Proceeds go to UCLA Jonsson Comprehensive Cancer Center for
Dr. Noah Federman's Research for Pediatric Cancers
OUR Mission is to keep pushing pediatric cancer research towards more revolutionary breakthroughs. The proceeds help fund Dr. Federman's clinical trials at the UCLA Sarcoma Program. One of his studies is investigating if a new drug called TK216 will work in the treatment for recurring Ewing Sarcoma. Phase I will test to find the highest dose that can be given without severe side effects. This study laid the groundwork for research that led to FDA approval of a new drug called Larotrectinib. This drug is the first ‘tumor agnostic’ agent to be approved for unique advanced and metastatic pediatric and adult solid tumors presenting with a genomic TRK fusion. C2C also helped fund a pediatric liver cancer research and clinical trial. The money raised helps fund a precision medicine initiative to discover novel targets and screen novel therapeutics in pediatric liver cancers, so as to treat each patient individually.
Dr. Noah Federman is the Director of the Pediatric Bone and Soft Tissue Sarcoma Program at UCLA, part of the UCLA Sarcoma Program and UCLA's Jonsson Comprehensive Cancer Center. He specializes in treating children, adolescents and young adults with these aggressive cancers and runs a incredibly comprehensive and multidisciplinary program involving pediatric and medical oncologists, radiation oncologists, orthopedic oncology surgeons, musculoskeletal radiologists and pathologists, nuclear medicine specialists, physical therapists and prosthetic specialists. Dr. Federman also specializes in conducting clinical trials and leads an experienced clinical research team devoted to providing access to clinical trials for children with refractory, recurrent and metastatic bone and soft tissue cancers.
Pediatric Sarcomas, specifically Ewing Sarcoma (ES), is what C2C junior board member, Mafalda von Alvensleben, was diagnosed with in 2014. It is a highly aggressive bone and soft tissue cancer that affects mainly children ages 5 to 25. Only 3 in a million cases are reported each year, which makes it one of the rarest pediatric cancers. The pelvis is the most commonly affected area, followed by the femur, tibia, humerus, and scapula. The 5-year-survival rate for a localized tumor is 70%, whereas it is 15-20% when the tumor has spread at time of diagnosis. Higher dosages of chemotherapy drugs in shorter intervals have improved the outcome over the past 30 years. It is critical to be treated at a hospital like UCLA that has experience with ES.
Hepatoblastoma is a rare malignant liver cancer occurring primarily in infants and children under the age of three. C2C junior board member Jax Blumenthal was diagnosed with this form of cancer in 2013. His treatment included surgical removal of the tumor, adjuvant chemotherapy and liver transplantation. Primary liver transplantation provides long-term, disease-free survival rate in the range of 80%. The presence of metastases is the strongest predictor of a poor prognosis. Hepatoblastoma is diagnosed in 2 to 3 children in a million children anually.