VABILO

Nacionalni inštitut za biologijo Vas vabi, da se udeležite seminarja

»Improving outcomes for children with relapse and refractory acute myeloid leukemia«,

ki ga bo predstavil

dr. Richard Aplenc, MD, PhD

Mai and Harry F. West Endowed Chair in Pediatric Research
Professor of Pediatrics and Epidemiology
AVP and Chief Clinical Research Officer
The Children’s Hospital of Philadelphia
Perelman School of Medicine at the University of Pennsylvania
 

Seminar bo v ponedeljek, 17. marca 2025, ob 13. uri na Nacionalnem inštitutu za biologijo,
Večna pot 121, 1000 Ljubljana, v veliki predavalnici v prvem nadstropju, glavni vhod C/D.

Abstract:

Acute myeloid leukemia (AML) is the second most common pediatric leukemia (500 cases per year). Current treatment regimens for AML push the limits of chemotherapy intensity but cure only 60% of children. Given the remarkable success of chimeric antigen receptor (CAR) therapies for acute lymphoblastic leukemia (ALL), we seek to develop CAR T-cell (CART) therapies for children with AML. Although AML CAR therapies have a promising efficacy signal, these therapies have not yet replicated the remarkable response rates seen in children for relapsed B-ALL, where the response rate exceeds 80%. We are actively investigating multiple strategies to improve AML CAR efficacy, including identifying novel targets uninvolved with normal hematopoiesis and Phase I clinical trials. We have developed a novel cell surface protein enrichment protocol and target discovery analytic pipeline that integrates transcriptomic and proteomic data. We have applied this workflow in two independent sample sets and identified over two hundred novel potential AML CAR targets. Taken together, our sample sets comprise the largest proteogenomic target discovery effort to date in pediatric or adult AML. Data analyses integrating transcriptomic and proteomic data identified over two hundred potential targets for pediatric AML CAR development. Notably, multiple targets were identified based on proteomic data and would not be identifiable through standard transcriptomic discovery approaches. We are also conducting a Phase I clinical trial with an anti-CD123 CAR which has shown generally acceptable toxicities and an initial efficacy signal.

Vljudno vabljeni. Seminar bo potekal v angleškem jeziku.

Na seminar se je potrebno prijaviti preko spletnega obrazca.

Dodatne informacije: doc. dr. Anže Smole, anze.smole@nib.si.