New Hope for Down Syndrome Leukemia: Protein Target Discovered
A groundbreaking study has shed light on the mechanism behind the increased risk of leukemia in children with Down syndrome. The research, published in Science, shows that a specific protein plays a crucial role in the development of the disease.
Princess Margaret researchers have mapped the origins and development of leukemia in infants with Down syndrome for the first time. They discovered that the extra copy of chromosome 21 in Down syndrome predisposes individuals to leukemia by causing alterations in hematopoietic stem cells. These blood-forming stem cells are implicated in the initiation of the disease as early as the second trimester of fetal development.
The study found that a unique protein cell surface marker, CD117/KIT, is present on preleukemic stem cells. This protein causes cell proliferation, leading to the progression of the disease. Dr. John Dick, the senior author of the study, and his team discovered that targeting and eliminating these preleukemic stem cells using CD117/KIT inhibitors can prevent the progression to acute leukemia in preclinical models. This preventative strategy could potentially be used in Down syndrome newborns and other childhood leukemias initiated during fetal development.
The research was supported by various organizations, including the Human Frontier Science Program and The Leukemia & Lymphoma Society. It provides a significant step forward in understanding and potentially preventing leukemia in children with Down syndrome.
The study published in Science on July 9, 2021, offers a promising avenue for preventing leukemia in Down syndrome infants. By targeting and eliminating preleukemic stem cells, the progression to acute leukemia can be halted. This breakthrough could have a significant impact on the lives of children with Down syndrome and other childhood leukemias.
