Nestled in the pons (the red area above), the area that controls breathing, DIPG tumors have been impossible to biopsy and analyze for therapeutic insights. Until now. (MEXT Integrated Database Project/Wikimedia Commons)
Brain tumors can be very difficult to treat, but at least we know what to do about them. For years, a mix of surgery, radiation and chemotherapy has been used to treat brain tumors like medulloblastoma.
These treatments are fairly successful, but for a rare, almost always fatal tumor called diffuse intrinsic pontine glioma (DIPG), we haven’t had any success—in fact, we haven’t known where to start.
The problem has to do with where DIPGs are located: nestled among the nerves in a portion of the brain stem, the pons, that controls critical functions like our breathing, blood pressure and heart rate.
“For 40 years, we lacked the neurosurgical techniques to biopsy DIPGs safely,” say Mark Kieran, MD, PhD, director of the Brain Tumor Program at Dana-Farber/Children’s Hospital Cancer Center (DF/CHCC). “In fact, one of the first lessons every oncologist is taught still is, ‘Don’t biopsy brain stem gliomas.’ The dogma was that the risk of severe or fatal damage was too great.” And because we couldn’t biopsy them, we couldn’t study them to learn what makes them tick.”
A lot can change in four decades. Techniques for operating on the brain have advanced considerably, as have the tools for probing tumors at the molecular level. So, looking to turn the dogma about DIPGs on its head, Kieran has launched a clinical trial that aims to use advanced surgical and diagnostic tools to target and individualize DIPG treatment. Full story »
Margaret Coughlin is a Senior Vice President and the Chief Marketing and Communications Officer at Boston Children’s Hospital.
Here at the TEDMED conference, it’s all about horizontal or lateral thinking – coming at problems from new directions, without regard to conventional boundaries. I like the thoughts of Edward DeBono (not a TEDMED speaker), who coined the term “lateral thinking” in 1967:
Some people are unhappy about lateral thinking because they feel it threatens the validity of vertical thinking. This is not so at all. The two processes are complementary, not antagonistic. Lateral thinking enhances the effectiveness of vertical thinking by offering it more to select from. Vertical thinking multiplies the effectiveness of lateral thinking by making good use of the ideas generated.
Lateral thinking is, in a way, an antidote to the way we’re all taught—vertically and specifically. Our education systems seem to be getting more vertical – more concerned with meeting prescribed benchmarks, and, in so doing, discarding the creativity and imagination of learning that is critical to real innovation and real forward movement. As for medical education, radiation oncologist and TEDMED speaker Jacob Scott said it has replaced creativity in the brain with a warehouse. Full story »
Lewis Silverman, MD, thinks he may have a powerful new tool for treating children with relapsed acute lymphoblastic leukemia. (VashiDonsk/Wikimedia Commons)
The news that your child has cancer always comes as a shock, but for one cancer, acute lymphoblastic leukemia (ALL), parents can take comfort in the fact that doctors are really good at treating it. The cure rate for ALL has, over the last 40 years, climbed to nearly 90 percent.
Less comforting is the fact that some 10 to 20 percent of children who initially respond well to treatment suffer a relapse within five years. And right now, the drugs at our disposal aren’t very good at turning a relapse back into a remission.
“We have standard treatment regimens for newly diagnosed and relapsed ALL, both of which rely heavily on corticosteroids like prednisone and dexamethasone,” says Lewis Silverman, MD, director of the Pediatric Hematologic Malignancy Service at Dana-Farber/Children’s Hospital Cancer Center (DF/CHCC). “But we know that leukemias with any level of steroid resistance are more likely to relapse. Anything we can do to overcome that resistance would let us help many children.”
Silverman has launched a clinical trial that will try a new strategy for tearing down ALL cells’ barriers against corticosteroids. Full story »
Some children with autism are "bloomers" and are able to move to the high-functioning category. They're also more likely to have mothers who are white and educated.
A child with autism is more likely to do well if his mother is white and educated.
This is the message of a study just released in the journal Pediatrics, and it’s something we need to pay attention to—now.
Researchers from Columbia University wanted to find out what happens to children with autism over time. So they looked at the records of more than 6,000 children with autism who were enrolled in California’s Department of Developmental Services (DDS). To get into DDS they had to be referred, and their diagnosis had to be confirmed by someone with expertise in autism.
What they found was that when it came to social and communication skills, for the most part the kids fell into groups ranging from low-functioning to high-functioning. The kids did make progress; the most rapid gains were before age six, and the high-functioning kids tended to make more progress than the low-functioning ones. Even as they made progress, they tended to stay in the group they started in—with one notable exception. That exception was a group the researchers called the “Bloomers.” Full story »
Measuring the total amount of DNA damage within a tumor’s cells could help doctors predict its vulnerability to drugs like cisplatin. (Haukeland universitetssjukehus/Flickr)
Drugs like cisplatin that break DNA are some of the strongest weapons we have against breast, ovarian and other cancers. The problem, common to every form of chemotherapy, is that cisplatin doesn’t work for everyone. Given the potential side effects that go along with the drug—including vomiting, hearing loss and muscle cramps, just to name a few—the decision to give it to a patient becomes something of a gamble: Does the benefit outweigh the risk?
There are tests that examine individual genes and which can give doctors a limited view as to which tumors might respond best to cisplatin. But a multicenter team co-led by Zoltan Szallasi, MD, of Boston Children’s Hospital’s Informatics Program (CHIP), thinks they may have a solution that looks beyond individual genes to see which tumors might succumb to cisplatin and other drugs like it. Full story »
When children return home from the hospital after surgery, parents can be overwhelmed by the written information and instructions for follow-up. At the MIT Media Lab’s Health and Wellness Hackathon earlier this year, the focus was on empowering patients to take an active role in their health. As my colleague Brian Rosman described, our team from Boston Children’s Hospital attended and spent two weeks developing “Ralph,” a mobile application for managing post-operative care that incorporates an avatar and features of gaming to engage and motivate children to follow their regimen. I was one of the primary programmers for our group.
We won third place, working alongside five other talented teams. Here are some snapshots of what they were up to — helping patients manage asthma, diabetes, pain, cardiac rehab and more. Full story »
In his essay, “We, the Web Kids,” Polish poet and pundit Piotr Czerski writes: “We don’t use the Internet…we live on the Internet and along it…communicating with one another in a way that comes naturally to us, more intense and more efficient than ever before in the history of mankind.”
As Czerski emphasizes, we want the option of “here and now, without waiting for the file to download.” We (myself included) expect immediacy. So in my role as a public health advocate in the digital age, waiting for an official infectious disease outbreak report to come weeks after the outbreak started—as often happens with traditional reporting methods—is unacceptable. Earlier detection of disease outbreaks means earlier response—and more lives saved. This video produced by NPR illustrates the “web kid” mindset when it comes to public health:
Brain MRIs from mice after stroke. Mice lacking Hv1 (right panels) had a much smaller volume of infarcted tissue than normal mice. Hv1 can also be blocked chemically.
Whether it’s in adults or in children with clotting disorders or other conditions such as sickle-cell disease, a stroke can be likened to an atomic bomb. Just as ongoing radiation can do more damage than the bomb itself, the secondary damage of a stroke can devastate the brain.
In an ischemic stroke, accounting for nearly 90 percent of all stroke cases, it happens like this: When vessels supplying blood and oxygen to the brain are blocked by a narrowing or a clot, immune cells in the brain sense the low-oxygen conditions, suspect an invading organism and try to kill it by producing molecules known as reactive oxygen species or ROS’s. These, unfortunately, have an inflammatory effect that actually damages the brain further, injuring and killing neurons.
“Stroke produces inflammation, and that’s one of the main things people have been after in trying to reduce stroke damage,” says David Clapham, MD, PhD, chief of the Basic Cardiovascular Research Laboratories at Boston Children’s Hospital.
Right now there’s nothing that can do this. Most existing stroke drugs are aimed at preventing the stroke or dissolving blood clots once the stroke is happening – but they can’t deal with the aftermath. Full story »
David Williams wants to turn cells from Fanconi anemia (FA) patients into stem-like iPS cells. To do that, though, he needs to get the patients' cells to reboot properly. (_rockinfree/Flickr)
About a decade ago, David Williams, MD, set out to solve a problem. The chief of Dana-Farber/Children’s Hospital Cancer Center’s Hematology/Oncology division wanted to treat Fanconi anemia (FA)—a rare, inherited bone marrow failure disease—using gene therapy. In the process, he’d be able to replace patients’ faulty bone marrow cells with ones corrected for the genetic defect that causes FA.
There was one big problem though. “The main bar to attempting gene therapy in FA is that you need to be able to collect a certain number of blood stem cells from a patient in order to be able to give enough corrected cells back,” he says. “In our early clinical trials, we were unable to provide enough corrected stem cells to reverse the bone marrow failure we see in these patients.”
One way around the supply issue would be to create the necessary blood stem cells from FA patients’ own cells by first reprogramming skin cells into what are called induced pluripotent stem (iPS) cells. Using one of several methods, scientist can reboot mature skin cells into an immature, stem cell-like state—essentially turning the cells’ biological clocks back to a time when they could grow into anything the body might need. Full story »
Debra Weiner speaks with Emergency Department Fellow Joel Hudgins who holds one of the iPhones piloting BEAPPER
For Debra Weiner, MD, PhD, working in the Emergency Medicine Department is a numbers game. During a 12-hour shift she works with more than 50 other providers, sees up to 25 patients and analyzes multiple lab results. Every day she’s also meeting new staff members in addition to new patients.
“People don’t know each other,” Weiner said at a recent Innovators’ Forum, a monthly internal lecture series intended to showcase and encourage new developments at Children’s Hospital Boston. “We have over 100 nurses and physicians and over 200 trainees that filter in [every two to four weeks]… it’s hard to remember who everyone is and what they do.”
Coupled with the frenetic pace of Children’s Emergency Department (ED), remembering names and managing the flow of patients becomes a constant challenge. Full story »
