Join #FasterCures Tweet Chat on research ethics this Thursday | Blog: Young scientists, partnerships and resources among the focus of final 21st Century Cures Initiative roundtable | Scientific advances fuel refined approach to trials, as patient access opens up
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September 16, 2014
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Stem cell research offers hope, hype
As many as 4,500 clinical trials are underway in the U.S. testing stem cell-based treatments on Parkinson's disease, HIV, diabetes, spinal cord injuries, heart disease, blood cancers, blindness and other conditions. Stem cell technology also helps researchers identify potential drug targets, including one for amyotrophic lateral sclerosis. Initial safety trials have been positive, but there is no evidence supporting stem cell therapy for skin conditions or bunions, for example, and offering these treatments not only suggests that the research is further along than it really is, it might put patients at risk, some experts say. The New York Times (tiered subscription model) (9/16)
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News from FasterCures
Join #FasterCures Tweet Chat on research ethics this Thursday
Join FasterCures for a Tweet Chat on Thursday, Sept. 18, from 1 p.m. to 2 p.m. on research ethics in a modern world. What can online conversations teach traditional R&D, and how is social media changing what is ethical? Answering those questions and more posted by the audience will be Susannah Fox of the Pew Research Center's Internet Project, Michelle N. Meyer of Harvard, John Wilbanks of Sage Bionetworks and Margaret Anderson of FasterCures. Join the conversation at
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Blog: Young scientists, partnerships and resources among the focus of final 21st Century Cures Initiative roundtable
Investing in the next generation of scientists, incentivizing partnerships, exploring new approaches to clinical trials and ensuring stable funding for research were among issues top of mind for nearly two dozen members of Congress at an Energy and Commerce Committee roundtable last week. Led by Chairman Fred Upton and Rep. Diana Degette, the roundtable caps a four-month effort to gather solutions designed to accelerate the path to cures, the focus of the Committee's 21st Century Cures Initiative. The roundtable featured top health leaders, including Health and Human Services Secretary Sylvia Burwell, National Institutes of Health Director Francis Collins, Food and Drug Administration Commissioner Margaret Hamburg, and FasterCures Founder and Chairman Michael Milken. Read the blog.
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Science and Technology
Scientific advances fuel refined approach to trials, as patient access opens up
Cancer trials in the U.S. have been changed radically in recent years as advances in biology and research contribute to improved study design, and the Internet offers patients ready access to information about clinical trials. Patients are discovering studies of new drugs online, sometimes looking into them before consulting their doctors. Meanwhile, studies are becoming refined as data show fewer patients are necessary to support findings. The Philadelphia Inquirer (9/15)
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PCORI projects leverage technology to improve outcomes
The Patient-Centered Outcomes Research Institute has a number of projects leveraging health data to improve clinical outcomes, writes Sarah Greene, an associate director at PCORI. Initiatives include developing a research database of information gathered from clinics and stored in standardized, interoperable, secure formats; a study to test how well specialty care can be delivered via videoconferencing to Parkinson's patients; and a study comparing conventional booklets with tablet computer-based information to help men navigate prostate cancer treatment options. Government Health IT online (9/15)
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Finance and Economics
Funding crunch tempts researchers to cut corners
The scramble for scarce medical research funding in the U.S. is prompting researchers to cut corners, which is affecting seriously ill people including those with amyotrophic lateral sclerosis, Richard Harris writes. Researchers are tempted to oversell weak findings, National Institute of Neurological Disorders and Stroke Director Story Landis said. Researchers have little incentive to verify results from other laboratories, American Society for Cell Biology Executive Director Stefano Bertuzzi said. National Public Radio/Shots blog (9/15)
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From hearts and bones to eyes and ears: VCs shift focus
Venture firms are investing more in treatments for blindness and hearing loss, according to The Wall Street Journal. Meanwhile, they are "pulling back from two historical leaders, heart and orthopedic conditions." The Wall Street Journal (tiered subscription model)/Briefly blog (9/14), The Wall Street Journal (tiered subscription model) (9/15)
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Public-private partnership to tackle African diseases
A public-private partnership that includes GlaxoSmithKline along with the U.K. and South African Medical Research Council will study noncommunicable diseases in Africa with more than $8 million contributed by the three entities. The agreement "signifies growing agreement among the scientific community that collaboration is key to defeating some of the world's biggest health problems," said Patrick Vallance, GSK's president of pharmaceuticals, research and development. (U.K.) (9/10)
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NIH gives $64M to support development of cell signature network
Six research institutions in the U.S. will receive a portion of a $64 million NIH grant to create the Library of Integrated Network-based Cellular Signatures, which will collect, analyze and store cellular functions and molecular activities for research purposes. The institutions are Harvard Medical School, Oregon Health and Science University, two Broad Institute teams, Icahn School of Medicine at Mount Sinai and the University of California, Irvine. GenomeWeb Daily News (free registration) (9/12)
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Policy and Regulation
Ice Bucket Challenge should be cold water in Congress' face
The amount the NIH spends per patient on amyotrophic lateral sclerosis research is one-tenth the amount spent on arthritis research, though ALS is far more deadly, writes Robert Sepucha, whose father died of ALS and who serves on the board of directors at ALS Therapy Development Institute. The overwhelming success of the Ice Bucket Challenge shows Americans' willingness to pay for the basic research the NIH supports, but NIH funding adjusted for inflation is 25% lower today than it was in 2003, and Congress has refused to reverse the trend, Sepucha writes. "The result is that the critical bench science that can lead to medical breakthroughs is either being done in other countries, or simply not at all," Sepucha writes. Roll Call (free content) (9/10)
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Society and Ethics
Oncologists debate consequences of emphasis on screening
An emphasis on cancer screening and increasingly sensitive technologies help detect cancer at very early stages, but they may also be leading to detection and treatment of abnormalities that would never cause harm, while failing to place adequate emphasis on the most serious diseases, experts say. A National Cancer Institute advisory panel is calling for new guidelines for cancer diagnosis, treatment and terminology; screening programs that focus on finding the most dangerous cancers; registries to monitor lower-risk malignancies; and calling slow-growing, precancerous tumors "indolent lesions of epithelial origin" instead of "cancer." The Wall Street Journal (tiered subscription model) (9/14)
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Why the focus on drug prices is missing the point
The U.S. health care system is not set up to pay for expensive cures, and that should change, Sylvia Pagán Westphal writes. She recommends value-based pricing, changes to Medicaid's "best-price" rule and initiatives that would allow the amortization of treatment costs over years. The Boston Globe (tiered subscription model) (9/14)
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On the FastTrack
Register for tomorrow's webinar on crowdfunding in medical research
With today's patients and their supporters more engaged than ever in raising both funds and awareness for promising science, crowdfunding has become an increasingly popular vehicle among bioscience entrepreneurs and philanthropists. Register today for a webinar tomorrow, Sept. 17, from 1 p.m. to 2 p.m. Eastern, where an expert panel will discuss some of the most interesting crowdfunding models and outcomes, and address the potential for impact on a larger scale. Register today.
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FasterCures is an action tank that works across sectors and diseases to improve the effectiveness and efficiency of the medical research enterprise. FasterCures, a center of the Milken Institute, is nonpartisan and independent of interest groups.
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