Leukemia is a cancer of the blood and bone marrow, diagnosed in about 60,000 Americans each year. Treatment varies widely by type — acute leukemias require immediate intensive chemotherapy, while chronic forms may be managed with pills for years. Survival has improved dramatically over the past two decades, especially for certain subtypes.
What's actually going on in research
Trials are testing CAR T-cell therapies, bispecific antibodies that redirect immune cells to cancer, targeted pills that block specific mutations like FLT3 and IDH, and new combinations that may cure more people without stem cell transplant. Researchers are also working to make transplant safer and to develop treatments for leukemias that have stopped responding to current drugs.
CAR T-cell therapy
After success in other blood cancers, CAR T is now being tested in acute leukemias. Early results show some people achieving remission when standard treatment failed.
Bispecific antibodies
Drugs like blinatumomab connect a person's own immune cells to leukemia cells, forcing the immune system to attack. Several bispecifics are now FDA-approved, and trials are testing newer versions.
Targeted mutation drugs
Pills that target FLT3, IDH1, and IDH2 mutations are extending survival in acute myeloid leukemia. Trials are testing them in combination with chemotherapy and in earlier stages of disease.
What to know before you search
Eligibility typically depends on leukemia type and subtype, chromosome or gene mutations, prior treatments, remission status, and whether transplant has been done.
What types of trials are currently open
- Treatment trials — Testing new chemotherapy combinations, targeted pills, or immunotherapies to see if they lead to deeper or longer remissions than current treatment.
- CAR T and cell therapy trials — Studies of genetically modified immune cells designed to find and kill leukemia cells.
- Maintenance trials — Testing whether continuing treatment after remission — with pills or other therapies — prevents relapse.
- Transplant trials — Studies aimed at making stem cell transplant safer, reducing complications, or determining who needs it.
- Relapsed disease trials — Testing new drugs or combinations for leukemia that has come back or stopped responding to treatment.
Recently added Leukemia trials
Asciminib and Inotuzumab Ozogamicin for Relapsed or Refractory Philadelphia Chromosome-positive B-cell Acute Lymphoblastic Leukemia (R/R Ph+ B-ALL)
This is a multi-center phase 1 open-label study to determine the safety of ABL001 and INO in patients with relapsed or refractory Philadelphia chromosome-positive b-cell acute lymphoblastic leukemia.
CD19 Sequential CD22 In Vivo CAR-T Cell Immunotherapy for Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia
This is a single-center, open-label, single-arm, prospective, investigator-initiated dose-escalation study to evaluate the safety, tolerability, preliminary efficacy, and cellular kinetics of an in vivo CAR-T cell immunotherapy targeting CD19 sequentially followed by CD22, in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). The investigational product is a targeted lentiviral vector that transduces the patient's endogenous T/NK cells in situ; no leukapheresis, ex vivo manufacturing, or lymphodepleting conditioning is required. Participants receive an intravenous infusion of CD19-targeted in vivo CAR-T on Day 0, followed by an intravenous infusion of CD22-targeted in vivo CAR-T on Day 7. Before the Day 7 infusion, the investigator will assess each participant to determine whether the second infusion should proceed, based on: (i) the proportion of CAR-positive T/NK cells in peripheral blood, which reflects the success of in vivo transduction and early expansion after the first infusion; (ii) the maximum grade and current control of adverse events occurring after the first infusion, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS); and (iii) the participant's general condition, including Eastern Cooperative Oncology Group (ECOG) performance status and vital organ function (cardiac, hepatic, renal, pulmonary, and coagulation). The second infusion will be postponed or cancelled if any of these assessments indicates that the risk outweighs the benefit, for example grade \>=3 CRS or grade \>=2 ICANS that has not resolved to grade \<=1, or investigator-judged deterioration in organ function. Dose escalation follows a traditional 3+3 design with planned dose levels of 1×10\^9, 3×10\^9, and 6×10\^9 transducing units (TU), guided by dose-limiting toxicities (DLTs) assessed during Days 0-28; higher dose levels may be explored if the maximum tolerated dose is not reached. Treatment-emergent adverse events and serious adverse events, including CRS, immune effector cell-associated neurotoxicity syndrome (ICANS), hematologic toxicity, and organ toxicity, will be assessed. Preliminary antitumor activity will be evaluated by objective response rate (ORR), overall survival (OS), and progression-free survival (PFS), and the expansion and persistence of in vivo-generated CAR-T cells in peripheral blood will also be evaluated.
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