At TEDx Longwood this spring, Leonard Zon, MD, founder and director of the Stem Cell Program at Boston Children’s Hospital, took the stage. In his enthusiastic yet humble style, he took the audience on a journey that included time-lapse video of zebrafish embryos developing, a riff by Jay Leno and a comparison of stem cell “engraftment” to a college kid coming home after finals: “You sleep for three days, and on day 4, you wake up and you’re in your own bed.” Three takeaways:
1) Stem cells made from our own skin cells can help find new therapeutics. With the right handling, they themselves can be therapeutics, producing healthy muscle, insulin-secreting cells, pretty much anything we need. (So far, this has just been done in mice.)
2) Zebrafish, especially when they’re see-through, can teach us how stem cells work and can be used for mass screening of potential drugs. The Zon Lab boasts 300,000 of these aquarium fish, and can mount robust “clinical trials” with 100 fish per group.
3) Drugs discovered via zebrafish are in human clinical trials right now: A drug to enhance cord blood transplants for leukemia or lymphoma, and an anti-melanoma drug originally used to treat arthritis.
Zon, who co-founded the biopharm company Fate Therapeutics, will be part of a judging panel of clinicians and venture capitalists for the Innovation Tank at Boston Children’s Global Pediatric Innovation Summit + Awards (Oct. 30-31). Don’t miss it!
Clinical excellence is the foundation of patient care. But at a recent TEDx Longwood event, Elaine C. Meyer, PhD, RN, co-founder and director of the Institute for Professionalism and Ethical Practice at Boston Children’s Hospital and an Associate Professor of Psychology at Harvard Medical School, offered insight on the other half of the health care equation: the human connection and the power of conversation.
Meyer’s moving presentation makes clear how communication—listening and sharing words of comfort—profoundly impacts patient experiences, as does its absence. Through heartfelt stories, including her own experience as a patient, her talk empowers physicians, nurses, social workers, psychologists and other medical staff to “be present” and communicate with patients and families compassionately.
“Dig deep, find your inspiration to have conversations,” Meyer says, because patients remember the words spoken to them and how those words made them feel.
(Credit: John Earle Photography)
Growing up, my grandmother’s eyes were always a problem. For years, she was losing her central vision to glaucoma, and numerous surgeries and treatments did not seem to help. Later in life, she could not see my face but could always tell who I was when I was close.
Glaucoma is the leading cause of irreversible blindness worldwide. While FDA-approved medications such as latanoprost can prevent vision loss by reducing pressure in the eye, their beneficial effects are limited by poor patient compliance: At six months of treatment, compliance is estimated to be little more than 50 percent.
Why? First, the medications are typically delivered as eye drops, and the drops themselves can cause stinging and burning. The drops also contain preservatives that can cause ocular surface disease.
Perhaps most importantly, latanoprost and other glaucoma drugs halt the disease’s progression but do not reverse it. Taking the drugs does not provide positive feedback that will motivate patients, such as relieving pain. Full story »
A restored, clear cornea grown from ABCB5-positive limbal stem cells. (Image courtesy of the researchers)
Severe burns, chemical injury and certain diseases can cause blindness by clouding the eyes’ corneas and killing off a precious population of stem cells that help maintain them. In the past, doctors have tried to regrow corneal tissue by transplanting cells from limbal tissue—found at the border between the cornea and the white of the eye. But they didn’t know whether the tissue contained enough of the active ingredient: limbal stem cells.
How cancer research led to a regenerative treatment for blindness.
Results have therefore been mixed. “Limbal stem cells are very rare, and successful transplants are dependent on these rare cells,” says Bruce Ksander, PhD, of the Massachusetts Eye and Ear/Schepens Eye Research Institute. “If you have a limbal stem cell deficiency and receive a transplant that does not contain stem cells, the cornea will become opaque again.”
Limbal stem cells have been sought for over a decade. That’s where a “tracer” molecule called ABCB5—first studied in the context of cancer—comes in. Full story »
Joseph Caputo originally wrote this post for the Harvard Stem Cell Institute (HSCI). Vector editor Nancy Fliesler contributed.
The fat cells shown in yellow are descended from transplanted human mesenchymal stem cells (green) inside of a mouse after co-transplantation. The red stain shows native mouse fat cells.(Courtesy Juan Melero-Martin)
Stem cell scientists had what first appeared to be an easy win for regenerative medicine when they discovered mesenchymal stem cells several decades ago. These cells, found in the bone marrow, can give rise to bone, fat and muscle tissue, and have been used in hundreds of clinical trials for tissue repair.
Uses range from tissue protection in heart attack and stroke to immune modification in multiple sclerosis and diabetes. Unfortunately, the results of these trials have been underwhelming. One challenge is that these stem cells don’t stick around in the body long enough to benefit the patient. Full story »
Eugenia Chan, MD, MPH, is a developmental-behavioral pediatrician and health services researcher in the Division of Developmental Medicine at Boston Children’s Hospital. She runs the Developmental Medicine Center’s ADHD Program and is co-developer of ICISS Health, a web-based disease monitoring and management system.
A randomized trial will soon test whether web-based updates from parents and teachers improve outcomes in ADHD, autism and more.
When I set out with my collaborator Eric Fleegler, MD, MPH, to build a web-based tracking system for children with attention deficit hyperactivity disorder (ADHD), we focused on a single problem—getting parents and teachers to fill out symptom questionnaires in time to help doctors make informed clinical decisions at follow-up visits. We had no inkling of the possibilities that this kind of software platform could hold, or how it might grow in the future. Full story »
Prenatal cell therapy could avoid the need for invasive surgery to repair myelomeningocele.
The neural tube, which becomes the spinal cord and brain, is supposed to close during the first month of prenatal development. In children with spina bifida
, it doesn’t close completely, leaving the nerves of the spinal cord exposed and subject to damage. The most common and serious form of spina bifida, myelomeningocele, sets a child up for lifelong disability, causing complications such as hydrocephalus, leg paralysis, and loss of bladder and bowel control.
New research from Boston Children’s Hospital, though still in animal models, suggests that standard amniocentesis, followed by one or more injections of cells into the womb, could be enough to at least partially repair spina bifida prenatally.
Currently, the standard procedure is to operate on infants soon after delivery. Full story »
Alina Morris, Archivist, Boston Children’s Hospital, contributed to this post.
In 1914, Boston Children’s Hospital, then simply called The Children’s Hospital, constructed the 145-bed Hunnewell Building, joining Harvard Medical School as one of several founding members of the Longwood Medical Area.
As the hospital’s oldest continuously occupied building, Hunnewell has presided over many of the century’s great medical advances and innovations. We celebrate a portion of them in this slideshow honoring Hunnewell’s 100th anniversary—and invite you to help write the next 100 years of history October 30-31 at Boston Children’s Global Pediatric Innovation Summit + Awards 2014.
My daughter just surprised me by signing up for fifth grade band starting this fall. To my further delight, some new research—using both cognitive testing and brain imaging—suggests that as she practices her clarinet, she also may be honing her executive functions.
Like a CEO who’s on top of her game, executive functions—separate from IQ—are those high-level brain functions that enable us to quickly process and retain information, curb impulsive behaviors, plan, make good choices, solve problems and adjust to changing cognitive demands. While it’s already clear that musical training relates to cognitive abilities, few previous studies have looked at its effects on executive functions specifically.
The study, appearing this week in PLOS ONE, compared children with and without regular musical training, as well as adults. To the researchers’ knowledge, it’s the first such study to use functional MRI (fMRI) of brain areas associated with executive function and to adjust for socioeconomic factors. Full story »
Juan Melero-Martin, PhD, runs a cell biology and bioengineering lab in the department of Cardiac Surgery at Boston Children’s Hospital. In May, he received an Early Career Investigator Award from Bayer HealthCare, part of the prestigious Bayer Hemophilia Award.
A bioengineered network of blood vessels
In 1982, insulin became the first FDA-approved protein drug created through recombinant DNA technology. It was made by inserting the human insulin gene into a bacterial cell’s DNA, multiplying the bacteria and capturing and purifying the human insulin in bioreactors. Full story »