A team of trans-Atlantic researchers led by Children’s Hospital of Philadelphia (CHOPs) recently received nearly $680,000 from a group of charities led by Solving Kids’ Cancer UK to study slowly progressive, or “indolent” neuroblastoma, which does not respond to chemotherapy and lacks other treatment options. The grant will fund research that could ultimately lead to better diagnosis of this form of the disease, as well as targeted treatment options. The interdisciplinary group involves researchers from CHOP; the Icahn School of Medicine at Mount Sinai; The Institute of Cancer Research, London; Texas Tech University Health Sciences Center; and Seattle Children’s.
What is Neuroblastoma?
Neuroblastoma is a type of cancer that forms in developing nerve cells and can present differently depending on the form of the disease. Some types can spontaneously regress and become benign, whereas others progress steadily and are fatal. The rate of progression varies. While certain forms of the disease progress rapidly but can be treated effectively with chemotherapy, other forms, like indolent neuroblastoma, progress slowly and are resistant to chemotherapy. The latter form predominantly affects older children, teenagers and adults, who have very little chance of surviving their disease.
The Need: Precision Testing to Improve Treatment
Currently, researchers lack tools to identify patients with indolent neuroblastoma and therapies with which to cure them. The joint award from Zoé4Life, Joining Against Cancer in Kids (JACK), Merryn Lacy Trust, Oscar Knox Fund, Solving Kids’ Cancer (US) and Solving Kids’ Cancer UK will allow the trans-Atlantic group of researchers to validate a specific and sensitive molecular test in tumors, which will be further developed for use with patient blood, in order to reliably identify patients with slowly progressing disease, rather than waiting for chemotherapy to fail them. The research team will also create and validate robust laboratory models of indolent neuroblastoma, which they will use to test combinations of immunotherapy with targeted small molecular therapies.
Nick Bird, Research Trustee at Solving Kids’ Cancer UK, emphasized the challenge today: “Putting your child through multiple rounds of chemotherapy and watching them suffer from horrible side-effects only to find it’s made no difference to their disease is a truly devastating blow for parents.” He continued, “We have to identify these children at diagnosis so we can spare them from chemotherapy that doesn’t work, and then we have to find better ways to treat their disease instead.”
Ultimately, these researchers seek to rapidly move a completely new therapy to an international clinical trial.
Principal Investigator Point of View
“This grant will allow us to make progress in diagnosing and treating so-called indolent neuroblastoma, which is currently fatal in most patients,” said principal investigator John M. Maris, MD, pediatric oncologist at the Cancer Center at CHOP and Giulio D’Angio Chair in Neuroblastoma Research at CHOP. “If we can identify signatures that allow us to diagnose this form of neuroblastoma in patients early, we can avoid unnecessary chemotherapy, which is not effective in these patients, and instead work toward targeted treatments that could ultimately improve patient outcomes.”
John M. Maris, MD, pediatric oncologist at the Cancer Center at CHOP and Giulio D’Angio Chair in Neuroblastoma Research at CHOP.