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What’s New in Wilms Tumor Research?

Over the past few decades, research into Wilms tumors has led to many advances and much higher cure rates for this type of cancer. Still, not all children are cured., and even those who are cured might still have long-term side effects from treatment, so more research is needed.

In the United States, much of the research on Wilms tumors is coordinated by the (COG), whose main goal is to improve the treatment and quality of life for children with Wilms tumors and other types of cancer. COG is a large group of doctors, nurses, scientists, and other health professionals whose hard work has already saved the lives of many children with Wilms tumors.

The information below is an overview of the latest research into Wilms tumors, including genetics and treatments.

Research into the genetics of Wilms tumors

Researchers continue to learn how changes in certain genes inside kidney cells cause Wilms tumors and affect how aggressive these tumors are likely to be.

As doctors have learned how better to treat Wilms tumors, they’ve looked for ways to determine which children might need more intense treatment to be cured, and which children might be spared from intense treatment (and its possible side effects).

For example, studies have shown that Wilms tumors with cells that have certain changes on chromosomes 1, 11, or 16 seem to be more likely to come back after treatment. Doctors are now studying if children with such tumors might benefit from more intense treatment.

Researchers are also studying the gene changes that seem to cause Wilms tumor cells to grow and spread. This may lead to treatments that specifically target these changes.

Research into the treatment of Wilms tumors

Researchers continue to study ways to improve treatment for children with Wilms tumors.

Using less treatment when possible

Earlier studies found treatments that were very effective in curing most Wilms tumors, especially those with favorable histology (see What Are Wilms Tumors?). Current clinical trials are studying ways to cure these cancers while reducing side effects as much as possible.

For example:

  • Studies are looking at whether young children who have Wilms tumors with very favorable features need any treatment other than surgery.
  • Research has suggested that some children with tumors that have spread to only one spot in the lung might not need radiation therapy to the lungs if the spot goes away with chemotherapy. Radiation to the lung can cause long-term side effects.

Newer approaches for tumors that are harder to treat

The outlook for some children with Wilms tumors, such as those with anaplastic histology, is not as good. Doctors are looking for better treatments for these children.

Different chemotherapy (chemo) drugs or a combination of drugs that include topotecan, irinotecan, or ifosfamide are now being tested.

Some studies are looking at stem cell transplants (also known as bone marrow transplants), which let doctors give higher doses of chemo than the body normally could tolerate. This approach might help treat tumors that are not responding to standard treatments or that would otherwise have a poor outlook.

As researchers have learned more about the gene changes in cancer cells, they have developed newer drugs that target these changes. Targeted drugs work differently from standard chemo drugs. They sometimes work when chemo drugs don’t, and they often have different (and less severe) side effects. Targeted therapies have become standard treatments for some kinds of cancers.

For children with Wilms tumors who might not be cured with current treatments, some clinical trials are now testing the tumor cells for certain gene changes. If one of these changes is found, treating the tumor with a drug that can target the change might be helpful.

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Developed by the American Cancer Society medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).

Dix DB, Seibel NL, Chi YY, et al. Treatment of stage IV favorable histology Wilms tumor with lung metastases: A report from the Children's Oncology Group AREN0533 study. J Clin Oncol. 2018;36(16):1564-1570.

Fernandez CV, Geller JI, Ehrlich PF, et al. Chapter 24: Renal Tumors. In: Blaney SM, Adamson PC, Helman LJ, eds. Pizzo and Poplack’s Principles and Practice of Pediatric Oncology. 8th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2021.

National Cancer Institute. Wilms Tumor and Other Childhood Kidney Tumors Treatment (PDQ?)–Health Professional Version. 2024. Accessed at https://www.cancer.gov/types/kidney/hp/wilms-treatment-pdq on November 21, 2024.

Smith V, Chintagumpala M. Treatment and prognosis of Wilms tumor. UpToDate. 2024. Accessed at https://www.uptodate.com/contents/treatment-and-prognosis-of-wilms-tumor on November 21, 2024.

 

Last Revised: January 21, 2025

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