Leukemia is four very different diseases — AML, ALL, CML, and CLL — each with its own treatments and outlook. CML is now controlled long term with daily pills, CLL has moved largely to targeted therapy, and AML is seeing a wave of new drug combinations after decades of slow progress.
What's actually going on in research
Trials are testing menin inhibitors and FLT3-targeted drugs in AML, BTK and BCL-2 inhibitor combinations in CLL, and CAR-T therapy for relapsed ALL. Researchers are also studying whether some CML patients can safely stop treatment, less toxic stem cell transplant approaches, and better matching of treatment to each leukemia's genetic features.
Targeted pills
Drugs that block specific genetic drivers (FLT3, IDH, BCL-2, BTK, menin) are replacing or reducing chemotherapy in many leukemias. Several have been approved in the past few years.
CAR-T for ALL
CAR-T cell therapy is curing some children and adults with relapsed acute lymphoblastic leukemia. Trials are extending it to AML and to earlier lines of treatment.
Stopping treatment safely
In CML, studies show that some patients with deep, long remissions can stop their daily pill and stay in remission. Research is identifying who can try this safely.
What to know before you search
Eligibility depends on the specific leukemia type (AML, ALL, CML, CLL), genetic and chromosomal features of the disease, age, prior treatments, and overall health.
What types of trials are currently open
- Treatment trials — Testing new drugs or drug combinations in people with leukemia to see if they work better than current options.
- CAR-T and cell therapy trials — Studies of engineered immune cells designed to attack leukemia cells, often for relapsed disease.
- Stem cell transplant trials — Testing newer transplant approaches that aim to be safer and effective for more patients.
- Maintenance trials — Testing treatments given during remission to lower the chance the leukemia comes back.
- Supportive care trials — Testing ways to reduce infections and other complications during intensive treatment.
Recently added Leukemia trials
Dose Finding Study to Evaluate the Safety of BSB-2002 in Relapsed or Refractory Acute Myeloid Leukemia (AML) Patients With NPM1 Mutation
The goal of this clinical trial is to test BSB-2002 which is a new type of cellular therapy to treat blood cancer (AML). It will evaluate the safety of BSB-2002 and also determine whether it works to prevent relapse of your cancer.
Cord Blood Transplantation in Children and Young Adults With Blood Cancer
The purpose of this study is to find out whether Cord Blood Transplantation/CBT as the first or second transplant is an effective treatment for children and young adults with blood cancer.
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