PRECISION MEDICINE

What is Precision Medicine? Scientists are getting more precise when they diagnose and treat cancer! By looking at the DNA of tumors and comparing it to kids’ healthy cells, they can pinpoint the genetic mutations that caused their cancer in the first place. Targeting these mutations with the right drugs gives doctors the power to fight cancer more accurately — and avoid harming healthy cells. This means kids will experience fewer side effects than they would from chemotherapy.

How are we making a difference? Precision medicine is unlocking the secrets of cancer cell biology and some of the causes behind how kids get cancer in the first place. The Department of Pediatrics at MSK is home to a new and game-changing Pediatric Translational Medicine Program (PTMP) — and Kids Walk is driving its progress by providing important funding. The Program promises to deliver targeted therapies to more kids battling cancer than ever before. By looking at the DNA of each patient’s tumors for answers, researchers expect to make increasing strides in battling all pediatric cancers.

What’s the latest? MSK treats more kids with cancer than any other hospital in the United States, and its researchers are now offering to sequence the genomes of every one of them through the PTMP. The Program has the power to identify more precise treatments for kids based on their DNA because of MSK-IMPACT, a system that identifies the 468 mutations (changes to the DNA) that are known to cause cancer. Kids Walk funding gives doctors the tools they need to search through all of those genes to find the cancer-causing mutations and match patients with the most effective treatments possible.

These personalized therapies may be more effective and have fewer side effects — and that’s the goal! Kids Walk’s funding for the precision oncology program will give doctors the ability to better understand and target pediatric cancers on a DNA level. Today, researchers are expanding the analysis so they can comprehensively assess all 25,000 genes in the human body. These methods will likely benefit all patients with cancer — not just kids, but adults too.

NEUROBLASTOMA

What is Neuroblastoma? Neuroblastoma is a nerve-tissue cancer that is most common in young children. Often, neuroblastoma spreads throughout the body. If it spreads to the brain this has historically been very hard to treat.

How are we making a difference? MSK treats more kids with neuroblastoma than any other hospital in the world — and researchers are making huge strides in treating this disease. The Department of Pediatrics has developed new treatments to address the worst forms of neuroblastoma, specifically where disease has spread to the brain. These new therapies have improved the survival rate from below five percent up to nearly 50 percent.

What’s the latest? Funding from Kids Walk helped researchers at MSK create and test a new drug called omburtamab that was recently granted Breakthrough Therapy Designation by the FDA. The treatment was so effective for some kids who otherwise had no chance of survival that the FDA prioritized its approval and made it available as soon as possible to patients around the world. In 2018, another drug, naxitamab, was also granted Breakthrough Therapy Designation by the FDA. These two important new drugs provide real hope — and possibly even a cure — to kids with advanced neuroblastoma.

LEUKEMIA

What is Leukemia? Leukemia is the most common type of pediatric cancer. It begins in the blood, making white blood cells — which normally help your body fight infection — grow out of control and change into cancer cells.

How are we making a difference? Doctors at MSK pioneered new chemotherapy treatments that have dramatically increased long-term survival for kids and young adults with leukemia.

What’s the latest? Kids Walk continues to fund two promising clinical trials that aim to keep chemotherapy working for the long-term. When chemotherapy stops working, kids may need a bone marrow transplant to replace cancer blood cells with healthy ones. Kids Walk is supporting efforts to improve how MSK carries out those bone marrow transplants for kids.

Another approach to treating leukemia is using CAR T cells — a recently FDA-approved immunotherapy approach that uses a patient’s own infection-fighting T cells against cancer. First, doctors remove the patient’s T cells, then insert new DNA into the cells and put them back into the patient as CAR T cells. The CAR T cells can target and attack cancer, and are currently used to treat a form of childhood leukemia at MSK. CAR T is especially important for kids because it’s a very powerful treatment, offering patients hope for a cure when chemotherapy isn’t working.

PEDIATRIC FAST-TRACK

What is Pediatric Fast-Track? It’s a three-step plan designed to speed up the clinical trial process and make life-changing treatment options accessible to kids with cancer as soon as possible.

How is it making a difference? First, MSK is working to expand access to drugs currently only available to adults by lowering the age of clinical trials to 12 years old. Next, newly discovered and promising drugs that work successfully on adults could be moved into trials for kids faster than ever. Lastly, very sick pediatric patients could have access to treatments that were originally created for other kinds of cancer, giving them a new option they didn’t have before.

What’s the latest? This leading-edge initiative will give researchers in the Department of Pediatrics the resources they need to quickly and safely adapt new cancer therapies for kids. Fewer than 10% of all cancer drugs are approved for kids with cancer, so this could transform the drug approval process for pediatric patients around the world.