February 2023

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Advances in Childhood Cancer

Making Personalized Treatments a Reality

Children often get bumps, bruises, sneezes, and sniffles. It doesn’t seem like they could get something as scary as cancer. But they can.

Every year, more than 10,000 kids in the U.S. will get a diagnosis of cancer. Some cases are caused by changes in certain genesStretches of DNA you inherit from your parents that define features, like your risk for certain diseases. that are passed from parents to their children. But these are rare. The causes of most cases of cancer in kids aren’t known.

Advances made over the last few decades have led to more children surviving cancer than ever before. And a better understanding of the disease means that more children with cancer are getting personalized treatments.

Treating Childhood Cancer

Cancer isn’t just one disease. There are many different kinds. But all cancers start when some of the body’s cells begin to grow uncontrollably. Sometimes, these cells spread to other parts of the body.

“Leukemias are the most common types of cancer that we see in children,” says Dr. Nita Seibel, a childhood cancer specialist at NIH. These are cancers that start in certain types of blood cells.

Next most common are cancers of the brain, nerves, and spinal cord. Lymphoma—another type of blood cancer—also occurs in kids and teens. Children may be affected by tumors of the eyes, adrenal gland, kidneys, muscles, and bones.

Common treatments for childhood cancers include surgery, chemotherapy, and radiation therapy. These can be used in combination or alone.

Chemotherapy and radiation therapy work by killing fast-growing cancer cells. But these treatments can also damage normal cells. This damage can cause side effects like infection, hair loss, and nausea. But these often go away. Other side effects, like hearing loss, may not go away. And some can appear after treatment stops.

Some kids may need other treatments, such as immunotherapyTreatments that train your body’s disease defense system to fight cancer. or a stem-cell transplant. Different types of cancer need different treatments.

Until recently, it was common for all kids with the same type of cancer to receive the same treatments, explains Dr. Will Parsons, a child cancer specialist at Texas Children’s Hospital. But the discovery of certain gene changes, called mutations, in cancer cells has started to change this. These mutations cause cancer cells to grow out of control.

A new type of treatment called targeted drugs can block the effects of these harmful mutations. They cause cancer cells to stop growing, or to die, without causing as much damage to normal cells. Targeted drugs often have fewer side effects than chemotherapy.

“Targeted drugs have gone from a theory to a reality over the past decade,” says Parsons. This breakthrough has been aided by faster and cheaper tests to pinpoint cancer mutations, he explains.

Researchers are testing ways to expand gene mutation screening for kids with cancer. One ongoing study is called Pediatric MATCH. The study is trying to match the mutation found in a child’s tumor with a drug targeted for the specific gene mutation, instead of for a specific type of cancer.

Personalizing Treatment

CAR T cells are the newest, most personalized cancer treatment. To make CAR T cells, immune systemA collection of specialized cells and organs that protect your body against invading germs and other microscopic threats. cells called T cells are collected from the blood of a person with cancer. The T cells are then modified in the lab so they can find and kill cancer cells. Millions of these designer cells are then grown and infused back into the patient.

“CAR T cells have made a big difference for kids whose leukemia has come back after treatment,” says Seibel.

“Right now, they’re reserved for patients whose disease has come back, or who have a high risk of relapse,” says Dr. Nirali Shah, a child cancer specialist at NIH. One reason for that is that standard treatments usually work very well. Another is that CAR T cells are expensive to make because they’re custom made every time.

Researchers are testing so-called off-the-shelf CAR T cells, which could be made in big batches and used for many kids. This could reduce the cost and time needed to produce them. “But we need to know if their effect is going to last as long, and if the side effects are going to differ,” she says. “So they need to be further evaluated in clinical trials.”

CAR T cells and many other new treatments for children with cancer are being tested in studies called clinical trials. If your child has cancer, talk with your health care team. They can help you learn about all the treatment options.

When Less is More

Personalizing treatment can also mean adjusting the amount of therapy a child gets. That often means a lower risk of side effects.

For some cancer types, researchers can predict which tumors have a low risk of coming back. Kids at low risk of relapse may now be able to get lower doses of chemotherapy or radiation therapy. They may even be able to skip some toxic treatments entirely.

Reducing the amount of treatment can make a big difference in quality of life both during and after treatment. But no matter what treatments are needed, many side effects can be managed. For example, drugs can manage symptoms caused by chemotherapy, like nausea.

Some cancer treatments may have what are called late effects. These are side effects that can occur months or even years after treatment. They include trouble with learning and development, damage to the heart, and increased risk of other cancers later in life. This is another reason why researchers want to reduce the amount of treatment whenever possible.

For some treatments, late effects are still unavoidable. So personalized care after treatment, called survivorship care, is vital for any child who’s been treated for cancer. Survivorship care plans help guide the type of care kids will need to lead their healthiest life possible.

“It’s part of the whole package of cancer treatment,” says Seibel. “We want to make sure that kids have the highest quality of life possible as survivors.”

Find advice and information about supporting a child with cancer.