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Clinical Trials

When it comes to finding the right treatment for your child's cancer, a clinical trial may be an option. Your child will have access to new or improved therapies under study and not yet on the market. Discuss with your child's doctor the possibility of participating in a clinical trial, where treatment is administered in a safe, closely monitored environment.

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Research Approaches

Genetics of Leukemia. Researchers are studying how changes (mutations) to the DNA inside normal bone marrow cells can cause them to develop into leukemia cells. There is a need to identify these genetic variations and 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 that are tailored to specific disease characteristics in each child.

New Drugs and Treatment Regimens. Researchers are working to develop effective and safer treatments for ALL. New treatments are needed for children with high-risk ALL and children with relapsed and refractory disease. ALL treatment can be very effective for most children, but it does not cure all children who have this disease. Researchers are studying new drugs for ALL, as well as the use of existing drugs given in different doses and with different methods of administration. 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 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®)
    • Tyrosine kinase inhibitors (TKIs), such as ruxolitinib (Jakafi®)
  • Immunotherapy. Immunotherapy treatments use substances that can stimulate and/or suppress the immune system to help the body fight cancer. Agents under study include:
    • Bispecific T-cell engager (BiTE) antibodies, such as blinatumobab (Blincyto®)
    • Antibody drug conjugates, such as inotuzumab ozogamicin (Besponsa®)
    • Chimeric antigen receptor (CAR) T-cell therapy, such as tisagenlecleucel (Kymriah®)


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