-sized tumor in his brain. The AAV, researchers came to believe, spurred the development of the tumor after it mutated an unintended gene. Luckily, the story has a happy ending. The tumor was successfully removed, and the development of the patient's cognitive abilities remains on track."

"But it's hard not to worry about this newly discovered risk. Researchers involved with the case believe that this outcome is a rarity, Science noted, but it'll surely weigh on other patients - and their loved ones - who undertake similar treatments in the future. Gene therapy - attempting to help a patient by modifying a gene expression in their body - has shown immense promise for treating a growing range of severe illnesses and disabilities."

Gene therapy aims to treat severe illnesses by modifying gene expression in a patient’s body. Recent advances have shown promise for conditions such as Huntington’s disease and congenital deafness, but the approach carries risks because viral delivery systems can affect genetic material in unintended ways. A Hurler syndrome patient received an adeno-associated virus (AAV) to deliver a therapeutic gene after a bone marrow transplant failed. After treatment, brain development improved, but by age five doctors found a walnut-sized brain tumor. Researchers concluded the AAV likely promoted tumor development by mutating an unintended gene. The tumor was removed successfully, and cognitive development remained on track, though the risk may affect future patients’ decisions.

Read at Futurism