Dr. Michael Kaplitt, our Vice Chair for Research, made news this summer when he became the first in New York to use Magnetic Resonance-guided Focused Ultrasound (MRgFUS) to treat patients with essential tremor, just a week after the technology received FDA approval. The completely non-invasive technique uses 1,000 low-energy ultrasound waves focused on a precise spot in the brain, each individual wave sparing healthy tissue while the combined energy destroys its target. Read more
Perhaps the greatest milestone in 2016 came when Dr. Uday Bhanu Maachani established two gliomatosis cerebri cell cultures from living patients. From one of these lines he created a xenograft: A mouse model with a GC tumor sampled from an actual patient. This is the only animal model anywhere in the world with a GC tumor cultured from a patient. Read more
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. Read more
There’s an old adage that says if you want to go fast, go alone; if you want to go far, go together. Research science rarely adheres to that—investigators spend a lot of lonely hours peering into microscopes and scrolling through endless data points, all in the hope that someday all that solo work will add up to a breakthrough. We often go alone, and go far, but usually not fast.
The Children’s Brain Tumor Project has also defied that adage in that we are going fast, but together. This requires teamwork like no other—we need other labs at Weill Cornell, tissue banks around the country, and other scientists comparing notes and sharing findings—because we know we need to move quickly, and get far. Read more
On New Year’s Eve 2010 our lives took an unimaginable turn: A doctor told us that our seemingly healthy 19-year-old daughter, Elizabeth, had inoperable brain cancer. We were advised to take her home, find palliative care, and expect her to succumb to her disease within the year. Words cannot describe our devastation, despair, and anger. How is it possible in this day, when we send space probes to the edge of the solar system and develop driverless cars, that there can there be no effective treatment options? There was literally no hope—only prayers for a miracle. Read more
They say that life is a highway and its milestones are the years,
And now and then there’s a toll-gate where you buy your way with tears.
—Joyce Kilmer (“Roofs,” 1917)
September is all about milestones, with its back-to-school firsts and the excitement of new beginnings. This year we have a special milestone: After 40+ years in my childhood home, including 14 years raising our family in it, the Andersons have moved! That house was a home base we had only ever planned on expanding, but Hurricane Irene five years ago, then Sandy a year later, was the beginning of the end. We began to mourn our home then, but we never could have imagined what was yet to come. I’ve learned a lot in the past five years about “the best laid plans.” Read more
Thanks to a number of grants and private donations, in July and August the CBTP lab team was joined by several young researchers working on specific projects to advance the field of pediatric neuro-oncology. This “summer sprint” was an unprecedented effort that produced some excellent results.
Umberto Tosi, funded by a POST grant from the Alex’s Lemonade Stand Foundation, worked on a project to improve the measurement of drug delivery to the brain via “theranostic” (therapeutic and diagnostic) agents.
The usual method of determining whether a drug has been successfully delivered is to wait for a clinical response, a “wait-and-see” approach that is neither timely nor precise. If researchers could modify a drug to make it fluorescent—and therefore visible on PET or MRI imaging—they would be able to see in real time whether that drug has reached its target. The key is to make delivery of the drug visible and measurable without reducing its effectiveness. Read more
As many of you probably read about on Facebook, on September 8 we treated the final patient in our Phase I clinical trial of convection-enhanced delivery (CED) for diffuse intrinsic pontine glioma (DIPG). The trial, which had enrolled 27 patients over the past four years, was designed to test the safety of CED as a means of delivering a cancer-fighting drug directly to the site of a DIPG tumor. Four patients received a second infusion, bringing the total number of treatments to 31. Read more
“Exploring the role of inflammation in the malignant transformation of low-grade gliomas.” Journal of Neuroimmunology, 2016 Aug 15;297:132-40. Epub 2016 May 25.
“A novel magnetic resonance imaging segmentation technique for determining diffuse intrinsic pontine glioma tumor volume.” Journal of Neurosurgery, Pediatrics, 2016 Jul 8:1-8. [Epub ahead of print]
“A Novel Methodology for Applying Multivoxel MR Spectroscopy to Evaluate Convection-Enhanced Drug Delivery in Diffuse Intrinsic Pontine Gliomas.” AJNR American Journal of Neuroradiology, 2016 Jul;37(7):1367-73. Epub 2016 Mar 3.
“Gliomatosis cerebri: A consensus summary report from the First International Gliomatosis cerebri Group Meeting, March 26-27, 2015, Paris, France.” Pediatric Blood Cancer, 2016 Jul 28. [Epub ahead of print]
“Convection-Enhanced Delivery for Diffuse Intrinsic Pontine Glioma Treatment.” Current Neuropharmacology, 2016 Jun 13. [Epub ahead of print]
“Clinical Genomics: Challenges and Opportunities.” Critical Reviews in Eukaryotic Gene Expression, 2016;26(2):97-113.
Click on any of the links above to read summaries of these papers on PubMed.com
This summer is going to be an amazing one in the Children’s Brain Tumor Project laboratory. Thanks to grants and gifts from families and foundations, we will have six summer researchers conducting several different lines of investigations, which will give our research a tremendous boost.