Targeting leukemia with a clinical trial of CAR T-cell therapy

relapsed leukemia targeted therapy CAR T-cell immunotherapy

One of the immune system’s basic jobs is to tell “self” from “non-self.” Our cells carry markers that the immune system uses to recognize them as being part of us. Cells that don’t carry those markers—like bacteria and other pathogens—therefore don’t belong.

Cancer cells, however, fall into a gray area. They’re non-self, yet they also bear markers that connote self-ness—one of the reasons the immune system has a hard time “seeing” and reacting to cancer.

Can we focus the immune system’s spotlight on cancer cells? The provisional answer is yes. Research on cancer immunotherapy—treatments that spur an immune response against cancer cells—has boomed in recent years. (The journal Science recognized cancer immunotherapy as its Breakthrough of the Year in 2013.)

And one of the more recent methods—called chimeric antigen receptor (CAR) T-cell therapy—is now in a clinical trial for relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL) at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

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What we’ve been reading: Week of May 4, 2015

Boy reading in grass

Apple Has Plans for Your DNA (MIT Technology Review)
Apple is collaborating with U.S. researchers to help launch apps that would offer iPhone owners the chance to get their DNA tested.

Why Your Future Vaccination Might Not Be A Shot (NPR)
Mark Prausnitz – a professor at Georgia Teach – is collaborating with the CDC and a group at Emory University to create an “ouchless” bandage-like vaccine.

Splice of life (Nature)
In light of the recent news that Chinese scientists genetically modified human embryos, the author calls for transparent discussions on the risks and ethics of editing human embryos.

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My work, my life: Gena Koufos, RN, MS, MBA

Gena Koufos, RN, MS, MBA, is program manager in Boston Children’s Hospital’s Innovation Acceleration Program. Her role entails designing new programs to support innovation acceleration across the institution. She offers resources and strategic guidance to cultivate and advance early stage innovators through product development and care delivery projects.

Learn more about the connection between nursing and innovation by exploring Koufos’ work and life. Click on the images and icons in the photo below to see what makes Koufos tick.

Explore the Innovation Acceleration Program at Boston Children’s.
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Sounding out intracranial pressure with a hearing test

Heidary ear ICP measurement croppedBrain tumors, traumatic head injury and a number of brain and nervous system conditions can cause pressure to build up inside the skull. As intracranial pressure (ICP) rises, it can compress the brain and result in swelling of the optic nerves, damaging brain tissue and causing irreversible vision loss.

That’s what nearly happened to a 13-year-old boy who had three weeks of uncontrolled headaches and sudden double vision. His neuro-ophthalmologist at Boston Children’s Hospital, Gena Heidary, MD, PhD, found reduced vision in the right eye, along with poor peripheral vision, an enlarged blind spot and swelling of both optic nerves.

As Heidary suspected, he had idiopathic intracranial hypertension, a condition that can raise ICP both in children and adults. Heidary performed an operation around the optic nerve to relieve the pressure, and vision in the boy’s right eye gradually improved, though not completely. Heidary has had to monitor his ICP ever since to protect his visual system from further irreversible damage.

Unfortunately, such monitoring currently is pretty invasive.

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Proteomics provides new leads into nerve regeneration

Nerve regeneration. From Santiago Ramón y Cajal’s “Estudios sobre la degeneración y regeneración del sistema nervioso” (1913-14). Via Scholarpedia.

nerve regeneration proteomicsResearchers have tried for a century to get injured nerves in the brain and spinal cord to regenerate. Various combinations of growth-promoting and growth-inhibiting molecules have been found helpful, but results have often been hard to replicate. There have been some notable glimmers of hope in recent years, but the goal of regenerating a nerve fiber enough to wire up properly in the brain and actually function again has been largely elusive.

“The majority of axons still cannot regenerate,” says Zhigang He, PhD, a member of the F.M. Kirby Neurobiology Center at Boston Children’s Hospital. “This suggests we need to find additional molecules, additional mechanisms.”

Microarray analyses—which show what genes are transcribed (turned on) in injured nerves—have helped to some extent, but the plentiful leads they turn up are hard to analyze and often don’t pan out. The problem, says Judith Steen, PhD, who runs a proteomics lab at the Kirby Center, is that even when the genes are transcribed, the cell may not actually build the proteins they encode.

That’s where proteomics comes in. “By measuring proteins, you get a more direct, downstream readout of the system,” Steen says.

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What we’ve been reading: Week of April 27, 2015

shutterstock_138760415

Crowdfunded science is here. But is it legit science? (Wired)
More and more scientists are turning to crowdfunding to pay for research. Is it the end of science as we know it or a meaningful and realistic evolution?

The world’s top 10 most innovative companies of 2015 in robotics (Fast Company)
Medical applications are well-represented in this compendium of companies working on the world of tomorrow today.

What Uber drivers can teach health care (KevinMD.com)
Doctors can learn from enterprising and fiercely independent Uber drivers.

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The silk scaffold: A promising road to hollow organ reconstruction

Silk photo_black backgroundSilk production and global interest in the lustrous fiber date back to prehistoric times. Today, the natural protein is solidifying itself as a biomaterials alternative in the world of regenerative medicine.

A recent study conducted by Boston Children’s Hospital urologist Carlos Estrada, MD and bioengineer Joshua Mauney, PhD, shows two-layer, biodegradable silk scaffolds to be a promising cell-free, “off-the-shelf” alternative to traditional implants for the reconstruction of hollow gastrointestinal structures such as the esophagus.

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RNSafe: Remote video checks of bedside drug dosing

RNSafe-Bunker nurse view Screenshot_2015-04-16-10-31-32When a nurse gives a complex medication at the bedside, a second nurse must come in to observe and verify the dose. But flagging down a nurse on a busy hospital floor can be pretty challenging, especially when the nurse has to “suit up” because of infection control precautions in the patient’s room. During a Nursing Morbidity and Mortality (M&M) Conference at Boston Children’s Hospital, a group of nurses expressed concern that this arrangement could potentially jeopardize safety. “We thought we should be able to do better,” says project co-developer Jennifer Taylor, MSEd, BSN, RN-BC, CPN.

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Looking beyond allergies: Does IgE keep a wary eye out for cancer?

watchdog IgE allergies cancer

Allergies of all kinds—to food, pollen, pets, etc.—can be blamed on a kind of antibody called IgE. Cousins of the more common IgG, IgE antibodies work with immune cells called mast cells to trigger the symptoms we associate with an allergic reaction (itchy skin, runny nose, closing throat, etc.).

Edda Fiebiger, PhD, has been studying IgE and allergies for years, and has noticed a curious association in several epidemiologic studies: people with high levels of IgE in their blood (as in people with allergies) have a lower risk of certain cancers. This—and the discovery of human IgE antibodies that bind to tumor antigens—suggests that IgE may help protect the body from cancer, and has given rise to a whole new field dubbed AllergoOncology.

But how does it work? In a recent paper in Cell Reports, Fiebiger and her colleagues reveal a pathway by which IgE may keep watch for tumor cells, one that’s totally separate from its allergic role.

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Inside the Lancet Commission on Global Surgery: A conversation with John G. Meara

meara-global-surgery
John G. Meara, MD, plastic surgeon-in-chief at Boston Children’s Hospital, co-chaired the Lancet Commission on Global Surgery, which released its findings today.

Although global health has come a long way over the past 25 years, access to surgical care remains very uneven across the world. Five billion people lack access to basic surgical care; this translates into unnecessary death and disability. More than one-third of all global deaths are from conditions requiring surgical care—more than the number of deaths from HIV/AIDs, tuberculosis and malaria combined. In addition, one-quarter of the world’s disability has been attributed to surgically treatable conditions.

In January 2014, an international team of 25 surgeons and public health experts launched The Lancet Commission on Global Surgery to address the widespread need for surgical care around the world. After 14 months of global consultation and four international meetings, the commission published a 32,000- word report today in The Lancet that provides a strategy for governments, policy makers, non-profits, funding agencies, academic institutions, professional associations, health care providers and local communities to engage in concrete action in low- and middle-income countries.

On May 6, the commission hosts its North American launch in Boston to present its key findings and priority action items. John G. Meara, MD, DMD, MBA, Plastic Surgeon-in Chief at Boston Children’s Hospital and the Kletjian Professor of Global Surgery at Harvard Medical School, is one of three chairs of the commission. We sat down with Meara to learn more about the commission’s work, which he describes as one of the “most impactful things he has done in his career to date.”

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