Director, Children’s Brain Tumor Project
One of the most important — and challenging — aspects of working with a disease like gliomatosis cerebri is access to tissue samples. The best way for us to study this tumor is by seeing, testing, and studying many different examples of it, and finding out as much as we can about the young patients diagnosed with it. We believe that’s the key to personalized medicine: Exploring what these tumors, and these patients, have in common, and how they’re different, will help us understand how GC works and how we can defeat it.
That’s no easy task — there are only a handful of individuals each year diagnosed with gliomatosis cerebri, so the universe starts out as a small one. If the patient happens to live in a major urban area, with access to a comprehensive medical center, odds are that the surgeons there know about the Children’s Brain Tumor Project and will alert us to the case. In smaller towns and cities across the United States, though, a patient will often be seen and treated first at a local hospital, and we won’t find out about the diagnosis — at least not right away.
That’s one of the most critical pieces — timing is everything when it comes to tissue samples. We’re grateful for the number of samples we’ve already received from our partners, but too often these samples are taken during an autopsy, after the tumor has won the battle. Those samples bear the scars of that battle — the tumor has been irradiated or subject to chemotherapy, which changes its molecular makeup. Our lab can learn a lot from these samples, but we could learn so much more if we had access to tumor tissue taken during the original biopsy, before the tumor has been affected by treatments.
The challenge is not only one of spreading the word, but of putting the infrastructure in place to retrieve those samples. Imagine what’s entailed in this: First, the medical and surgical teams at a hospital have to know we even exist and need the samples. Then a frightened and overwhelmed patient and family have to learn about us and our work, and give their consent to donate samples. Finally, the sample has to be taken during a biopsy and then shipped to us overnight in a freezer package so it can be received here and stored safely in our lab for study. You can imagine how it would be easy to overlook this entire process in a small hospital, with a medical team that has probably never seen a single case of gliomatosis cerebri before.
There’s more to a registry than just tissue samples, too. We want Elizabeth’s Hope to be a source of support and hope to families receiving a diagnosis of gliomatosis cerebri. Thanks to our supporters we have begun the effort — if you type “child with gliomatosis cerebri” into Google you’ll find the Elizabeth’s Hope web site, built and maintained with donor dollars, right up there with giants like St. Jude and the Children’s Hospital of Boston. We have heard from patients all around the United States who have found us online. That exposure is exactly what we need to get the word out about what we do, but it’s an ongoing effort that needs to keep growing every day.
So the task at hand is as daunting as it is important. The first part — forming the partnerships with major hospitals — is already in place. The second — getting the word out to the surgeons and hospitals that might be doing a GC biopsy today and to the patients and families who might be getting that diagnosis tomorrow — is much more difficult and will take a lot more day-to-day effort.
I remain ever grateful for the support of our donors as we continue this important work — not only in the lab but on all the other fronts where we’re fighting this battle.