The National Institutes of Health maintains a library of drugs, the Clinical Collection, that are safe for humans but failed in clinical trials or didn’t make it to the market for other reasons. These compounds, numbering 450 to date, are just sitting on the shelf, waiting for a researcher to identify a disease process they might treat.
Repurposing such drugs could potentially save the pharmaceutical industry time and money. Getting a new drug from R&D to market currently takes $2 to 3 billion and 13 to 15 years. In contrast, some estimate that repurposing a safe drug could cost just $300 million and take just 6.5 years.
Pfizer, one of the biggest pharma companies in the world, saw the appeal. It just launched SpringWorks Therapeutics, a mission-driven company dedicated to reviving shelved drugs to treat underserved diseases. In its pipeline are experimental therapies to treat four diseases that currently have no cure.
One of the earliest-stage candidates is senicapoc. …
One of the biggest challenges facing cancer researchers — and lots of other medical researchers, in fact — is that experimental models cannot perfectly replicate human diseases in the laboratory.
That’s why human Organs-on-Chips, small devices that mimic human organ environments in an affordable and lifelike manner, have quickly been taken up into use by scientists in academic and industry labs and are being tested by the U.S. Food and Drug Administration.
Now, the chips have helped discover an important link between breathing mechanics and lung cancer behavior. …
As the opioid epidemic deepens and drug overdoses increase, effective non-addicting painkillers are desperately needed. Efforts to discover new pain pathways to target with new drugs have thus far had little success. Other promising research is investigating triggerable local delivery systems for non-opioid nerve blockers, but it’s still in the early stages.
A new collaboration between Boston Children’s Hospital and the biopharmaceutical company Amgen is aimed at accelerating new pain treatments. Announced yesterday, it will revolve around patients with rare, perplexing pain syndromes. The scientists hope that the genetic variants they find in these patients will shed new light on pain biology and lead to new ways of controlling pain.…
A multi-center clinical trial is now offering nationwide genetic profiling services to pediatric and young adult cancer patients across the U.S. The goal is to identify gene mutations that can be individually matched with targeted drugs.
Sponsored by the National Institute of Cancer (NCI) and the Children’s Oncology Group (COG), the so-called NCI-COG Pediatric MATCH trial will screen patients’ tumors for more than 160 gene mutations related to cancer. Nearly 1,000 patients are expected to participate in the trial and it is estimated that 10 percent of those patients will be matched with a targeted therapy. …
Medical implants can save lives by correcting structural defects in the heart and other organs. But until now, the use of medical implants in children has been complicated by the fact that fixed-size implants cannot expand in tune with a child’s natural growth.
To address this unmet surgical need, a team of researchers from Boston Children’s Hospital and Brigham and Women’s Hospital have developed a growth-accommodating implant designed for use in a cardiac surgical procedure called a valve annuloplasty, which repairs leaking mitral and tricuspid valves in the heart. The innovation was reported today in Nature Biomedical Engineering. …
David Williams, MD, the principal investigator of the clinical trial, discusses gene therapy and its impact on children with adrenoleukodystrophy
Adrenoleukodystrophy — depicted in the 1992 movie “Lorenzo’s Oil” — is a genetic disease that most severely affects boys. Caused by a defective gene on the X chromosome, it triggers a build-up of fatty acids that damage the protective myelin sheaths of the brain’s neurons, leading to cognitive and motor impairment. The most devastating form of the disease is cerebral adrenoleukodystrophy (CALD), marked by loss of myelin and brain inflammation. Without treatment, CALD ultimately leads to a vegetative state, typically claiming boys’ lives within 10 years of diagnosis.
To run our circadian clocks, regulate sleep and control hormone levels, we rely on light-sensing neurons known as M1 ganglion cell photoreceptors. Separate from the retina’s rods and cones, M1 cells specialize in “non-image” vision and function even in people who are blind.
Reporting in today’s Cell, neuroscientists at Boston Children’s Hospital describe an unexpected system that M1 cells use to sense changing amounts of environmental illumination. They found that the cells divvy up the job, with particular neurons tuned to different ranges of light intensity.
“As the earth turns, the level of illumination ranges across many orders of magnitude, from starlight to full daylight,” says Michael Do, PhD, of the F.M. Kirby Neurobiology Center at Boston Children’s Hospital, senior author on the paper. “How do you build a sensory system that covers such a broad range? It seems like a straightforward problem, but the solution we found was a lot more complex than expected.” …
New research reveals why treatment might appear to fail to control glucose levels in many people with Type 2 diabetes: not taking their medication as prescribed.
“When patients have poor glycemic control, their physicians may assume that there was a medication failure when they were, in fact, not filling their prescriptions,” says Ken Mandl, MD, MPH, of Boston Children’s Hospital, the senior author of a new report in Diabetes Care.
The study raises the question of whether the same might be true for patients with other conditions. …
WATCH: DNA nanoswitches change shape in the presence of biomarkers. The shape change is revealed in a process called gel electrophoresis. Credit: Wyss Institute at Harvard University
“Nanoswitches” — engineered, shape-changing strands of DNA — could shake up the way we monitor our health, according to new research. Faster, easier, cheaper and more sensitive tests based on these tools — used in the lab or at point of care — could indicate the presence of disease, infection and even genetic variabilities as subtle as a single-gene mutation.
“One critical application in both basic research and clinical practice is the detection of biomarkers in our bodies, which convey vital information about our current health,” says lead researcher Wesley Wong, PhD, of Boston Children’s Hospital Program in Cellular and Molecular Medicine (PCMM). “However, current methods tend to be either cheap and easy or highly sensitive, but generally not both.”
To stay healthy, our lungs have to maintain two key populations of cells: the alveolar epithelial cells, which make up the little sacs where gas exchange takes place, and bronchiolar epithelial cells (also known as airway cells) that are lined with smooth muscle.