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.
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Here are examples (and some descriptions) of specific agents that are under study in clinical trials for ALL.
- Bortezomib (Velcade®)—This drug, approved to treat myeloma and mantle cell lymphoma, is now being studied in combination with other standard chemotherapy drugs for the treatment of relapsed or refractory ALL. It is also being studied for treating newly diagnosed pediatric patients with T-cell ALL.
- Clofarabine (Clolar®)—Already approved to treat pediatric ALL, clofarabine is now showing promising results in studies of adults with ALL. It is also being studied in combination with other drugs such as mitoxantrone in clinical trials for the treatment of children whose ALL is relapsed or refractory.
Janus Kinase (JAK) 1 and 2 Inhibitor
- Ruxolitinib (Jakafi®)—Already approved to treat myelofibrosis and polycythemia vera patients, this drug is being studied in clinical trials in the treatment of pediatric refractory and relapsed ALL. It is also being studied in combination with several chemotherapy drugs in the treatment of children with Philadelphia-like (Ph-like) ALL and CRLF2 and JAK alterations.
Special Chemotherapy Combination
- Augmented Hyper-CVAD—The hyper-CVAD (cyclophosphamide, vincristine, doxorubicin (Adriamycin®) and dexamethasone) combination is a wellestablished treatment regimen for adult ALL. The augmented hyper-CVAD formulation was designed in 2011 and it includes intensified doses of vincristine, and dexamethasone along with asparaginase. Researchers are studying the efficacy of this combination for ALL treatment with other drugs including decitabine (Dacogen®).
- Rituximab (Rituxan®)—Rituximab is a monoclonal antibody that is being studied to treat certain patients with B-cell ALL. It binds to CD20, a protein that is found on the surface of healthy B cells and on the lymphoblastic B cells of approximately one half of adults with B-cell ALL. When rituximab binds to CD20, it signals the cell to die. Rituximab is not used alone to treat ALL, but it is added to a chemotherapy regimen. Some studies have shown that the addition of rituximab to standard chemotherapy improved survival among adults with CD20-postive ALL. Side effects may include infections, chills, fatigue, body aches and low blood cell counts.
- Tisagenlecleucel (Kymriah®)—Chimeric antigen receptor (CAR) T-cell therapy is a type of immunotherapy that consists of engineering patients’ own immune cells to first recognize and then attack cancer cells. Tisagenlecleucel (Kymriah®) is FDA approved for patients up to age 25 with relapse or refractory B-cell ALL. It is the result of reprogramming the patient’s own T cells to locate and destroy cells that have the CD19 protein on their surface. In some cases, the leukemia cells do not have CD19 on the surface. Such cells can evade tisagenlecleucel and multiply. A research goal is to find other markers on leukemia cells that can be targeted along with the CD19, such as CD22.
For more information on this type of therapy, see the LLS booklet Chimeric Antigen Receptor (CAR) T-Cell Therapy Facts.
For information about the drugs listed on this page, visit Drug Listings.
- Download or order The Leukemia & Lymphoma Society's free booklet, Acute Lymphoblastic Leukemia.