Pediatric Comprehensive Bone Marrow Failure Center News

Meet physician-scientist Stella T. Chou, MD, the new Chief of the Division of Hematology. Read about the promise of emerging therapies, what makes CHOP unique and more.

New research from Children’s Hospital of Philadelphia (CHOP) uncovered a previously unknown side effect of Acute Myeloid leukemia (AML), the second most common blood cancer in children, that leaves a long-lasting imprint on healthy blood-forming stem cells, influencing immune responses long after the disease has entered remission. The results were published recently in the journal Cancer Letters.

In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel treatment strategy for patients with Fanconi anemia (FA), a rare genetic disease resulting in loss of blood-forming stem cells in the bone marrow. The researchers successfully used in situ mRNA delivered directly into the body by lipid nanoparticles (LNPs) to target bone marrow cells and transiently restore stem cell function in patients with FA.

Inspired by her own journey, a CHOP patient helps other children deal with the stress of managing an illness.

Study also found that a readily available bile salt could be used as a treatment to restore proper function.

CHOP researchers find exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.

Dr. Oved brings deep expertise to the group, having completed scientific and clinical training that included tenures as a Fulbright Fellow with the Weizmann Institute in Israel, the NYU School of Medicine with two years as a Howard Hughes Medical Fellow, followed by a residency in Pediatrics at NYP Weill Cornell Medical College.

Millicent, a dedicated BMFC staff member, was selected as a Spark Most Valuable Person (MVP) for the Mentor All Star Awards.