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March 2015 ACTS Connection, Issue 1
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Translational Science 2015 is Just Over a Month Away


Translational Science 2015 is just over a month away! Join us April 16 -18 at the Omni Shoreham for the meeting that brings together a multidisciplinary audience of clinical and translational researchers, for the unique benefit of networking and education with leaders.

Translational Science 2015 is offering 24 concurrent sessions designed to reach everyone from trainees to senior investigators. Notable sessions include:

How to Write a Successful K24 Grant to Support Your Mentoring, featuring Ellen W. Seely, MD, Harvard Medical School; Barbara A. Woynarowska, PhD, National Institute of Diabetes and Digestive and
Kidney Diseases

mHealth in Medicine and Biomedical/Translational Research, featuring Jennifer Kwan, MD, PhD, University of Illinois, Chicago; David Levin, MD, Vendome Healthcare Media; Evan Muse, MD, PhD, Scripps Translational Science Institute; Satish Raj, MD, University of Calgary; Linda Ricci, FDA/CDRH/ODE; Tim Shi, MD, PhD, SAS, CTM, GlobalMD Organization; Ivana Schnur, MD, PhD, Sense.ly Inc.

Register Now!
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Don't Forget to Book Your Hotel Room!

Translational Science 2015 will be held at the Omni Shoreham Hotel in Washington, DC on April 16th-18th, 2015. A group rate at the hotel will be available for meeting attendees. As a premier travel and conference location, April is a busy time of year in Washington, D.C. To maximize your travel budget, it is best to book your travel and accommodations early! The discounted group rate for Translational Science 2015 attendees will be available until March 25, 2015.

Upon booking your room, don't forget to join the Omni Loyalty Program to receive various perks and incentives during your stay – including complimentary WiFi service in your guestroom!

Book your room now!
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From the Hill--ACTS Advocacy News

Each spring, during Translational Science 2015, established clinical and translational researchers visit Capitol Hill with young investigators and researchers in training. This Day-on-the-Hill is the community’s best annual opportunity to educate legislators about the value and importance of supporting and funding the full spectrum of medical research. This year’s Advocacy Day is scheduled for Thursday, April 16th.

This will be a critical year for advocates to connect with policymakers on Capitol Hill as the topic of research funding is a critical one! Join the community for Hill Day and lend your voice to advocacy. Please note, you must register ahead of time to participate so that we can schedule congressional meetings for you and place you on a team with other advocates.
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House FY16 LHHS Hearing on NIH

On Wednesday, March 3rd, the House Labor-Health and Human Services-Education (LHHS) Appropriations Subcommittee held a hearing on the budget for the NIH. Consistent with previous years, this hearing occurred after the release of the President’s budget request to Congress and as part of the annual appropriations process (for Fiscal Year 2016). While the only witness to provide testimony before the Subcommittee was the NIH Director, Francis Collins, MD, PhD, additional NIH Institute directors where available to answer questions from the Subcommittee.

While making few commitments related to increasing funding through the annual appropriations process, the Subcommittee expressed universal support for NIH. Read key items from the hearing:


See more details on the LHHS Subcommittee hearing here.
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NIH Clinical Research Advisory Board Meeting [March 30th]

Pursuant to section 10(d) of the Federal Advisory Committee Act, as amended (5 U.S.C. App.), notice is hereby given of a meeting of the NIH Advisory Board for Clinical Research.

The meeting will be open to the public as indicated below, with attendance limited to space available. Individuals who plan to attend and need special assistance, such as sign language interpretation or other reasonable accommodations, should notify the Contact Person listed below in advance of the meeting.

NIH Advisory Board for Clinical Research
March 30, 2015, 10:00 a.m. to 1:30 p.m.
Agenda: To review the FY16 Clinical Center Budget.
National Institutes of Health, Building 10, CRC Medical Board Room 4-2551, 10 Center Drive, Bethesda, MD 20892.

Contact Person: Maureen E. Gormley, Executive Secretary, (301) 496-2897.
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News from ACTS


Share your exciting stories...Membership News!

Translational Science News


NIH-Led Effort Launches Big Data Portal for Alzheimer's Drug Discovery
Novel Financing Technique May Unlock Funding for Developing 'Orphan' Drugs to Treat Rare Diseases
Rare Diseases at FDA: A Successful Year for Orphan Products
Universities Fear the Federal Research Funding Pipeline Is Withering

Grant Opportunities


RGI Launches Rare Disease Research Challenge
Advancing Translational and Clinical Probiotic/Prebiotic and Human Microbiome Research (R01)
Eunice Kennedy Shriver NICHD Cooperative Multicenter Neonatal Research Network (UG1)
Big Data to Knowledge (BD2K) Advancing Biomedical Science Using Crowdsourcing and Interactive Digital Media (UH2)


News from ACTS


Share your exciting stories...Membership News!

Does your institution have news you want to share with the ACTS Connection readership? Do you have an investigator doing something innovative? Let us know! From innovative projects, star scholars and trainees, to award winning faculty, help us share the news that is important to you by submitting it through our online form.

Your news may be shared on the ACTS website, and other ACTS social media sources!
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Translational Science News


NIH-Led Effort Launches Big Data Portal for Alzheimer's Drug Discovery

The National Institutes of Health (NIH) announced the launch of a new Alzheimer's Big Data portal as part of a public-private partnership to transform and accelerate drug development in the field. The portal is part of the Accelerating Medicines Partnership (AMP), which brings together NIH, Food and Drug Administration, industry, and academic scientists to help translate knowledge faster and more successfully into new treatments. The launch of the AMP-AD Knowledge Portal, along with the release of the first wave of data, will allow for sharing and analyses of large and complex biomedical datasets. "We are determined to reduce the cost and time it takes to discover viable therapeutic targets and bring new diagnostics and effective therapies to people with Alzheimer. That demands a new way of doing business," said NIH Director Francis S. Collins, MD. "The AD initiative of AMP is one way we can revolutionize Alzheimer research and drug development by applying the principles of open science to the use and analysis of large and complex human data sets."

From "NIH-Led Effort Launches Big Data Portal for Alzheimer's Drug Discovery"
NIH News (03/04/15)
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Novel Financing Technique May Unlock Funding for Developing 'Orphan' Drugs to Treat Rare Diseases

A new study published in the journal Science Translational Medicine discusses the potential of a new financing approach for reducing risks associated with developing orphan drugs. The study was part of a collaboration of scientists at the National Center for Advancing Translational Sciences (NCATS) and economists at the MIT Laboratory for Financial Engineering. It demonstrated how megafunds, or large diversified portfolios of biomedical projects, can offer attractive returns to investors while reducing some of the risks involved in investing in early stage exploration of new medicines for rare diseases. The authors used data from two of NCATS' rare diseases programs to evaluate the risks and rewards of a simulated portfolio, comprising data from 28 active projects funded and currently managed by NCATS. See the rest of the study at the link: Financing translation: Analysis of the NCATS rare-diseases portfolio

From "Novel Financing Technique May Unlock Funding for Developing 'Orphan' Drugs to Treat Rare Diseases"
News-Medical.Net (02/28/15)
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Rare Diseases at FDA: A Successful Year for Orphan Products

Gayatri Rao, director of the Food and Drug Administration's Office of Orphan Products Development (OOPD), commemorated Rare Disease Day on February 28 by highlighting achievements in rare diseases over the past year. For instance, in 2014 the OOPD received 440 new requests for orphan drug designation, compared with less than half that number seven years ago. The OOPD also designated and approved more orphan drugs in 2014 than in previous years, with nearly 300 drugs designated and 48 approved, including both novel and repurposed drugs. Forty-one percent of all novel new drugs approved by the Center for Drug Evaluation and Research in 2014 were for the treatment of rare diseases. Many of these orphan drug approvals were innovative, including Sylvant to treat Castleman’s disease and Impavido to treat forms of a tropical disease called leishmaniasis. Meanwhile, the Center for Biologics Evaluation and Research approved for the first time a device through the Humanitarian Device Exemption pathway. This device, the CliniMACS CD34 Reagent System, helps control potentially serious immune reactions associated with stem cell transplantation in patients with acute myeloid leukemia. Additionally, the FDA launched an agency-wide blueprint to accelerate the development of therapies for pediatric rare diseases. Last year also saw the issuance of the first rare pediatric disease priority review voucher for the treatment of a rare lysosomal storage disease called type IVA (Morquio A syndrome) that affects about 1,000 patients nationwide.

From "Rare Diseases at FDA: A Successful Year for Orphan Products"
FDA Voice blog (02/27/15) Rao, Gayatri R.
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Universities Fear the Federal Research Funding Pipeline Is Withering

Around the country, university leaders are concerned about the level of federal investment in basic research. They say the government needs to spend more on discoveries with unknown and long-term payoffs. "You'll have no short-term results 20 years from now if you don't have enough in the basic research pipeline," warns Kimberly Andrews Espy, senior vice president for research and discovery at the University of Arizona, which received about $338 million in federal research and development funding in 2013. She was part of a group of research leaders from the Association of Public and Land-grant Universities who in February met with federal lawmakers to express their concerns about research funding. The sequester that triggered automatic spending cuts in 2013 is particularly taking a toll, and the upsurge in research spending linked to the 2009 stimulus law has run out. The university leaders support President Obama's proposal to reverse the sequester, but it is uncertain whether his proposal will be endorsed by the Republican-led Congress.

From "Universities Fear the Federal Research Funding Pipeline Is Withering"
Washington Post (02/25/15) Anderson, Nick
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Grant Opportunities


RGI Launches Rare Disease Research Challenge

The Rare Genomics Institute (RGI) has teamed up with 18 technology firms and research services organizations to support rare disease research. The Helping Empower and Accelerate Research Discoveries (BeHEARD) challenge is a global competition that award technology products and services worth more than $600,000 to researchers involved in rare disease research. "For many rare diseases, there are not sufficient resources dedicated to advance the research towards diagnostics, therapies, and cures," RGI President Jimmy Lin said. "By partnering with top biotechnology companies, we are providing rare disease groups access to the latest life sciences innovations and technologies." Researchers must submit proposals including rationales and plans for each technology for which they are applying. These include mouse models, drug repositioning services, bioinformatics analysis services, and CRISPR cell lines. Research proposals are due by May 17, 2015. More information about the program is available at BeHEARD 2015

From "RGI Launches Rare Disease Research Challenge"
GenomeWeb News (03/03/15)
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Advancing Translational and Clinical Probiotic/Prebiotic and Human Microbiome Research (R01)

The National Institutes of Health has issued a funding opportunity announcement (FOA) for Advancing Translational and Clinical Probiotic/Prebiotic and Human Microbiome Research (R01). The announcement aims to accelerate translational and clinical Phase I and II a/b safety and efficacy studies for substantiating measurable functional benefits of probiotic/prebiotic components and/or their combinations, and to understand the underlying mechanisms of their actions, and variability in responses to these interventions. The FOA will involve interdisciplinary collaborations across scientific disciplines engaged in microbiome and pro/prebiotic research, including nutritional science, microbiology, virology, microecology and microbiome, genomics, immunology, computational biology, chemistry, bioengineering, as well as integration of omics and computational approaches in DNA technologies. The deadline for applications is June 5, 2015.

From "Advancing Translational and Clinical Probiotic/Prebiotic and Human Microbiome Research (R01)"
NIH Grants (02/26/15)
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Eunice Kennedy Shriver NICHD Cooperative Multicenter Neonatal Research Network (UG1)

The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) has issued a funding opportunity announcement for the Cooperative Multicenter Neonatal Research Network (UG1). NICHD is inviting applications from researchers willing to participate with it in clinical trials and observational studies under a cooperative agreement in an ongoing multi-center clinical program designed to investigate problems in neonatal medicine. The program aims to facilitate the advancement of neonatal care by establishing a network of academic centers that, with rigorous patient evaluation using common protocols, can study the required numbers of patients and can provide answers more rapidly than individual centers acting alone to provide evidence to guide clinical practice. Areas of potential projects include trials of agents and strategies to improve short-term and long-term outcomes for infants, and observational studies of areas where there is little or no evidence available for clinical management. NICHD plans to commit $4.88 million in fiscal year 2016 to fund up to 18 awards. Applicants may request up to $200,000 in direct costs per year, with a maximum project length of five years. The application deadline is June 3, 2015.

From "Eunice Kennedy Shriver NICHD Cooperative Multicenter Neonatal Research Network (UG1)"
NIH Grants (02/23/15)
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Big Data to Knowledge (BD2K) Advancing Biomedical Science Using Crowdsourcing and Interactive Digital Media (UH2)

The National Institutes of Health (NIH) has issued a Big Data to Knowledge funding opportunity announcement to support the development of interactive digital media that engages people in performing some aspect of biomedical research via crowdsourcing. Each application should pose a challenging biomedical research problem and propose the development of interactive digital media that uses crowdsourcing as a key part of how the problem is solved. Areas of biomedical research that are amenable to interactive digital media and crowdsourcing approaches include biomedical text comprehension, transcription, and parsing; biomedical data enrichment; biological complexity; biomedical image analysis; feature extraction; structural biology; biomedical network analysis; categorization; language parsing and interpretation; and elucidating disease evolution and propagation. For fiscal year 2016, NIH plans to commit $2.5 million for an estimated 6-10 awards. Applications are due by June 3, 2015.

From "Big Data to Knowledge (BD2K) Advancing Biomedical Science Using Crowdsourcing and Interactive Digital Media (UH2)"
NIH Grants (02/23/15)
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