17:16 PM

5 Things You Should Know about Childhood Cancer Research

Dr. Alan Gamis, Associate Division Director, Section of Oncology

Childhood cancer research plays a critically important role when it comes to treating and curing the disease, but the reality is very few cancer therapies are developed for kids. (Only 4% of the National Institute of Health’s budget is dedicated to children’s cancer research.) Most cancer therapies are first discovered to be beneficial in adults and then used for treatment in children.

That’s in large part because childhood cancer is considered rare. It’s estimated 1 in 300 children will develop cancer before their 16th birthday. Now compare that to 1 in 3 adults that will be diagnosed with cancer (includes skin cancer and easy, curable cancers) and you can see why the pharmaceutical industry develops treatment for adults rather than children. Simply put, there’s more incentive and opportunity for pharma to recoup all the dollars put into development of the drug.

However, pediatric research is imperative to future generations and here’s why.

Research Helps Identify the Most-Effective Treatments

Since most therapies are designed for adults, we have to find ways to make treatments work for kids. Research helps us better define and delineate the characteristics of the cancers that develop in children to determine if there are common factors in adult cancer – even if it’s a different type of cancer.

For instance, it was discovered that 10% of children diagnosed with a nerve cancer called neuroblastoma have the same gene mutations found in adult lung cancer. With this discovery, we can now identify which drugs currently being used to treat lung cancer can be most effective in treating kids with neuroblastoma.

It’s this kind of research that will change the way we look at cancer. Instead of calling it lung cancer or neuroblastoma, I would argue we should start calling cancer by the gene. Therefore, treatment can target the cause and not the organ that it happened to occur in.

Genomic Sequencing Leads to Better Treatment

Genomic sequencing is done on every child that is diagnosed with cancer at Children’s Mercy. Because of research, we are discovering so many new things about mutations that cause cancer. Not just one mutation, but a sequence of mutations. It’s these discoveries that lead to better refinement of identifying new treatment.

One outcome from all this research is that I believe cancer will one day be treated like a chronic disease rather than a deadly disease. Just as people take a pill to keep high blood pressure under control, the same will be true for cancer. In some instances it’s already happening.

Gleevec® is the first targeted therapy to treat byproducts of the mutations that are abnormal proteins called oncoproteins. The drug, taken daily, prevents the cells from dividing, therefore preventing cancer from growing out of control. Research shows that Gleevec’s continued use can help prevent leukemias with the specific mutation from returning.

Bench-to-Bedside Research is Critical

For new treatments to be discovered, it’s critical to have researchers in the lab working alongside clinical researchers treating the patients. We can tease apart the molecular and identify genomic steps of getting to a cancer or ways to prevent the side effects, but it's worthless if we can't translate it to the bedside.

This bench-to-beside process is even more necessary when it comes to developing an extremely personalized approach to treatment. In the future, we envision the ability to create a targeted agent for each individual child and their particular cancer.

While we’re not there yet, we’ve made a lot of progress in the right direction. Children’s Mercy has been on the cutting-edge of using a groundbreaking CAR T therapy called Kymriah which uses the body’s own reengineered T-cells to recognize B-cell acute lymphoblastic leukemia cells as foreign and destroy the cancerous cells.

Improves Quality of Life After Cancer

Eighty percent of children diagnosed with cancer today are now cured, thanks to research and improved treatments over the past few decades. Unfortunately, chemotherapy and radiation can have permanent life-long ramifications, which are dependent upon the type of cancer, the location of the cancer and then the type of treatments used to eradicate the cancer.

Now that more kids are surviving, we need to develop ways to treat cancer that are less-toxic to the child. Typically, the strongest therapy is used first, because any relapse of cancer creates a much more resistant cancer, which means we can’t necessarily try the mild therapy out of the gate because if it doesn’t work the child will likely die.

Ongoing research helps us determine when it’s appropriate to decrease intensity of certain therapies to prevent side effects. It also helps us discover new therapies that are less poisonous to the child (such as Kymriah).

Finally, we know some children may be more predisposed to cancer, but now genomics can also be used to identify kids that may be predisposed to side effects. That explains, in part, the reason we'll see one child sail through treatment without any problems and another child that suffers from several side effects.

Research Impacts Everyone

What we learn about childhood cancer is quite applicable to adult cancer. If it doesn't help our child or our grandchildren, it has a very good chance of benefiting ourselves or our loved ones that do develop cancer.

Childhood cancer research will also help save the next future Einstein, the astronaut to Mars, or the future doctor or nurse who will care for our children. Without research, these kids would otherwise die from their cancer. Now, they can contribute to society and help all of us. And it's always been my belief, the measure of a society is how well we take care of our most vulnerable citizens and there are few that are more vulnerable than kids.


Learn more about the Cancer Center at Children’s Mercy.

Learn more about the Children’s Research Institute at Children’s Mercy.