Rimas J. Orentas, PhD, Seattle Children's Research Institute, Seattle, WA, outlines the development of FGFR4-targeted chimeric antigen receptor (CAR) T-cells for the treatment of rhabdomyosarcoma. FGFR4 is upregulated in rhabdomyosarcoma tumors compared to normal tissue, making it a suitable candidate for CAR-T therapy. Two novel CAR-T therapies targeting FGFR4 which demonstrated high cellular toxicity and tumor specificity in vitro are being further developed and will be tested for in vivo efficacy against metastatic and intramuscular rhabdomyosarcoma. This interview took place at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021.

Excerpt from the September 2021 ICARE Genetics Case Conference focused on Rhabdomyosarcoma and Neuroblastoma with guest expert Douglas Stewart, MD.

Patient: Brooklyn, 10 Years Old
Cancer:  Embryonal Rhabdomyosarcoma, Stage 3 Group 3
Diagnosed: October 2020
Treated at: Atrium Health Levine Children’s

After a month of pneumonia-like symptoms, Brooklyn was diagnosed with Embryonal Rhabdomyosarcoma in October of 2020.  A tumor was discovered in her right lung, an extremely rare location for Rhabdomyosarcoma to originate from. Since her cancer diagnosis, she has endured 42 weeks of chemo, 23 days of radiation, 5 surgeries, countless in-patient hospital stays, and a feeding tube placement due to weight loss.

“These last few months have been extra hard on her, but the last two weeks have been the hardest. Her last two chemotherapy infusions really hit her hard and she can’t seem to recover. She has been so sick and has only had 2 good days out of the last 14.  Her body just can’t handle much more”

“Brooklyn desperately wants to return to her normal life of being a 10-year-old girl in the 5th grade. She wants to play with her 3 sisters and her puppy without getting tired and feeling sick. She wants to go back to school and enjoy her time left in elementary school and see all of her friends, classmates, and teachers. She wants to be able to do all the things that any normal child gets to do.”

After successful removal of the tumor, Brooklyn has been declared NED (no evidence of disease.  But Rhabdomyosarcoma is notorious for relapse due to its fast-growing nature, so the treatment is just as intense.

Last week Brooklyn completed 42 weeks of her aggressive chemotherapy regimen.  This intensive treatment to her body over such a long time period has lead to many side effects including mouth sores, ongoing fevers, nausea and stomach cramps, rapid heartbeat, severe nose bleeds, and neuropathy in her legs. Before Brooklyn moves on to the next phase in her treatment, she will have PET/CT scans Monday morning.  If the results of her scans continue to show no evidence of disease, she will switch over to maintenance chemotherapy.  Fortunately, maintenance chemotherapy is less aggressive and Brooklyn is looking forward to getting back to being just a kid again!

Rhabdomyosarcoma is a type of sarcoma, cancer of soft tissue such as muscle, connective tissue, or bone.