A New Initiative on Precision Medicine Collins, Francis S; Varmus, Harold
New England journal of medicine/The New England journal of medicine,
02/2015, Letnik:
372, Številka:
9
Journal Article
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President Obama has announced a research initiative that aims to accelerate progress toward a new era of precision medicine, with a near-term focus on cancers and a longer-term aim to generate ...knowledge applicable to the whole range of health and disease.
“Tonight, I'm launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes — and to give all of us access to the personalized information we need to keep ourselves and our families healthier.”
— President Barack Obama, State of the Union Address, January 20, 2015
President Obama has long expressed a strong conviction that science offers great potential for improving health. Now, the President has announced a research initiative that aims to accelerate progress toward a new era of precision medicine (www.whitehouse.gov/precisionmedicine). We believe that the time is right for this visionary initiative, . . .
Despite dramatic advances in the molecular pathogenesis of disease, translation of basic biomedical research into safe and effective clinical applications remains a slow, expensive, and failure-prone ...endeavor. To pursue opportunities for disruptive translational innovation, the U.S. National Institutes of Health (NIH) intends to establish a new entity, the National Center for Advancing Translational Sciences (NCATS). The mission of NCATS is to catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of diseases and conditions. The new center's activities will complement, and not compete with, translational research being carried out at NIH and elsewhere in the public and private sectors.
In this Viewpoint directors of the NIH and NIDDK summarize the agency's 2020-2030 Strategic Plan for NIH Nutrition Research, a roadmap to establish a science of nutrition as a means to prevent and ...treat illness and to develop actionable, evidence-based dietary recommendations across cultures and settings.
The diary entry of an 8-year-old girl with cystic fibrosis indicates that Aug. 25, 1989, was an important day for her (Figure 1). That was the day the research teams at the University of Michigan and ...the Hospital for Sick Children, Toronto, announced the discovery of the cystic fibrosis gene and the most common mutation, a three-base deletion that results in a missing phenylalanine in codon 508 (denoted the Phe508del
CFTR
mutation). We hoped that the gene discovery would someday lead to effective treatments for children and adults with cystic fibrosis, but we knew that would be a long road.
2,3
. . .
A public-private partnership and platform for harmonized clinical trials aims to accelerate licensure and distribution
There is an unprecedented need to manufacture and distribute enough safe and ...effective vaccine to immunize an extraordinarily large number of individuals in order to protect the entire global community from the continued threat of morbidity and mortality from severe acute respiratory syndrome–coronavirus 2 (SARS-CoV-2). The global need for vaccine and the wide geographic diversity of the pandemic require more than one effective vaccine approach. Collaboration will be essential among biotechnology and pharmaceutical companies, many of which are bringing forward a variety of vaccine approaches (
1
). The full development pathway for an effective vaccine for SARS-CoV-2 will require that industry, government, and academia collaborate in unprecedented ways, each adding their individual strengths. We discuss one such collaborative program that has recently emerged: the ACTIV (Accelerating COVID-19 Therapeutic Interventions and Vaccines) public-private partnership. Spearheaded by the U.S. National Institutes of Health (NIH), this effort brings together the strengths of all sectors at this time of global urgency. We further discuss a collaborative platform for conducting harmonized, randomized controlled vaccine efficacy trials. This mechanism aims to generate essential safety and efficacy data for several candidate vaccines in parallel, so as to accelerate the licensure and distribution of multiple vaccine platforms and vaccines to protect against COVID-19 (coronavirus disease 2019).
The Next Phase of Human Gene-Therapy Oversight Collins, Francis S; Gottlieb, Scott
New England journal of medicine/The New England journal of medicine,
10/2018, Letnik:
379, Številka:
15
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As gene therapy continues to change, so must the federal framework set up to oversee it. As new biotechnologies continue to emerge, the NIH and the FDA are proposing reductions in duplicative ...oversight and changes to the role of the Recombinant DNA Advisory Committee.
This Viewpoint describes how the National Institutes of Health is partnering with more than a dozen biopharmaceutical companies and multiple other agencies to develop an international strategy for a ...coordinated research response to the COVID-19 pandemic, including prioritizing vaccine and drug candidates.
The Role of Science in Addressing the Opioid Crisis Volkow, Nora D; Collins, Francis S
New England journal of medicine/The New England journal of medicine,
07/2017, Letnik:
377, Številka:
4
Journal Article
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The National Institutes of Health will work with private partners to develop better overdose-reversal and prevention interventions; find new medications and technologies to treat opioid addiction; ...and find safe, effective, nonaddictive strategies to manage chronic pain.
As we mark the 1-year anniversary of the declaration by the World Health Organization (WHO) of COVID-19 as a global pandemic, the world has suffered a staggering and tragic human toll. During this ...dark time, the scientific community has been called to rise to the occasion in unprecedented ways. The intensity of the work and the sense of urgency have been unremitting and exhausting. As we sort out the triumphs and frustrations, we can begin to reflect on what we have learned.
First FDA Authorization for Next-Generation Sequencer Collins, Francis S; Hamburg, Margaret A
New England journal of medicine/The New England journal of medicine,
12/2013, Letnik:
369, Številka:
25
Journal Article
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The FDA has granted marketing authorization for a high-throughput genomic sequencer, Illumina's MiSeqDx, which will allow the development of innumerable new genome-based tests. Access to genomic data ...may transform research, clinical care, and patient engagement.
This year marks 60 years since James Watson and Francis Crick described the structure of DNA and 10 years since the complete sequencing of the human genome. Fittingly, today the Food and Drug Administration (FDA) has granted marketing authorization for the first high-throughput (next-generation) genomic sequencer, Illumina's MiSeqDx, which will allow the development and use of innumerable new genome-based tests. When a global team of researchers sequenced that first human genome, it took more than a decade and cost hundreds of millions of dollars. Today, because of federal and private investment, sequencing technologies have advanced dramatically, and a human genome . . .
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