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Overcoming Challenges With Treating Young Sarcoma Patients

Gina Columbus @ginacolumbusonc
Published: Tuesday, Jun 21, 2016

Noah Federman, MD

Noah Federman, MD

Although advances have been made in treatment options for patients with a variety of sarcomas, researchers are still actively working to improve outcomes for a prevalent population with the disease—pediatric patients and adolescent and young adults (AYA).

“If you have an advanced soft tissue sarcoma or a recurrent sarcoma, the outcomes for these patients are on the order of 20%,” explains Noah Federman, MD. “That means that 8 out of 10 of our children are passing away from this disease. Where are we going in terms of the outcomes for these patients?”

Research being conducted for this population includes nanotechnology, imaging modalities, and novel chemotherapy regimens, Federman adds.

In an interview with OncLive, Federman, director of the Pediatric Bone and Soft Tissue Sarcoma Program, part of the UCLA Sarcoma Program and UCLA's Jonsson Comprehensive Cancer Center, discusses challenges with treating pediatric and AYA patients with sarcomas and ongoing efforts to overcome these obstacles.

OncLive: How often do you treat pediatric and AYA patients with this disease?

Federman: The bone and soft tissue cancers can be seen at any point in a person’s life from several months of age to 100 years of age. However, by and large, many of these are actually prevalent within the second and third decades of life—so age 10 to age 20. The patients I treat are generally in that age range. Although I am a pediatrician, there are many patients I treat who are age 18 to 25.

Why are sarcomas so prevalent in younger patients?

The connections with sarcomas and children and many pediatric cancers are very different than the connection that we see with cancer in adults. In adults, we believe that there is some environmental trigger, such as smoking, alcohol, or asbestos. In pediatric cancer, there really seems to be a genetic predisposition. What the predisposition is, we don’t know exactly.

For example, one of the clinical trials that we do involves whole-exome sequencing for patients. This has been a large clinical trial that is supported by UCLA’s Jonsson Comprehensive Cancer Center to basically characterize the genomes of patients, the tumors, and their parents to look for novel genetic predispositions and pathways for these cancers. Our goal is to find the genes and pathways that might lead to an understanding of some of these inherited predispositions, as well as an understanding of the potential of immunotherapy in the pathways.

What issues are there that this particular patient population faces?

The population of AYA patients is very unique. It is a very difficult population, as they are just asserting their independence. Their outlook on life is that they are immortal, and when they’re faced with a diagnosis of cancer, you have taken all of that away from them.

It’s a very delicate balance between treating them as adults, but also giving them all of the support they need from their family and psychosocial needs to all of the medical support.

Historically, when we look at clinical trials, the AYA population has really been left out of the clinical research in this field. We have been really good at enrolling patients less than 10 years old. We are also getting better at enrolling adult patients, but between the AYA and adult population is really where we need participation in trials.

What are some ongoing sarcoma trials you are working on right now?

The research that we are doing with the Jonsson Comprehensive Cancer Center at UCLA is centering on patients with rare bone and soft tissue cancers. Generally, these are children, adolescents, and young adults.

Two of the clinical trials we are doing are looking at imaging—specifically, PET/CT scans—where we would like to see an increase in activity, to see the cancers more accurately, and to stage them more accurately. Then, we want to look at the response to our treatments, so that, hopefully, we can see whether patients are responding to them earlier in the course of their disease.

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