CB-103: A novel CSL-NICD inhibitor for the treatment of NOTCH-driven T-cell acute lymphoblastic leukemia: A case report of complete clinical response in a patient with relapsed and refractory T-ALL.
Michael MedingerTill JunkerDominik HeimAlexandar TzankovPhilip M JermannMaria BobadillaMichele VigoloRajwinder LehalFlorian D VoglMichael BauerJakob PasswegPublished in: EJHaem (2022)
Relapsed T cell acute lymphoblastic leukaemia (T-ALL) has a very poor prognosis. A 24-year-old patient with relapsed high-risk T-ALL (PTEN gene deletion; NOTCH1 mutation), was treated with the NOTCH inhibitor CB-103. Within 1 week of starting CB-103, the bone marrow was free of T-ALL blast infiltration (MRD+) and successfully underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT). Sequential samples of ctDNA to monitor the disease after allo-HSCT showed a decrease of circulating Notch1 and PTEN alterations. This is the first T-ALL patient treated with CB-103. The observed clinical response encourages further exploration of CB-103 in ALL.
Keyphrases
- acute lymphoblastic leukemia
- allogeneic hematopoietic stem cell transplantation
- poor prognosis
- acute myeloid leukemia
- cell proliferation
- case report
- bone marrow
- multiple myeloma
- hodgkin lymphoma
- long non coding rna
- diffuse large b cell lymphoma
- mesenchymal stem cells
- pi k akt
- liver failure
- intensive care unit
- dna methylation
- newly diagnosed
- respiratory failure
- signaling pathway
- study protocol
- hematopoietic stem cell
- acute respiratory distress syndrome