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UPMC, Geisinger experts: New treatments offer hope for childhood cancer patients

For a parent, hearing that your child has cancer is probably one of the most devastating diagnosis. But, for a growing number of parents, that is their reality.

“Within the United States, if we look at cancer under the age of 15, we see about 15,000 cases — plus or minus 1,000 cases — a year,” said Dr. Louis Rapkin, clinical director of oncology at UPMC Children’s Hospital of Pittsburgh.

That number is slowly growing, Rapkin said, as the population grows in this country.

“If we extend that to adolescents, the numbers go up significantly. Under the age of 15, the rate is less than 1% of pediatric cancers, while in the 15 to 39 year-olds, which is considered adolescent/young adults, the numbers increase to about 7% or about 75,000 cases in that

age group. The rest of the 92% or so in the United States for cancer is for those older than 40,” he explained.

Although the prognosis for most patients with pediatric cancer is good, one of the differences in treatment between childhood and adult cancer patients is the lack of new drugs being approved by the Federal Drug Administration (FDA) for use on children.

“So historically, when I was in fellowship in the year 2000, at that point, I think there were maybe five chemotherapy drugs that were FDA approved in kids. That doesn’t mean we only used five drugs, but it meant that the FDA had not made a strong push to study these drugs in children. And if the drug companies could get approval for these drugs without doing the additional research in children, then they took that route. And I don’t mean to make that sound negative. I’m just saying that that’s the state of the world in the 80s, 90s and 2000s,” he said.

“In the last 10 years, the FDA has been pushing hard and trying to come up with rules and regulations to make drug companies do specific research in kids to get approval,” he said.

Drug companies will most likely not do studies on drugs that have been around for 30 to 40 years because they already know how they affect pediatric patients. There also has not been the pipeline for pediatric drug discovery the same way that there has been for adults.

“The main reason for that is we only have 15,000 kids a year with pediatric cancer, and we have 1.7 million adults with cancer, right? What is a drug company going to do from a profit margin standpoint? There are steps in place to try and encourage drug companies to do more approval for kids, but those steps don’t equal the benefit of doing a larger market,” he said.

“So again, let’s go back to that 15,000 kids. And let’s say we have an 80% survival rate. That means that the kids who are going to come up for trials for new drugs, meaning a child who relapses and therefore becomes a candidate, ethically, for the new drug, something that hasn’t been tried, that’s going to number a total of 3,000 kids- 3,000 kids across, let’s say 50 to 100 diagnoses,” he explained.

“We do not have a lot of kids to try all the new drugs that come out. So what we do a lot of times is, if there’s something that looks great, we’ll try it. But a lot of times we let the adults try the new medications, first to see what works in adults, and then we try and adapt these uses to kids. And so now, more and more drugs are beginning to come out with pediatric FDA approval,” he said.

Rapkin pointed to a brand new drug, Blinatumomab that had been in a national clinical trial and was recently approved by the FDA for kids two years of age and older.

“So we’re beginning to see these trials lead to more FDA approvals in kids,” Rapkin said.

Dr. Jagadeesh Ramdas, director of Pediatrics Hematology Oncology at Geisinger Medical Center, cited some immunotherapy interventions which have come up in the treatment of childhood cancers, specifically leukemia, which are making a big difference in the outcome or in management.

For the last 20 to 30 years we did not have any new drugs because we were more focusing on how to stratify them, how to put them in different buckets, and then do the treatment, Ramdas said.

The biology and science of leukemia has advanced and as of 2024, Ramdas said, immunotherapy treatments have also become an important role in childhood leukemias.

There have been a couple of immunotherapy interventions which work differently in the body and have proven to be effective in the management and treatment of leukemia, Ramdas said.

The No. 1 cancer in children is acute lymphocytic leukemia (ALL) with about 3,500 cases a year in this country. About 40% of the cancers in children are either leukemia or lymphoma. Leukemia could be ALL, AML — myelogenous leukemia — which accounts for about 1,200 cases a year, and then other forms of lymphoma, like Hodgkin’s lymphoma, or Parkinson’s lymphoma. About 35% of the kids they treat at UPMC Children’s Hospital have some form of a brain tumor.

In the next age group, the ages 15 through 39, there is a shift from leukemia to lymphoma.

“We start to see adult-style sarcomas occurring in kids. We see a rise in thyroid cancer beginning in late adolescence, teenage years and we begin to see in males testicular cancer begin to develop. Testicular cancers are the most common cancer in the third decade of life,” Rapkin said.

When children are diagnosed with cancer, it’s hard to think that they have been exposed for a long period of time to environmental hazards that have been linked to cancer in adults. That leads to the idea that children develop cancer because of genetic factors.

“This is an important distinction between adult and pediatric cancers,” Ramdas said.

Adult cancers such as lung cancer are “triggered by environmental or lifestyle habits like smoking or alcohol use,” he said.

Rapkin agreed, but added there can also be a “small input” from genetics.

“For example, if we talk about BRCA one and two for breast cancer…we know that accounts for 15% of breast cancer…maybe a little bit more. But most breast cancer is sporadic, meaning it’s associated with environmental features, not genetic,” Rapkin said.

About 20 years ago, it was thought that about 5% of pediatric cancers were genetic. Today that number has increased to 25% because the knowledge of genetics has increased.

“That doesn’t mean there’s going to be a family history. Many times these mutations are new — de novo — in the child and the parent didn’t have them or the mutation didn’t manifest in the parent but it is manifesting in the child,” Rapkin said.

For this reason family history isn’t always 100% reliable as a predictor of a child developing cancer.

“We have some national efforts right now in the Children’s Oncology Group (COG) and the National Institute of Health (NIH), where we are checking more and more — it started with very specific groups of cancer — looking for every child in the United States who’s on a pediatric oncology group study, We’re looking at all their genomics to see if we can find new associations,” Rapkin said.

Compared to adults who develop cancer, children with cancer usually have a higher survival rate than adults.

Rapkin explained that adults are considered cured after five years, but many adults relapse after that. In the pediatric population, if the patient reaches the five-year mark, they are considered cured.

The rates of survival improve if a pediatric patient is enrolled in a clinical study. Seeking treatment at a dedicated pediatric hematology oncology center is also important. There are 250 COG institutions in North America, so most children can get to one, Rapkin noted.

By doing all these measures, essentially the outcome of childhood cancers have improved a lot, Ramdas said.

The overall survival rates are now approaching 75 to 80% of childhood cancers that can be cured with all the effective treatments, he added.

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