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Clinical Trials

Taking part in a clinical trial may be the best treatment choice for some acute lymphoblastic leukemia (ALL) patients. Clinical trials are under way for patients at every treatment stage and for patients in remission. Today's standard treatments for cancer are based on earlier clinical trials. The Leukemia & Lymphoma Society continues to invest funds in ALL research.

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Current ALL Research and Clinical Trials

New approaches are under study in clinical trials for acute lymphoblastic leukemia (ALL) treatment. Many of the trials are being supported by LLS research programs and hold the promise of increasing the rate of remission and finding a cure for ALL.

ALL patients are encouraged to explore participating in a clinical trial. Clinical trials test new drugs and treatments before they are approved by the Food and Drug Administration (FDA) as standard treatments. Every new drug or treatment regimen goes through a series of phases of clinical trials before it becomes part of standard therapy. Clinical trials are carefully designed and reviewed by clinicians and researchers to ensure as much safety and scientific accuracy as possible. Participation in a carefully conducted clinical trial may be the best available therapy. Clinical trials are designed to be accurate and very safe. There are clinical trials for newly diagnosed patients, for patients with advanced disease, and for patients who are either resistant to their medications or are unable to tolerate them.


Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here


Research Approaches

Scientific research is being done to learn more about ALL: how best to treat it and how to provide the best care to people diagnosed with this disease.

Genetics of Leukemia. Researchers are studying how changes (mutations) in the DNA of normal bone marrow cells can cause them to develop into leukemia cells. There is a need to identify these genetic variations in order to customize treatment options based on the genetic characteristics of the leukemia cells. Newer techniques in gene sequencing have revealed previously unknown mutations that may be involved in the development of ALL. This information will help researchers develop new targeted therapies, tailored to specific disease characteristics in each patient.

New Drugs and Treatment Regimens. Researchers are working to develop safer and more effective treatments for ALL. New treatments are needed for high-risk patients and for patients with relapsed and refractory disease. While ALL treatment can be very effective for most children, cure rates are lower for adults. Researchers are studying new drugs, as well as the use of existing drugs in different doses and with different methods of administration for adults with ALL. Treatment approaches under investigation include:

  • Chemotherapy. Chemotherapy is still the main treatment for most cases of ALL. Researchers are now studying different combinations of chemotherapy drugs to determine which is most effective while also limiting side effects. And they are continuing to modify and reformulate traditional chemotherapy drugs to improve overall survival. They are also evaluating combinations of chemotherapy drugs with newer targeted therapies.
  • Stem cell transplantation. Researchers continue to study stem cell transplantation to try to increase cure rates for ALL patients, reduce complications and determine which patients are most likely to benefit from this treatment.
  • Targeted therapy. This is a type of treatment that uses drugs or other substances to block the action of certain enzymes, proteins or other molecules involved in the growth and survival of cancer cells, while causing less harm to healthy cells. Targeted agents under study include:
    • Proteasome inhibitors, such as bortezomib (Velcade®) and carfilzomib (Kyprolis®)
    • Janus Kinase (JAK) JAK1 and JAK2 inhibitors such as ruxolitinib (Jakafi®)
  • Immunotherapy. Tisagenlecleucel (Kymriah®)—Chimeric antigen receptor (CAR) T-cell therapy is a type of immunotherapy that engineers a patient’s own immune cells to recognize and attack cancer cells. Researchers are conducting research to see whether these treatments are effective in adults

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