Thursday, October 3, 2019
New grants aimed at better understanding diseases, moving potential treatments closer to the clinic.
Of an estimated 6,500 to 7,000 known rare diseases, only a fraction maybe 5% have U.S. Food and Drug Administration-approved treatments. To increase that percentage, the National Institutes of Health has awarded approximately $31 million in grants in fiscal year 2019 to 20 teams including five new groups -- of scientists, clinicians, patients, families and patient advocates to study a wide range of rare diseases. An additional $7 million has been awarded to a separate data coordinating center to support these research efforts.
The grants, which support consortia that together form the Rare Diseases Clinical Research Network (RDCRN), are aimed at fostering collaborative research among scientists to better understand how rare diseases progress and to develop improved approaches for diagnosis and treatment. This is the fourth five-year funding cycle for the RDCRN, which is supported by multiple NIH Institutes and Centers and led by NIHs National Center for Advancing Translational Sciences (NCATS) and the NCATS Office of Rare Diseases Research.
Individually, most rare diseases affect only a few hundred to several thousand people; collectively, rare diseases affect more than 25 million Americans. Many rare diseases are life-threatening and about half of those affected are children.
Because rare diseases affect a small number of people, they can be extremely challenging to study. Scientists often lack basic information about a rare diseases symptoms and biology, and the ways a disease can affect people over time. Research funding can be scarce.
Over the years, RDCRN scientists have partnered with patients and advocates to develop new insights into the causes and progression of and potential therapies for rare diseases that were simply not receiving the attention they deserved, said NCATS Director Christopher Austin, M.D. Their pioneering work in discerning underlying clinical differences and commonalities in hundreds of rare conditions has already changed the rare disease landscape in immeasurable ways.
Established by Congress under the Rare Diseases Act in 2002, the RDCRN has included more than 350 sites in the United States and more than 50 in 22 other countries. To date, they have encompassed 237 research protocols and included more than 56,000 participants in studies ranging from immune system disorders and rare cancers to heart and lung disorders, brain development diseases and more.
Each RDCRN member is a consortium of clinical and scientific experts and patient groups who study a group of rare diseases. Each consortium must study three or more diseases, partner with rare disease patient advocacy groups, provide rare disease research training to investigators and perform natural history studies that chart the course and progression of diseases. The primary focus of the RDCRN is clinical research, and the network does not generally support clinical care outside of research activities.
A key component of the RDCRN is the Data Management and Coordinating Center (DMCC), which was awarded to the Cincinnati Childrens Hospital Medical Center. The DMCC manages shared resources and data from the RDCRN research studies. The DMCC emphasizes the standardization of data, increased data sharing and broad dissemination of research findings.
The RDCRN consortia have a rich history of accomplishment. For example, Lysosomal Disease Network scientists led crucial natural history studies and gene editing research that provided a foundation for first-in-human genome editing clinical studies for a rare metabolic disease. Primary Immune Deficiency Treatment Consortium members showed the advantage of early stem cell transplants for patients with a rare immune system disorder, severe combined immunodeficiency, and the groups work contributed to advances in gene therapy-based treatments for the disease.
New groups, new emphasis
The five new consortia are:
According to ORDR director Anne Pariser, M.D., an important focus of the latest group of awards is on clinical trial readiness.
Some of the RDCRN research groups have been working together for 10 or 15 years and have gathered important data and developed a good understanding of the diseases they study, in addition to new potential therapies. Were emphasizing the need to be prepared to conduct clinical trials, Pariser said.
Were trying to get the drug candidates closer to be ready for clinical testing and de-risk the processes that lead to a successful clinical trial, said RDCRN program officer Tiina Urv, Ph.D. To get funding to conduct trials, they need to have strong natural history studies that show how the disease progresses, ways to measure outcomes of treatments and biomarker studies that provide indicators of how a drug is working in patients.
Collaboration is key. Consortia can involve numerous partner research teams from different sites, along with rare disease patients and advocacy groups. Scientists from different institutions come together to pool patients, data, experience and resources.
Scientists cant work alone. They wouldnt have enough patients, and they wouldnt have adequate resources and information about the diseases, Urv said. Patients and families help scientists decide what is important to study, test and treat.
To read more about the five new consortia, 15 continuing consortia and the DMCC, see: https://ncats.nih.gov/rdcrn/consortia
In addition to NCATS, other NIH funding support comes from the National Institute of Allergy and Infectious Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, the National Heart, Lung, and Blood Institute, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Dental and Craniofacial Research, the National Institute of Mental Health and the Office of Dietary Supplements.
About the National Center for Advancing Translational Sciences (NCATS):NCATS conducts and supports research on the science and operation of translation the process by which interventions to improve health are developed and implemented to allow more treatments to get to more patients more quickly. For more information about how NCATS is improving health through smarter science, visithttps://ncats.nih.gov.
About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.
NIHTurning Discovery Into Health
- Inflammation produced by bacterial infection 'alerts' the brain stem cells - News-Medical.net - November 29th, 2020
- Government of Canada and JDRF Canada announce new research funding to accelerate stem cell-based therapies for type 1 diabetes - India Education Diary - November 29th, 2020
- Cancer Stem Cell Therapy Market Research Report 2020 By Segmentations, Key Company Profiles and Demand Forecasts to 2026 - The Market Feed - November 29th, 2020
- Human Embryonic Stem Cell (hESC) Market Research Report 2020 with Manufacturing Process Analysis and Market Concentration Rate till 2026 - The Market... - November 29th, 2020
- Stem Cell Assay Market Research Report: Industrial Chain, Sourcing Strategy and Downstream Buyers with Forecast 2026 - Cheshire Media - November 29th, 2020
- Cord Blood Stem Cells Market 2019 | Analyzing The Impact Followed By Restraints, Opportunities And Projected Developments | Industry Growth Insights -... - November 29th, 2020
- Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User,... - November 27th, 2020
- Global Animal Stem Cell Therapy Market Research Report by Size, Data, Developments, Global Demand, In-Depth Analysis and Forecast 2020 to 2025 |... - November 27th, 2020
- Europe Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User, and... - November 27th, 2020
- Scaffold Technology Market Research Report by Product, by Application, by End-User - Global Forecast to 2025 - Cumulative Impact of COVID-19 -... - November 27th, 2020
- The Thorough Analysis Of The Global Stem Cell Media market Research During COVID-19 Health Crisis - The Courier - November 27th, 2020
- Autism genes affect development of neurons and glia - Spectrum - November 27th, 2020
- Global Live Cell Imaging Market Report 2020-2025: Market Size is Projected to Reach USD 2.8 Billion by 2025 from USD 1.8 Billion in 2020 - PRNewswire - November 27th, 2020
- North America Cell Therapy Instruments Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Product ; Cell Type ; Process ; End User,... - November 27th, 2020
- Post-doctoral Fellow in Computational Stem Cell Biology job with THE UNIVERSITY OF HONG KONG | 234737 - Times Higher Education (THE) - November 27th, 2020
- Circulating Tumor Cells and Cancer Stem Cells Market Research Report 2020 with Manufacturing Process Analysis and Market Concentration Rate till 2026... - November 27th, 2020
- Analyzing Impacts of Covid-19 on Global Cell Culture Media Market Effects, Aftermath and Forecast To 2025 - The Courier - November 27th, 2020
- Accretion: Current and Future Business Analysis in Cell Expansion Market - PharmiWeb.com - November 27th, 2020
- Adipose Tissue-Derived Stem Cells (ADSCS) Market Expected to Witness High Growth over the Forecast 2027 - Cheshire Media - November 27th, 2020
- Stem Cell Market to Witness a Pronounce Growth During (2020-2026) Cheshire Media - Cheshire Media - November 27th, 2020
- Demand with COVID-19 impact analysis on Cell Expansion Market - The Courier - November 27th, 2020
- Approval of Phase I/II Clinical Trial of ATG-016 (Eltanexor), a Second Generation Selective Inhibitor of Nuclear Export (SINE), in Mainland China for... - November 27th, 2020
- Researchers grow first plant-based gel to help organoid development for biomedical applications - Microbioz India - November 27th, 2020
- California's stem cell research agency looks to the future - Bond Buyer - November 25th, 2020
- Unexpected discovery about stem cell immortality study - News - The University of Sydney - November 25th, 2020
- Breakthroughs in Stem Cell Based Treatment of Heart Disease - The Connecticut College Voice - November 25th, 2020
- Mount Sinai Cardiologist Awarded $2.9 Million NIH Grant to Advance Work with Stem Cells and Heart Repair after Heart Attack - Cath Lab Digest - November 25th, 2020
- Stem Cell Therapy Global Market Report 2020-30: Covid 19 Growth and Change - Yahoo Finance UK - November 25th, 2020
- Global Cell Harvesting Market to Reach US$381,4 Million by the Year 2027 - PRNewswire - November 25th, 2020
- Middle East & Africa Cell Therapy Instruments Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Product ; Cell Type ; Process ;... - November 25th, 2020
- Global Stem Cell Banking Market to Get Expansion admist COVID 19, Scope With Advanced Technologies Top Key Players and Forecast 2020-2027 - The... - November 25th, 2020
- New study on CRISPR: the stake of unintended consequences in embryos - BioNews - November 25th, 2020
- The Stem Cell Characterization and Analysis Tools Market to grow incomparably in the next decade - The Market Feed - November 25th, 2020
- New solution to kill coronavirus on common surfaces in seconds - University of California - November 25th, 2020
- Agricultural Equipment Market to Witness Robust Expansion Throughout the Forecast Period 2027 - re:Jerusalem - November 20th, 2020
- Animal Stem Cell Therapy Market Projected to Witness Vigorous Expansion by 2027 - re:Jerusalem - November 12th, 2020
- The Canine Stem Cell Therapy Market To Move Away From Insipidness, Reach US$ 218.2 Mn - PRnews Leader - November 12th, 2020
- Rheumatoid Arthritis Stem Cell Therapy Market to Ride on Increased Prevalence of Rheumatoid Arthritis - TMR Research Blog - November 12th, 2020
- Stem Cell Therapy Market Growth Analysis 2020 By Industry Top Manufacturers, Business Opportunities, Industry Growth, Size, Gross Margin, Regional... - November 12th, 2020
- Stem Cell Media Market 2020 Industry Analysis and Trends Forecast to 2026 Thermo Fisher, CellGenix, Lonza, STEMCELL Technologies, Corning - The Think... - November 12th, 2020
- Autologous Stem Cell and Non-Stem Cell Based Therapies Market is Projected to Grow Massively in Near Future - The Daily Philadelphian - November 12th, 2020
- Global Live Cell Imaging Markets, 2020-2025 - Growing Adoption of High-Content Screening Techniques in Drug Discovery - Yahoo Finance UK - November 12th, 2020
- GLOBAL STEM CELL MARKET FORECAST 2021-2028 - GlobeNewswire - November 12th, 2020
- Flow Cytometry Market Is Expected to Generate Revenue of US$ 11.3 billion by 2030, Globally - GlobeNewswire - November 12th, 2020
- Stem Cell Therapy Market 2020 to 2025: Business Development Strategies and Top Manufacturers Analysis : Anterogen Co., Ltd. (South Korea), MEDIPOST... - November 12th, 2020
- ADC Therapeutics Reports Third Quarter 2020 Financial Results and Provides Recent Business Highlights - BioSpace - November 12th, 2020
- Stem Cell Characterization and Analysis Tool Market Size Analysis: Growth Factors, Share by Regional Production, Revenue and Regional Trends with Key... - November 12th, 2020
- Forma Therapeutics Reports Third Quarter 2020 Financial Results and Provides Business Update - Galveston County Daily News - November 12th, 2020
- Adipose Tissue-Derived Stem Cells (ADSCS) Market Expected to Witness High Growth over the Forecast 2027 - BCFocus - November 12th, 2020
- Marker Therapeutics Reports Third Quarter 2020 Operating and Financial Results - PRNewswire - November 12th, 2020
- CODA Biotherapeutics Receives Grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health... - November 12th, 2020
- Brain Development and Disorder Research Receives $1.5 Million NSF Boost - USC Viterbi School of Engineering - November 7th, 2020
- Many Initiatives Still Too Close to Call in California - Courthouse News Service - November 7th, 2020
- Election 2020: Californias ballot initiative system isnt working. - Vox.com - November 7th, 2020
- Stem Cell Therapy Market is estimated to be worth USD 8.5 Billion by 2030 - PRnews Leader - November 5th, 2020
- Cancer Stem Cell Therapeutics Market Outlooks 2020: Size, Cost Structures, Growth rate and Industry Analysis to 2027 - Eurowire - November 5th, 2020
- Biologics Safety Testing Market Research Report by Test Type, by Application - Global Forecast to 2025 - Cumulative Impact of COVID-19 - Yahoo Finance... - November 5th, 2020
- Global Regenerative Medicine Market (2020 to 2025) - Industry Trends, Share, Size, Growth, Opportunity and Forecast - ResearchAndMarkets.com -... - November 5th, 2020
- Stem Cell Media Market Projected to Witness Vigorous Expansion by 2027 - The Think Curiouser - November 5th, 2020
- Cell Isolation Market Research and Analysis by Expert: import/export details, Cost Structures and statistics till 2027 - KYT24 - November 5th, 2020
- MorphoSys to Present Data on Tafasitamab at the ASH Virtual Annual Meeting and Exposition - PharmiWeb.com - November 5th, 2020
- The Many Model Systems of COVID-19 - The Scientist - November 5th, 2020
- Soybean Trypsin Inhibitor Market Brief Analysis and Application, Growth by 2026 | Biological Industries, Thermo Fisher Scientific, Sigma-Aldrich... - November 5th, 2020
- Cell and Tissue Culture Supplies Market Analysis with Market Size, Industry Share, trends and Forecast to 2026 - The Think Curiouser - November 5th, 2020
- Voters asked to approve $5.5 billion for stem cell research - Madison.com - November 3rd, 2020
- A Review of Stem Cell-Based Therapies for Parkinson Disease - AJMC.com Managed Markets Network - November 3rd, 2020
- 10 health and science-focused races to watch on election night - STAT - November 3rd, 2020
- Animal Stem Cell Therapy Market 2020, Global Research Analysis Report Growth at CAGR Value, Industry Share, Key Company Profiles, Type, Applications,... - November 3rd, 2020
- Stem Cell Therapy Market To Exceed Revenues Worth US$ By The End Of 2020 2027 - Eurowire - November 3rd, 2020
- Stem Cell Banking Market Research Report 2020 By Segmentations, Key Company Profiles and Demand Forecasts to 2026 - Aerospace Journal - November 3rd, 2020
- Stem Cell Media Market Analysis And Demand With Forecast Overview To 2027 - Aerospace Journal - November 3rd, 2020
- Stem Cell Characterization and Analysis Tool Market : By Component, by Application, by Component Mode, and By Region Global Forecast up to 2026 -... - November 3rd, 2020
- Impact of COVID-19 on Plant Stem Cell For Cosmetics Market Overview with Detailed Analysis, Competitive Landscape & Forecast To 2026 - Eurowire - November 3rd, 2020
- 2020 Election Tracker: All The State Ballot Healthcare Initiatives - Forbes - November 3rd, 2020
- California Proposition 14 is about pikuach nefesh heres why - Forward - November 3rd, 2020
- Retinal Disorders Treatment Market: Advancements in Gene-therapy and Stem-cell Therapy to Bolster Market Growth - BioSpace - November 3rd, 2020
- Stem Cell Alopecia Treatment Market Insights and Development Trends from 2020-2027 - Aerospace Journal - November 3rd, 2020
- Global Stem Cell Therapy Market 2020 Research on Import-Export Details, Business Standards and Forecast to 2025 - PRnews Leader - November 3rd, 2020
- Canine Stem Cell Therapy Market Shows Expected Trend to Guide from 2020-2026 with Growth Analysis by Manufacturers, Regions, Type and Application -... - November 3rd, 2020
- Survival Benefit of GVHD in MDS Is Limited to Specific Patient Subgroup - Cancer Therapy Advisor - November 3rd, 2020