Leukemia Treatment For Children

Leukemia Treatment For Children

Healthcare Tech Outlook | Monday, July 25, 2022

Now, researchers are centering on targeted drugs and immunotherapies to treat leukemia in children.

FREMONT, CA: Regular leukemia treatments for children have been chemotherapy, radiation therapy, and stem-cell transplant. Unfortunately, despite significant advancements in survival for children with some types of leukemia, some treatments don't often work.

Additionally, some children later feel a relapse of their disease. Others stay with the side effects of chemotherapy and radiation therapy for the rest of their lives, underscoring the requirement for less harmful treatments.

Now researchers are centering on targeted drugs and immunotherapies to treat leukemia in children. Newer chemotherapy drugs are also being tried.

Targeted Therapies

Targeted therapies that have been authorized or are being studied for children with leukemia incorporate:

• Dasatinib (Sprycel) is allowed to treat children with CML and those with ALL(Acute Lymphoblastic Leukemia).

• The acceptance is for children whose cancer cells possess the Philadelphia chromosome.

• Sorafenib (Nexavar) is being tested in addition to standard chemotherapy for children with AML whose leukemia has changes in a gene called FLT3. Other drug trials testing drugs that target FLT3 more specifically than sorafenib will be tested in practices that are being planned.

• larotrectinib (Vitrakvi), tested in children with leukemia, has a specific change in a gene called NTRK.

More possible targets for treating childhood cancers are discovered every year. With these objectives, many new drugs that could be used to treat cancers are being tested using the Pediatric Preclinical In Vivo Testing Consortium (PIVOT)Exit Disclaimer.


CAR T-cell therapy has generated great excitement for treating children with relapsed ALL. One CAR T-cell therapy, tisagenlecleucel (Kymriah), was sanctioned in 2017 for some children with relapsed ALL.

Researchers continue to address the remaining challenges regarding the application of CAR T-cell therapy in children with leukemia:

• At times, leukemia can turn resistant to tisagenlecleucel. Researchers in NCI's Pediatric Oncology Branch have advanced CAR T cells that target leukemia cells differently. An ongoing clinical trial tests whether combining these two types of CAR T cells can offer longer-lasting remissions.

• CAR T cells are currently only accepted for use in leukemia that has relapsed or proved resistant to standard treatment. A clinical trial from COG is now experimenting with tisagenlecleucel as part of first-line therapy in children with ALL at great risk of relapse.

• More research is necessary to understand which children who receive CAR T cells are at high risk of developing resistance to treatment. Researchers also plan to test whether strategies like combining CAR T-cell therapy with other immunotherapies may support and prevent resistance from developing.

• Other research in NCI's Pediatric Oncology Branch and other institutions focuses on creating CAR T-cell therapies that operate on children with other types of childhood leukemia, like AML. Various clinical trials of these treatments, comprising one led by NCI researchers, are now underway.

Two other drugs that employ the body's immune system to fight cancer have shown promise for children with leukemia:

• A drug called Blinatumomab (Blincyto) attaches to T cells and cancer cells. By bringing these cells close together, the drug helps the T cells kill the cancer cells. The FDA has approved Blinatumomab for children with ALL who have relapsed after initial treatment. In clinical tests, the drug was proven more effective than chemotherapy in treating ALL that has relapsed in children and young adults.

• A drug named inotuzumab ozogamicin (Besponsa) is being tested in children with relapsed B-cell ALL. This drug comprises an antibody that can bind to cancer cells linked to a drug that can kill those cells.


Including targeted therapies and immunotherapies, researchers are also functioning to develop new chemotherapy drugs for leukemia and discover better ways to use existing drugs. For example, in 2018, a large clinical trial showed that adding nelarabine (Arranon) to standard chemotherapy enhances survival for children and young adults newly diagnosed with T-cell ALL.

Other drugs are being examined that may make standard chemotherapy drugs more effective. These drugs incorporate venetoclax, which has been accepted for older adults with some leukemia and is now being tested in children.


Children's developing brains & bodies can be specifically sensitive to the harmful effects of cancer treatment. Since many children treated for cancer go on to live long lives, they may be dealing with these late effects for years to come.

The NCI-funded Childhood Cancer Survivor Study, continuing since 1994, tracks the long-term harmful effects of treatments for childhood cancer and studies ways to reduce these effects. NCI also funds research into addressing ways to support cancer survivors to cope with and manage health issues stemming from cancer treatment and alter existing treatment regimens to make them less toxic in the long term.

For example, a recent study found that radiation therapy to prevent cancer from returning to the brain is likely unnecessary in children with ALL. In addition, the study found that radiation can even be omitted for children at the highest risk of cancer returning, reducing the risk of future problems with thinking and memory, hormone dysfunction, and other adverse effects of radiation on the brain.

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