Acute lymphoblastic leukemia (ALL) is a fast-growing blood cancer most common in children, though adults can get it too. Modern chemotherapy cures about 90% of children with ALL. Adults and people whose ALL returns after treatment face tougher odds, which is where newer therapies come in.
What's actually going on in research
Trials are testing CAR T-cell therapy (tisagenlecleucel approved for relapsed ALL in children and young adults), bispecific antibodies like blinatumomab that redirect immune cells to cancer, and targeted drugs for specific genetic subtypes. Researchers are also working to reduce treatment intensity for lower-risk patients and to combine newer drugs with chemotherapy for better outcomes in high-risk groups.
CAR T-cell therapy
Engineered immune cells have produced remissions in people whose ALL came back after chemotherapy. Trials are now testing CAR T earlier in treatment and in combination with other drugs.
Bispecific antibodies
Blinatumomab connects a patient's T cells directly to leukemia cells, forcing an immune attack. Studies are testing whether adding it to chemotherapy can prevent relapse in newly diagnosed ALL.
Targeted therapies
Drugs like imatinib for Philadelphia chromosome-positive ALL and venetoclax for certain genetic subtypes are being tested in combinations. The goal is to match treatment to each person's leukemia genetics.
What to know before you search
Eligibility typically depends on age, disease genetics (like Philadelphia chromosome status), treatment history, and whether measurable leukemia remains after initial treatment.
What types of trials are currently open
- Treatment trials — Testing combinations of chemotherapy with newer drugs like bispecific antibodies or tyrosine kinase inhibitors to improve cure rates.
- CAR T trials — Studies of engineered immune cells, often for people whose ALL returned after treatment or who have high-risk disease.
- Minimal residual disease trials — Testing whether highly sensitive tests can guide treatment decisions, allowing some people to stop therapy earlier or intensify sooner if leukemia persists.
- Reduced-intensity trials — Studies testing whether lower-risk patients can get less chemotherapy and avoid long-term side effects while maintaining high cure rates.
- Transplant trials — Studies comparing stem cell transplant timing and newer conditioning regimens for high-risk or relapsed ALL.
Recently added Acute Lymphoblastic Leukemia trials
CAYA Cancer Prospective Cohort Study
Cancer is a leading cause of illness and death among children, adolescents, and young adults(CAYAs), especially in low- and middle-income countries(LMICs), where access to timely diagnosis and treatment is often limited. As a result, patients in these settings may experience higher rates of treatment complications, interruptions, and poorer outcomes compared with those in high-income countries (HICs). This is a prospective, multicenter observational study that will follow children, adolescents, and young adults(CAYAs) with cancer who are receiving routine care at participating hospitals in low - and middle - income countries(LMICs). The study does not involve experimental treatments or changes to standard medical care. Information will be collected from medical records and from questionnaires that address access to care and social factors affecting treatment. By describing treatment outcomes and the challenges patients and families face during cancer care, this study aims to provide data that can help inform future efforts to improve access to care and cancer outcomes in resource-limited settings.
First-in-human Study of a New Treatment (4A10) for Patients With Relapsed or Hard-to-treat Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma, Focused on Safety and How the Drug Behaves in the Body and Early Signs of Effect.
ALT-101 is a first-in-human Phase 1 clinical trial testing a new antibody drug called 4A10 in patients with relapsed or hard-to-treat acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma. 4A10 is a targeted therapy designed to recognize and attach to a specific protein (CD127) found on leukemia cells. Once it binds, it works in two ways: it blocks growth signals that help cancer cells survive, and it helps the immune system find and destroy those cancer cells. In this study, patients receive 4A10 through an intravenous (IV) infusion once a week. The main goal of the trial is to find out if the drug is safe, what dose can be given, and how the body processes it. Researchers will also look for early signs that the treatment may be working. The study starts with small groups of patients receiving increasing doses to carefully monitor safety. Each patient is closely observed during the first treatment cycle (about 4-6 weeks) to watch for side effects. If the treatment is helping and is well tolerated, patients may continue treatment for up to six cycles. Overall, this study is an early step in testing a new, targeted immune-based therapy for difficult-to-treat blood cancers.
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