Research Roundup: FDA Approvel of Keytruda a Landmark Moment for Precision Medicine

As May comes to a close, we bring you a round-up of some recent research news and developments related to childhood cancers:

THE BIG NEWS -- Keytruda Approved by FDA : In a groundbreaking research development this month, the Food and Drug Administration has approved the first ever cancer therapy based on a patients' specific genetic traits, rather than cancer type. This approval includes children with the identified genetic trait whose cancer has progressed and who have no alternative treatment options. This is a huge leap forward for the field of precision medicine, which uses a patient's genetic makeup to deliver immuno-targeted therapy. (Reuters, May 23  |  The American Journal of Pharmacy Benefits, May 16)

SPEAKING OF PRECISION MEDICINE: The Precision Medicine Institute at NIH has been renamed the All of Us Research Program. 

SECONDARY CANCER RISK FOR HODGKIN'S SURVIVORS: European researchers found that Hodgkin's Lymphoma survivors are at an elevated risk for secondary cancers, with a higher risk in survivors that have a family history of cancer. In particular, the study highlights an increased breast cancer risk for females treated for Hodgkin's at a young age and lung cancers for those with a family cancer history. (Cancer Knowledge Network blog by Dr. Greg Aune | Journal of Clinical Oncology, May 2017)

NEW SUB-TYPES OF MEDULLOBLASTOMA IDENTIFIED: The most common malignant pediatric brain tumor, medulloblastoma, has been sub-divided into seven distinct molecular sub-groups. Prior to this new research done by Newcastle and Northumbria Universities, there were just four identified sub-groups. These more precise characterizations will lead to more precise treatment regimens, hopefully leading to improved survival rates. (The Lancet Oncology, May 22)

MOUSE TRIALS SHOW PROMISE IN TREATING 5 DEADLY PEDIATRIC BRAIN TUMORS: A pre-clinical mouse trial study shows promise with a new immunotherapy treatment for Group 3 medulloblastoma, atypical teratoid rhabdoid tumors, PNET, glioblastoma, and DIPG. The immunotherapy targets a protein called CD47 and has the unique ability of bypassing the blood-brain barrier. Phase Two trials will examine the critical question of whether the therapy shows promise in humans. (Mental Floss | Science Translational Medicine, March 15)

These advances emphasize the critical importance of investing in childhood cancer research. More than 90% of children with cancer in the U.S. are treated at a Children's Oncology Group institution and COG relies on steady NIH funding to maintain and grow its clinical trial program. Through the collaborative research network of COG, childhood cancer has evolved from a virtually incurable disease 50 years ago to its overall 80 percent survival rate today. By working together under the umbrella of the COG, childhood cancer research and treatment has advanced at a much faster pace than would have been possible with institutions working alone.

We thank Congress for recently adding $2 billion for NIH to the remainder of the FY2017 budget, and we are optimistic that bipartisan support for cancer research will continue as Congress begins to deliberate over the FY2018 budget. The Trump Administration is calling for a $6 billion cut to the NIH (with $1 billion coming from NCI), but we are hopeful that lawmakers on both sides of the aisle will fight for more research dollars, not fewer.

You can write Congress here to ask for their continued support of life-saving research funding through the National Institutes of Health.

Jessica Kean