Pediatric Translational Medicine Service (PTMS)

Pediatric Translational Medicine Service (PTMS)


Our Goal: A Cure for Every Child with Cancer

There has been tremendous progress in the efforts to control and cure children’s cancers over the past four decades. Yet still, some 20 to 30 percent of kids with cancer don’t respond to known treatments. Just recently we have gained the tools to better understand why.

What is translational medicine?

How Can We Help Your Child?

To learn more about the PTMS, call us to make an appointment. We’ll be happy to explain our program and how it may help your child.


Translational medicine carries medical findings “from bench to bedside.” Our researchers work with our doctors and other MSK Kids specialists with the goal of using their discoveries to create new treatments and procedures.

Through the Pediatric Translational Medicine Service (PTMS) at Memorial Sloan Kettering, every child and young adult with cancer has their tumor closely studied for genetic mutations. This information may provide valuable clues that doctors and researchers can use to choose the most effective therapy for each patient. It also allows them to start new research projects and develop lifesaving drugs. At MSK Kids, our goal is to provide the most-effective therapy from the start of your child’s treatment and reduce the number of kids who don’t respond to treatment to 0.

At MSK, It’s Routine

The PTMS is a precision medicine effort that unites experts from multiple MSK departments, including Pediatrics, Pathology, and Clinical Genetics. We pool our expertise, ideas, and passions to decipher the roots of cancer for every child cared for at MSK Kids. The PTMS team performs genetic testing of your child’s disease as a routine part of their care.

Rihanna's Story of Treatment on a Clinical Trial with Larotrectinib
Meet Rihanna, who was on a clinical trial with larotrectinib.

Cancer Genetics 101

The growth and development of every cancer is driven by mutations, or changes, in DNA (the genetic blueprint that tells cells how to behave). Some cancer mutations are inherited. That means they are passed down from generation to generation, from parent to child. People who inherit cancer mutations are born with them. But most cancer mutations happen after a person is born. The mutation makes a normal cell turn into a cancer cell. Cancer cells don’t know when to stop growing and multiplying. The genetic features of a cancer are called its profile. The tests we use to draw that profile are called genetic sequencing.

Your Child’s Cancer Profile

At MSK Kids, we use state-of-the-art methods to profile every child’s cancer in ways that no other cancer center can match. We use the latest genetic sequencing tests to analyze the cancer’s DNA from a sample of the tumor tissue, blood, or bone marrow, looking for mutations that cause cancer and make it grow. Here’s how it works:

  • We collect those samples during your child’s regular care. No extra procedures are needed to obtain them.
  • Our team explains everything to you and gets your consent before we do genetic sequencing. We make sure that you understand what will happen and how the information will be used.
  • Once we have the results of the sequencing tests, we’ll explain what they mean to you and describe the next steps in your child’s care.

What’s Next?

Once we know which mutations are present in your child’s cancer, here’s what may happen:

  • Choosing the best treatment. We’ll let you know if there are drugs available that target those mutations.
  • Joining a clinical trial. Your child may be able to participate in a clinical trial of an innovative therapy. These important studies enable your child to take advantage of promising treatments while they are still early in the evaluation process.
  • Talking with a genetic counselor. In some patients, the test results show that there may be an inherited cancer mutation that other family members may have. If that happens, we can refer you to the MSK Kids Clinical Genetics Service for follow-up.
  • Sparking ideas. Your child’s test results can also provide us with information we could use to launch new clinical trials and other research projects that will help other children with cancer in the future. We want to learn as much as we can from every tumor sample we have. We have a strong translational research program.

Whatever the genetic sequencing results show, know this: The MSK Kids team is with you every step of the way. We use every bit of information we gather to customize the most effective care plan for your child.

To learn more about the PTMS and how our team members work together to improve the future of children with cancer, click on any of the program’s components below.

Precision Tumor Testing

For decades, we have treated tumors based on the way they look under a microscope. Now we know that to treat cancer most effectively, we need to dive deeper than that. Two tumors may look alike under a microscope, but their growth may be driven by completely different mutations, so they may respond differently to treatment. On the other hand, tumors may begin in different parts of the body — a brain tumor and a sarcoma, for example — but have the same mutations and respond to treatment with the same targeted drug. This is why we conduct precision genetic testing. It helps us identify the most effective therapies while eliminating those we know won’t work. MSK is a leader in precision medicine testing. We offer multiple unique clinical-testing platforms to study pediatric cancer. Key to this effort is MSK-IMPACT™. This technology was created at MSK and can find hundreds of genetic mutations related to cancer.

Hereditary Cancer Testing

More than 15 percent of our pediatric patients were born with a mutation that contributed to their tumor. If your child’s genetic profile shows there is an inherited cancer mutation, the experts in MSK Kids’ Clinical Genetics Service are here to help. Our genetic counselors and geneticists can meet with you and any family members to help you understand if anyone else in your family may have an increased risk of cancer. We will offer genetic testing to any of your family members who are interested. It can be challenging to understand genetic testing results. Our team can help you make sense of the results and guide you toward steps that may reduce your risk of cancer or increase the chance of finding it earlier.

Learn more about inherited cancer mutations and how we address them.

Trials and Treatments

The PTMS is making it possible to bring the latest treatment options to young patients sooner. Your child may be able to have an investigational treatment through a clinical trial. These novel drugs frequently target mutations that we found using our precision medicine approach. These studies are also vital for making progress against cancer and have led to dramatic achievements in children’s cancer care. Some children’s cancers are driven by mutations present in adult cancers. This means we may be able to offer more clinical trials to children at MSK Kids based on the results of their tumor’s genetic testing.

Learn more about pediatric clinical trials.

Science and Innovation

The PTMS works directly with MSK Kids researchers to bridge state-of-the-art science with the latest clinical studies. For example, we can create models of a patient’s tumor in the laboratory using their own cells or tumor tissue. We then test thousands of drugs against those models. We can use the results of those tests to develop drug studies for children with similar types of cancer. The PTMS is also working with researchers and others to perform a type of genetic testing called whole-genome sequencing for certain tumor types. Whole-genome sequencing is a very broad type of genetic testing that allows us to evaluate all the DNA in a tumor. Our goal is to use the detailed information generated from whole-genome sequencing to design new approaches in the clinic.