Stem Cell-Based Strategies Offer Personalized Approaches for Treating Inherited Skin Disease

Two new studies funded in part by the NIAMS describe efforts to develop stem cell-based approaches for treating Epidermolysis Bullosa (EB), a rare, genetic skin disease. The findings, which were published jointly in the journal, Science Translational Medicine, may lead to individualized therapies for EB, and possibly other genetic diseases. People with EB have skin that is so fragile that the slightest friction causes blisters. The severity of the disease ranges from limited tearing of skin on the hands and feet to widespread blistering and scarring, including mucosal surfaces like the inside of the mouth and the airways. In some severe cases, complications such as infection or damage to internal organs can lead to premature death.

Some cells can repair EB-causing mutation in collagen XVII gene and produce functional protein (green). Photo credit: Angela Christiano, Columbia University.

Long-Term Follow-Up Data From Large Orthopaedic Studies May Help Guide Clinical Practice

Musculoskeletal conditions like spinal stenosis and anterior cruciate ligament tears in the knee affect people of all ages and can be debilitating. Recently reported long-term data from two large-scale studies funded in part by the NIAMS provide clinically valuable insights into the outcomes associated with treatments for these common, and sometimes costly, orthopaedic problems.

NIAMS’ Intramural Research Programs Foster Spirit of Discovery 

In this article from the March 15 issue of The Rheumatologist, the NIAMS Intramural Research Program and the NIH Rheumatology Training Program are recognized for excellence in training and innovation.

Scientists Make No Bones About First Study of Osteocyte Cultures on Space Station

NIAMS-funded research on osteocytes is headed to the International Space Station this spring for the first time. With their delivery on the next SpaceX commercial resupply services mission this month, the Osteocytes and mechano-transduction (Osteo-4) investigation team will analyze the effects of microgravity on this type of bone cell. Understanding these effects will be critical as astronauts plan for future missions that require longer exposure to microgravity, such as to deep space or Mars. The results derived from this study could also have implications for patients on Earth in the treatment of bone disorders related to disuse or immobilization, as well as metabolic diseases such as osteoporosis.

Mouse osteocytes within the bone. Photo credit: Dr. L Bonewald.

NIAMS Takes On “Language Access”

The Affordable Care Act has made it possible for millions of people to attain affordable health coverage. However, people with limited English proficiency face a daunting challenge upon entering the health care system—language barriers. According to recent U.S. Census Bureau estimates, nearly 20 percent of the U.S. population—about 55 million people—speak a language other than English at home. The NIH has addressed this challenge by establishing the NIH Language Access Plan, a comprehensive strategy being implemented across the agency to help people with limited English proficiency access NIH programs and activities. The NIAMS is at the forefront of making health information more accessible.

NIAMS To Host Twitter Chat on Vasculitis 

The NIAMS will host a Twitter chat on the topic of vasculitis on Wednesday, May 13, 2015, from 2 p.m. to 3 p.m. EDT. The chat will focus on the various forms of vasculitis, their symptoms and treatments, and current research. Followers will also learn how they can participate in NIH clinical studies. NIAMS experts Peter Grayson, M.D., M.Sc., and Elaine Novakovich, R.N., will answer questions from the audience. Other NIH Institutes and Centers may also participate. To follow the chat, go to https://twitter.com and type #NIHVasculitisChat in the search field.

NIH Selects Awardees To Help Speed Development of Health Technologies

The NIH has selected three new proof-of-concept hubs to help speed the translation of basic biomedical discoveries into commercial products, such as new drugs, devices and diagnostics, to improve patient care and enhance health. The hubs are part of the NIH-supported Research Evaluation and Commercialization Hubs (REACH) program and will be funded at $9 million over three years. REACH is a trans-NIH program and will be overseen by a committee with representatives from across the agency.

Study Demonstrates Success of NIH’s National Center for Advancing Translational Studies (NCATS) Rare Diseases Therapeutic Development Programs 

Developing new drugs can take years and cost billions of dollars. Because of the time, expense and likelihood that a promising drug will never make it to market, few companies are willing to investigate new drugs to treat diseases that may not be well understood or provide the potential for a good return on investment. The picture can be even more challenging when it comes to developing drugs for rare diseases. To counter these systematic challenges, NCATS runs two innovative late-stage preclinical drug development programs, Therapeutics for Rare and Neglected Diseases (TRND) and Bridging Interventional Development Gaps (BrIDGs).

Dylan, a young boy who has Proteous syndrome. Photo credit: Rick Guidotti, Positive Exposure/Proteous Syndrome Foundation.

NIH Announces “Follow That Cell” Challenge Finalists

The NIH has selected 16 finalists for Phase 1 of its Follow That Cell Challenge. The goal of the challenge is to stimulate the development of new tools and methods that will enable researchers to predict the behavior and function of a single cell in complex tissue over time. This ability could help reveal valuable information, such as how cells transition from a healthy to diseased state, or identify changes that influence a cell’s responsiveness to treatment.

Update on NIH’s Implementation of Federal Grant Policies

On February 5, the NIH announced its “NIH Interim Grant General Conditions” [PDF - 664 KB], the agency’s first step toward implementing federal-wide administrative requirements for the use of federal funds.

LabTV: Young Scientist Curious About the Immune System

Welcome to LabTV! If you haven’t already, take a look at this video. I hope you will enjoy meeting the first young scientist featured in this brand new series that I’ve chosen to highlight on my blog. The inspiration for LabTV comes from Jay Walker, who is the founder of PriceLine and curator and chairman of TEDMED, an annual conference focused on new ideas in health and medicine.

Cool Videos: Patching and Sealing the Cell Membrane

Bill Bement describes himself as a guy who “passionately, obsessively, and almost feverishly” loves to study cells. His excitement comes through in our final installment of the American Society for Cell Biology’s Celldance 2014. Bement, an NIH grantee at the University of Wisconsin, Madison, shares his scanning confocal microscope for this fascinating glimpse into the rapid response of cells to repair holes, tears and other structural damage in their protective outer membranes. As noted in the video, some forms of muscular dystrophy stem from an inherited inability to repair breaks in the cell membrane of skeletal muscle cells.

Snapshots of Life: Cell Skeleton on the Move

Cells are constantly on the move. They shift, grow and migrate to new locations—for example, to heal a wound or to intercept an infectious agent as part of an immune response. But how do cells actually move? In this image, Torsten Wittmann, an NIH-funded cell biologist at the University of California, San Francisco, reveals the usually-invisible cytoskeleton of a normal human skin cell that lends the cell its mobility.

Photo credit: Torsten Wittmann, University of California, San Francisco.

FDA Approves First Biosimilar Product Zarxio

The U.S. Food and Drug Administration (FDA) approved Zarxio (filgrastim-sndz), the first biosimilar product approved in the United States. A biosimilar is highly similar to biological products, which are among the medications used to treat rheumatoid arthritis, anemia, low white blood cell counts, inflammatory disease, psoriasis and various types of cancer.

Sun Protection and Spring Break—Reminders from the Centers for Disease Control and Prevention

Traveling for spring break? Don’t forget to pack, protect yourself from the sun and go! Don’t risk ruining your trip or your health with too much sun.

NEW PUBLICATIONS AND PRODUCTS

Spotlight on Scientific Imagery: Traumatized Muscle Tissue

This is a scanning electron microscope image of traumatized muscle tissue taken from a soldier injured during Operation Enduring Freedom. It shows a red blood cell entangled in a nanofibrous extracellular matrix. Highly fibrotic regions such as these are thought to precede bone formation during abnormal wound healing, leading to the formation of bone in locations outside the skeleton, such as soft tissue (heterotopic ossification). The image is in the public domain and is courtesy of Gregory Christopherson, Ph.D., and Leon Nesti, M.D., Ph.D., both formerly of the NIAMS Cartilage Biology and Orthopaedics Branch.

National Health Observances and Related NIAMS Resources

The NIAMS National Multicultural Outreach Initiative webpage highlights upcoming national health observances and related NIAMS resources. The NIAMS is featuring these five health observances that will occur in May:

• May Is Arthritis Awareness Month
• May Is Lupus Awareness Month
• May Is National Asian American/Pacific Islander Heritage Month
• May Is National Osteoporosis Awareness and Prevention Month
• May Is Native Hawaiian Heritage Month

NIH Public Access Plan for Increasing Access to Scientific Publications and Digital Data

The NIH, like other federal science agencies, has been developing a plan for increasing access to scientific publications and digital data in response to a White House Office of Science and Technology Policy memorandum [PDF - 53 KB] issued in 2013. The final NIH plan [PDF - 464 KB] and the plans developed by other U.S. Department of Health and Human Services (HHS) agencies were recently approved by Secretary Burwell. They are posted on the HHS Open Government website. The NIH Public Access Plan is also available on the NIH Office of Extramural Research’s sharing page.

Updated NIH Pain Consortium Website

The NIH Pain Consortium was established to enhance pain research and promote collaboration among researchers across the many NIH Institutes and Centers that have programs and activities addressing pain. The consortium supports initiatives, development of research resources and tools, and hosts events to promote collaboration and highlight advances in pain research. The News and Health Information Page now includes resources, such as Pain Research Advances, Pain Registries and the Office of Pain Policy Quarterly Newsletter.

NIH Research Matters

NIH Research Matters is a review of NIH research from the Office of Communications and Public Liaison, Office of the Director, NIH.

Autoimmune Disease Super-Regulators Uncovered

Genetic switches, called super-enhancers, help regulate the human immune system. The finding will inform research into autoimmune disorders, such as rheumatoid arthritis.

Human T cell. Photo credit: NIAID.

Human Epigenome Map Yields Insights Into Development, Disease

The sequencing of the human genome—the complete set of DNA in our cells—laid the foundation for understanding how variation in the genetic code can affect human health. The “epigenome” refers to the chemical modifications that affect how cells in different parts of the body use the same genome to form countless different types of cells and tissues. Epigenomic modifications don’t affect the DNA sequence itself. Rather, these alterations influence when specific genes are turned on and off, or “expressed.” These changes to DNA and its associated proteins tell cells what to do, where to do it and when.

The massive data from just one type of lung cell illustrates genome-wide measurements in 33 data sets. Image by the researchers, courtesy of Nature.

NIH News in Health

Read practical health information in NIH News in Health, which is reviewed by the NIH’s medical experts and is based on research conducted either by the NIH’s own scientists or by its grantees at universities and medical schools around the country.

Be a Partner in Clinical Research: Help Others, Help Yourself

Did you know that you can participate in clinical research? Whether you’re healthy or sick, young or old, male or female, you’re probably eligible to participate in some type of clinical study. Maybe you or a loved one has an illness, and you’d like to help scientists find a treatment or cure. If you’re healthy, you can help researchers learn more about how the body works or how sickness can be prevented.

MEETINGS

June NIAMS Advisory Council Meeting

The NIAMS Advisory Council Meeting will be held June 16, 2015, in Building 31, 6th Floor, C Wing, Conference Room 6, NIH Campus. A meeting agenda will be posted as soon as it is available. 

2015 NIH Pain Consortium Symposium—Advances in Pain Research: Looking Back and to the Future

May 26–27, 2015
NIH Campus, Natcher Auditorium (Building 45)
Cost: Free
View draft agenda
Registration is available here.

NIH Wednesday Afternoon Lecture Series

The NIH’s Wednesday Afternoon Lecture Series offers weekly lectures every Wednesday at 3 p.m. in Masur Auditorium, Building 10, NIH Campus. Renowned scientists from around the globe present research on a variety of topics. The lectures are Continuing Medical Education-certified, open to the public and available live via webcast.

Upcoming Lecture:

May 20, 2015
The Annual Marshall W. Nirenberg Lecture
David Page, MIT Whitehead Institute
“Lost in Translation: Do Males and Females Read Their Genomes Differently?”

NIH Science Lectures and Events Available via Internet

The NIH hosts a number of science seminars and events that are available online through real-time streaming video. You can watch an event at your convenience as an on-demand video or a downloadable podcast. Most events are available to all; a few are broadcast for the NIH or the U.S. Department of Health and Human Services and are marked as such. See additional details on events.

FUNDING ANNOUNCEMENTS

NIAMS Announcements

Request for Information: Core Centers for Clinical Research (CCCR)
(NOT-AR-15-010)

Notice of Change in Expiration Date for RFA-AR-15-014 “Validation of Pediatric Patient Reported Outcomes in Chronic Diseases (PEPR) Consortium (U19)”
(NOT-AR-15-015)

Advancing Mechanistic Probiotic/Prebiotic and Human Microbiome Research (R01)
(PA-15-135)
Letter of Intent Receipt date: Not applicable
Application Receipt Dates: Standard dates apply

NIH Common Fund Initiative Announcement

Notice Announcing Frequently Asked Questions (FAQs) for RFA-RM-15-001 “Metabolomics Core for the Undiagnosed Diseases Network (UDN) (U01)”
(NOT-RM-15-013)

Other Funding Announcements

Correction to Application Guide Instructions for Subawards Not Active in All Budget Periods of the Prime Applicant
(NOT-OD-15-073)

Notice of Correction to PA-15-087 “Development of Novel and Emerging Technologies To Support Zebrafish Models for Biomedical Research (R41/R42)”
(NOT-OD-15-074)

Notice of Correction to PA-15-086 “Development of Novel and Emerging Technologies To Support Zebrafish Models for Biomedical Research (R43/R44)”
(NOT-OD-15-075)

Notice of Change to the Eligible Activity Codes for PA-15-122 “Administrative Supplements for Common Basic Sociobehavioral Mechanisms and Processes That Facilitate or Impede Self-Management of Chronic Conditions (Admin Supp)”
(NOT-OD-15-076)

Eye on NIH Policy: OMB Uniform Guidance - What It Means for NIH & You (Video and Interactive Q&A - March 12, 2015)
(NOT-OD-15-077)

Use of Updated Inclusion Enrollment Format Now Required for Successful Submission of RPPR
(NOT-OD-15-078)

Notice of Update to the Public Health Service Policy on Humane Care and Use of Laboratory Animals
(NOT-OD-15-079)

Notice Regarding Requirement of Grantees and Contractors To Submit Invention Disclosures, Related Reports and Documents Via iEdison
(NOT-OD-15-080)

New Form To Capture Additional Indirect Costs in Multi-Project Grant Applications
(NOT-OD-15-081)

Reminder: NIH Grant Applications and the NIH Genomic Data Sharing Policy
(NOT-OD-15-083)

Reminder: NIH and AHRQ Biosketch Requirements for Due Dates On or After May 25, 2015
(NOT-OD-15-085)

Notice of National Center for Advancing Translational Sciences (NCATS) Participation in RFA-CA-15-006 “Big Data to Knowledge (BD2K) Advancing Biomedical Science Using Crowdsourcing and Interactive Digital Media (UH2)”
(NOT-TR-15-010)