The Children’s Brain Tumor Project made amazing progress this year, not only in terms of what we learned but also in what we shared. The fact is, getting our discoveries out into the scientific community is just as important as making them in the first place. Research is such an iterative process—we make a small but significant finding and share it by publishing an academic paper read by colleagues around the world, which they then use to advance their own work. They publish their new findings, which we then read and use to inspire our next steps. Back and forth, one step at a time, we move the science forward.
That’s why 2016 was such a big year for us. We made some very interesting findings, but we also concentrated on sharing them. The Internet is a big help here, since it shortens the time it takes to disseminate information. An accepted paper can spend months in the queue for publication, but academic journals now publish electronically in advance of print. That gets our findings into circulation much faster, and it also lets us learn from other labs in a more timely way.
For example, one of our papers appears in the current (November 2016) issue of the Journal of Neurosurgery: Pediatrics, but it was made available to other researchers in June. “A novel magnetic resonance imaging segmentation technique for determining diffuse intrinsic pontine glioma tumor volume,” by Dr. Souweidane’s team, describes an innovative way to measure DIPG tumors. With its irregular borders and diffuse characteristics, a pontine glioma is notoriously difficult to measure. As we move toward finding the right drugs to tackle DIPG, and the right way to deliver them, it becomes very important to know the exact volume of an individual tumor. This new method of measuring a tumor will help establish the best dosage for each patient.
Another milestone paper was released earlier this year in the journal Critical Reviews in Eukaryotic Gene Expression. That paper, “Clinical Genomics: Challenges and Opportunities,” was published by Dr. Greenfield’s team, including Ty Louis Campbell Fellow Sheng Li, and provided an overview of clinical genomics from study design to computational analysis. As the science of genome sequencing races forward, this kind of overview is invaluable to researchers working in the field of precision medicine.
Research presentations are ongoing, as we recognize the importance of sharing information as quickly as we can. As you read this newsletter, Christopher Marnell is attending the 2016 Annual Meeting of the Society for Neuro-Oncology, where he will present findings from his study of drugs with potential against DIPG. Chris started his study with 114 FDA-approved drugs and identified four that blocked the growth of DIPG cells. He is presenting his results on two of those drugs, perifosine and topoisomerase inhibitors, which have suppressed growth of those cells in vitro when used in combination.
This exciting project is another example of the benefits of collaboration: The DIPG cell cultures Chris tested were provided by Dr. Michelle Monje at Stanford University School of Medicine, and the compounds themselves were a gift from the Developmental Therapeutics Program of the NIH’s National Cancer Institute, Division of Cancer Treatment & Diagnosis.
Next month, Umberto Tosi will present his findings at the annual meeting of the Joint Pediatric Section of the AANS/CNS. Umberto has been working on modifying a promising drug, the kinase inhibitor dasatinib, to make it visible on PET scans as it enters, affects, and clears a DIPG tumor. His presentation will focus on one of several dasatinib variations, or analogues, he created; these analogues retain their effectiveness against tumors, but unlike the original dasatinib they can be seen and monitored on scans. This allows a neurosurgeon administering the drug via convection-enhanced delivery (CED) to confirm successful dosing, spot a missed delivery, and monitor the rate at which the drug clears the tumor. The most promising of the analogues will now undergo further testing, and the modification will also be attempted in another drug, panobinostat.