Can Small Molecule Agents Make a Big Difference?
This international collaboration of researchers and charities will rapidly introduce a new investigational drug to children and potentially result in significant clinical responses.
Project Title: A Phase I/II study of PF-06463922, a next-generation ALK inhibitor, in children with relapsed / refractory ALK-driven neuroblastoma
Investigators: Araz Marachelian, MD; Yael Mosse, MD; Lou Chesler, MD
Institutions: NANT Consortium, Royal Marsden Hospital, UK
Study Type: Phase I clinical trial
Targeted treatments for cancer using small-molecule agents have rapidly advanced in recent years. The discovery of activating mutations in the oncogene ALK indicated that children with neuroblastoma could benefit from therapies with oncogene-specific small-molecules. The first generation ALK inhibitor crizotinib had exhibited some responses in neuroblastoma trials, but inhibition of mutated ALK is complex and presented a therapeutic challenge with inherent resistance. However, researchers have now discovered the cause of this resistance and have overcome this in the newest ALK-inhibitor agent (PF3922). This international collaboration of researchers and charities will rapidly introduce a new investigational drug to children and potentially result in significant clinical responses. The hope is that this trial will result in rapid FDA and EMA approval so that this drug will be moved into frontline therapy in US and Europe in the next three years.
To learn more about this trial, visit clinicaltrials.gov
Charity Partners: Band of Parents, The Evan Foundation, Children’s Neuroblastoma Cancer Foundation, Ronan Thompson Foundation, Wade’s Army, The Catherine Elizabeth Blair Foundation, Solving Kids’ Cancer UK, Harrison Bate Memorial Fund, Ellen’s Friends and Family