Small interfering RNAs, or siRNAs, could be great targeted treatment tools for breast and other cancers. The problem is making sure they get packaged and delivered to where they need to go. (pscf11/Flickr)
Breast cancers whose cells carry the HER2 protein are pretty tough customers. They only account for about 20 percent of all breast cancers, but they are some of the most aggressive. While targeted drugs like trastuzumab (Herceptin) and lapatinib (Tykerb) have made these tumors easier to treat, those that resist these drugs, relapse or don’t have HER2 on their cells’ surfaces can still stymie oncologists.
A molecular phenomenon called RNA interference (RNAi)—in which small pieces of RNA silence the expression of individual genes—could provide an alternative solution for breast and other cancers.
Though it was first discovered in plants, researchers have known for about a decade that small interfering RNAs (siRNAs) are active in mammals like us, and are already working on ways to harness them for shutting down genes promoting cancer and other diseases.
The problem with siRNAs for treatment, however, is making sure they get exactly where they need to go. That’s a problem that Judy Lieberman, MD, PhD, has taken a big step toward solving. Full story »
What time is the right time to give a transfusion? Doctors at Boston Children's are turning a fresh eye on transfusion guidelines for children. (@alviseni/Flickr)
Cancer. Trauma. Sickle cell disease. Surgery. There are many reasons why a child might need a blood transfusion, but they all share a common theme: the need to replace blood or blood products (e.g., red blood cells, platelets) that have been lost or consumed, or make up for defects that keep the body from producing them in adequate amounts.
And though transfusions can be life saving, they come with risks, such as iron overload, inflammation or disease (a very low risk, thanks to improved screening tests). And blood products are expensive and scarce—another reason to be prudent about transfusions.
“There’s little science behind physicians’ current practices when deciding when to transfuse a patient,” says Jenifer Lightdale, MD, MPH, of Boston Children’s Hospital’s Division of Gastroenterology and Nutrition. “Many doctors use criteria their mentors passed down to them, which their mentors passed down to them, and so on. But ideally, the decision should be based on evidence, not tradition.” 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 »
Do the cells in this blood harbor a potentially harmful gene? If the answer is yes, but the person it belongs to donated it for unrelated research, it's not yet clear when - or how - to tell them. (JHeuser/Wikimedia Commons)
Snippets of tissue, vials of blood and tubes of DNA from hundreds of thousands of people sit in freezers and liquid nitrogen tanks right now in laboratories across the globe. They come from people like you and me, donated in the hope that our genes researchers will be able to glean insights for the next breakthroughs for diseases common and rare.
Whenever we sign a consent form and roll up our sleeve, we don’t just join the community of research. We also become part of a debate that has been raging among researchers, clinicians and ethicists for years: What if our DNA sequence turns up bad news unrelated to the research we signed up for?
This responsibility can quickly turn into a numbers problem – a massive administrative burden. Consider that there are more than 104,000 human genetic variations now cited in the medical literature with links to human disease. Full story »
Brain tumors like the diffuse, light gray one in this MRI do a remarkably good job of hiding from the immune system. A new treatment based on gene therapy could strip their camouflage away. (Filip Em/Wikimedia Commons)
If there’s anything that tumors are good at, it’s hiding themselves. Not from things like MRIs or CT scans, mind you, but from the immune system. Since a tumor grows from what were at one time normal, healthy cells it’s still “self,” still one of the tissues that makes you you.
“Tumor cells display very subtle differences that distinguish them from healthy cells, but by and large they look the same to your immune system,” says Mark Kieran, a pediatric neuro-oncologist at the Dana-Farber/Children’s Hospital Cancer Center and Children’s Hospital’s Vascular Biology Program. “The question is: How can we unmask tumors so that the immune system can do its job?”
Researchers have worked for years on cancer vaccines aimed at getting the immune system to wake up to the presence of a tumor and turn on it. With a Phase 1 safety trial , Kieran and his colleagues, including Children’s neurosurgical oncologist Lily Goumnerova, are evaluating a different strategy for patients with hard-to-treat brain tumors called malignant gliomas: They’re giving the tumors a cold. Full story »
Anesthesiologists have to get by and around a lot of things in order to put a breathing tube into a surgery patient. Kai Matthes thinks that using a pair of endoscopes could make the job easier. (National Cancer Institute)
Intubating the patient is a critical step in any surgery where general anesthesia is being used. But as any anesthesiologist will tell you, intubating a child is very different from an adult, largely because there is less space available in which to maneuver the breathing (aka endotracheal) tube.
There’s also less space in which to see. To place a breathing tube properly and keep the airway open, an anesthesiologist needs to see the patient’s vocal chords and the opening of his or her windpipe. Typically, the doctor uses a laryngoscope to see into the throat, but sometimes tumors, congenital anomalies, inflammation, or other obstructions block the view.
The next tool of choice would be a fiber optic endoscope – essentially a long, thin, tubular video camera – to peer within the throat. Sometimes, however, even the fiber optic scope can’t get a full view, and on occasion the scope and tube can get in each other’s way, making the anesthesiologist’s job harder and the procedure riskier.
But here’s a thought: If one scope can’t do the trick, what about two? Full story »
Brian Rosman holds up a tablet app he and a team of Children's and MIT Media Lab staff developed over the past two weeks during the Health and Wellness Hackathon
At 10 a.m. he’s directing two actors on set, at 10:34 a.m. he’s filling up a catheter and at 11:01 a.m. he’s gushing about the importance of pediatric avatars. Brian Rosman, a Robotic Surgery Research Fellow in the Department of Urology at Children’s Hospital Boston, has been working non-stop at the MIT Media Lab’s Health & Wellness hackathon to create a new app for post-operative care. His duties have included directing a video about the app, rounding up realistic props and explaining how the program works to judges and hackathon attendees.
Rosman and his team of coders, clinicians and industry professionals are competing against five other teams for a $10,000 prize awarded to the best open source healthcare application. Full story »
There are no best practices for turning patient's genome sequence into information that a doctor can easily understand…and act on. Children's Hospital Boston's CLARITY Challenge calls on the genomics community to come up with those practices, and possibly help three families in the process. (michab37/Flickr)
Personalized medicine, harnessing genomics to improve patient care, is a great idea on paper. But investigators have long struggled to find a smooth route from the bench – where patients’ DNA samples are sequenced – to the bedside, where a doctor can use a genomics report to diagnose illness, prescribe treatments and offer means of prevention.
Looking for innovations, Children’s Hospital Boston decided to use the incentive of competition, launching a contest called the CLARITY Challenge. The winner will be the company or group that can best translate the science of genomics into tools and methods that integrate into and inform everyday care. Full story »
If you look at the range of research models available to scientists today (from fungi to flies to mice and larger), one little guy stands out – a tropical freshwater fish from the rivers of Bangladesh called the zebrafish. While it may be small, this fish is having a big impact on medical science, especially in genetics, stem cell biology, and drug screening, as covered in today’s Wall Street Journal.
As we’ve mentioned previously on Vector, the zebrafish is swimming its way into many research programs, both here at Children’s Hospital Boston and across the country. As a model, they are quite attractive to researchers, in part due to their small size, their fecundity, and their surprising similarities to us (from a genetic standpoint, that is).
Richard White, who works with Leonard Zon in the Stem Cell Program at Children’s Hospital Boston, offers up an explanation for the fish’s popularity:
Medicine is a balancing act; how much of a drug is too much? A group of compounds called EETs provide a clear example of the possible dangers of giving patients too much of a good thing. (chris grabert/Flickr)
Usually when your doctor talks to you about lipids, he or she is talking about cholesterol (be it the good or bad kind). But cholesterol is only one kind of lipid. There are millions of these fatty molecules working in everyone’s body right now.
One family of lipids, known as EETs (or epoxyeicosatrienoic acids), is produced by the endothelial cells that line blood vessels, where they help control inflammation and the response to injury. Because EETs are also potent regulators of blood pressure, pharmaceutical companies are looking intently at compounds that raise bloodstream EET levels as a way of managing the cardiovascular aspects of more than 20 conditions, ranging from diabetes and stroke to kidney and eye diseases; some are currently in clinical trials.
There may be a catch, however: Some studies suggest that EETs promote angiogenesis, or blood vessel formation, and that the enzymes that process EETs have a relationship to cancer.
