Good things, including therapeutics, can come in small packages—and increasingly this means nano-sized packages. For a sense of the scale of these diminutive tools, a strand of human DNA is 2.5 nanometers in diameter.
Nanomedicine offers the promise of drugs that are activated by physiologic stimuli in the body (like the shear stress of blood flow that’s partially blocked by a clot), that can home to very specific targets in the body (like pancreatic islets that are being attacked by the immune system in diabetes) and that carry their own imaging agents—a built-in “metric” to show that they’re working. Biomaterials are being crafted to enhance their properties—like adding gold “nanowires” to heart patches to increase their electrical conductivity.
Vector’s new sister publication, Innovation Insider, looks at the promise and challenges of nanomedicine—both technical and regulatory. Read more about nanoscissors, theranostics, quantum dots and how the future is nano.
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Using a novel 3-D culture method, scientists were able to prod lung (bronchioalveolar) stem cells to produce colonies with the cell type of choice: airway (bronchiolar) epithelial cells, alveolar epithelial cells or both. (Images: Joo-Hyeon Lee)
Someday it may be possible to treat lung diseases like emphysema, pulmonary fibrosis or asthma by prodding the lungs to produce healthy versions of the cells that are damaged.
That’s the hope of researchers Carla Kim, PhD, and Joo-Hyeon Lee, PhD, of the Stem Cell Research Program at Boston Children’s Hospital. In the Jan. 30 issue of Cell, they describe a pathway in the lungs, activated by injury, that directs stem cells to transform into specific kinds of cells—and that can be manipulated to enhance different kinds of repair, at least in a mouse model.
By boosting the pathway, Kim, Lee and colleagues successfully increased production of alveolar epithelial cells, which line the lung’s alveoli—the tiny sacs where gas exchange takes place, and that are irreversibly damaged in diseases like pulmonary fibrosis and emphysema. Full story »
The footpads of diabetic mice given a cream containing XIB4035 (lower right) have new nerve terminals (shown in green), whereas mice given a control cream (lower left) do not. The top two panels represent healthy “wild type” mice.
of people with diabetes develop peripheral neuropathy. The most common form, small-fiber neuropathy, generally starts in the feet, causing pain, odd sensations like pricks and “pins and needles,” and—the most worrisome feature—a loss
of sensation that can increase the chance of ulcers and infections.
In some cases, that may lead to the need for amputation—as happened with my diabetic great-grandfather whose numbed feet, unbeknownst to him, got too close to the fire.
While there are some treatments to reduce pain, there’s nothing that restores sensation. Nor do any existing treatments address the underlying cause of the neuropathy: the degeneration or dysfunction of the endings of the sensory neurons in the skin. Full story »
One of the hot trends in drug discovery could be called drug re-discovery: finding new uses for drugs that have already received FDA approval for a different indication.
It’s an approach that allows researchers and clinicians to rapidly test potential treatments for rare or difficult-to-treat conditions. Because the drug’s safety profile is already known, much of the preclinical and early clinical work that goes into developing a drug can be bypassed.
It was this kind of strategy that Alejandro Gutierrez, MD, and A. Thomas Look, MD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and Jon Aster, MD, PhD, of Dana-Farber Cancer Institute and Brigham and Women’s Hospital, had in mind when they started screening a library of nearly 5,000 FDA-approved compounds, off-patent drugs and natural products using a zebrafish model of T-cell acute lymphoblastic leukemia (T-ALL).
And with that strategy, they may have struck gold. Just not in the way they had expected. Full story »
2013 saw an accelerated crumbling of borders and boundaries in health care, fueled by technological and scientific advances. Boundaries between high-tech Western medicine and global health practices have begun blurring in interesting ways, as are those between home and hospital, patient and doctor and even a patient’s own body and the treatment used for her disease.
Last year also saw a fierce political fight over the Affordable Care Act (ACA)—aka Obamacare—ending in some six million people crossing the boundary from uninsured to insured, according to HMS, if you count Medicaid and Children’s Health Insurance Program eligibles.
What does all this portend for 2014? This year, Vector asked leaders from all walks of life at Boston Children’s Hospital to weigh in with their predictions. Full story »
Obesity may set off innate immune factors that inflame the lungs.
Both asthma and obesity have surged in recent decades, and a growing body of literature is linking the two conditions. Various explanations have been proposed: One recent study suggests that hormonal factors in obesity may regulate airway diameter; another suggests that obesity activates asthma-related genes.
“Why obesity predisposes a person to asthma has been a real puzzle,” says Dale Umetsu, MD, PhD, who recently researched the problem with Hye Young Kim, PhD, and other colleagues in the Division of Allergy and Immunology at Boston Children’s Hospital. “Our goal was to find the connection between these two problems, which occur in both children and adults, and to explore possible new treatments.”
The team’s research indicates that obesity alters the innate immune system—the body’s first responder to infection—in several ways, resulting in lung inflammation. Published earlier this month in Nature Medicine, their work also suggests a completely new, “druggable” approach to treating patients with obesity-associated asthma, for whom standard asthma drugs often work poorly. Full story »
In a one-two-three punch, a rapid screen in zebrafish can quickly identify a short list of drug candidates to test in mice and in patient-derived cells.
Scientists have had little success in growing skeletal muscle for patients with muscular dystrophy and other disorders that degrade and weaken muscle. Undertaking experiments in zebrafish, mouse and human cells, researchers have identified a way to do that, creating cells that Leonard Zon, MD
, hopes to see tested in patients in the next several years.
But what really excites Zon, director of the Stem Cell research program at Boston Children’s Hospital, is the power of the chemical screening platform he and his colleagues used. Described last week in the journal Cell, it found a cocktail of three compounds that induced human muscle cells to grow—in just a matter of weeks. Zon believes it could fast-track drug discovery for multiple disorders. Full story »
In mice, VEGF-A modRNA visibly improved blood supply to heart muscle (right image) as compared with no treatment.
Heart attacks cause the death of billions of the heart’s muscle cells. If these cardiomyocytes could be made to regenerate after an infarct, the heart could potentially be mended and its function restored.
Researchers have struggled to find the right approach to regeneration. Cell transplants have been tried, but the cells don’t engraft well long term and haven’t shown efficacy. Gene therapy to spur regeneration has been tested in animals, but dosage is hard to control and there’s a risk of genes going where they shouldn’t, causing tumors and other problems. Protein drugs have been tried, but they have short half-lives, being degraded or eliminated by the body before they can do much good. They are also hard to target to the heart.
A more recent approach to cardiac regeneration is to stimulate the body itself—and, specifically, progenitor cells— to repair the heart from within. Full story »
Drug approval is increasingly intertwined with pricing questions.
At last month’s BioPharm America conference, what I originally thought would be a run-of-the-mill panel wound up being a frank discussion about regulatory and pricing challenges that pharma and biotech companies are facing today. I hadn’t realized these two challenges are intertwined so closely.
The regulatory and pricing paths for new drugs in the United States have become increasingly difficult to navigate. Due to outside policy pressures, the FDA is scrutinizing drugs more than in the past, requiring much more data. Even when a drug is approved, there is no guarantee that payers will cover its full cost, as they are starting to consider the drug’s overall value—improving quality of life and decreasing costs—along with its effectiveness.
Meanwhile, in many European single-payer countries, pharmaceutical companies are being told how to price their drugs before they are considered for approval by the regulatory agencies. The likely effect is less return on investment on new drugs, which could in turn decrease the pace of innovation.
Vaughn Kailian, managing director of MPM Capital, a health care venture capital investment firm, led an eye-opening conversation around these topics. Full story »