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
Share your medical records to help us understand a rare childhood leukemia
The study aims to conduct a real-world investigation of children with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in China, assessing their diagnosis, treatment status, survival outcomes, and prognostic factors. The research will involve children diagnosed with Ph+ ALL between January 1, 2015, and December 31, 2024. Our objective is to understand the incidence and prognosis of CML-like ALL, compare the diagnostic and treatment differences between CML-like ALL and typical Ph+ ALL, and provide clinical evidence for standardized treatment of CML-like patients. Through a multicenter retrospective study, we hope to improve the diagnostic and treatment strategies for children with Ph+ ALL, enhancing patient survival rates and quality of life.
Try a digital health program designed for families managing childhood cancer
This multicenter randomized controlled trial aims to develop and evaluate two Smart Holistic Health Care Platforms (SHHCPs) for families of children with cancer in Taiwan: the Parent Smart Holistic Health Care Platform (P-SHHCP) for parents and the Child Smart Holistic Health Care Platform (C-SHHCP) for children. Children diagnosed with leukemia or brain tumors and their primary caregivers (parents) will be recruited from pediatric oncology and hematology units at four medical centers in northern Taiwan. The SHHCPs are designed to strengthen communication and connection between families and health care providers and to provide holistic support for physical, psychological, social, and spiritual needs during cancer treatment.The study will evaluate the effects of the P-SHHCP and C-SHHCP on the physical, psychological, social, and spiritual well-being of children with cancer and their parents during treatment.
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