Patient-Reported Outcomes and the Elephant in the Conference Room | Calling all change-makers: Reserve your spot at Partnering for Cures | Lab-created organoids yield insights into how the brain works
April 27, 2017
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Patients' willingness to take risks aided development of life-saving drug
People with chronic myeloid leukemia endured repeated blood tests and bone marrow biopsies as Dr. Brian Druker and his team honed in on the right dosage of an experimental compound that would become Gleevec. Druker and three patients describe their experiences.
STAT (tiered subscription model) (4/25) 
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News from FasterCures
Patient-Reported Outcomes and the Elephant in the Conference Room
Patient-reported outcomes (PROs) are an important part of patient-centered research and care. But across the spectrum, the applications of PROs are varied and complex. At FasterCures, we see great potential in more effectively using PROs to advance the science of patient input. This year we are launching a new project to help provide a wider angle view of PROs and to reveal the big picture of their potential. Learn more in our most recent blog post.
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Calling all change-makers: Reserve your spot at Partnering for Cures
Partnering for Cures (P4C) is the go-to meeting for accelerating patient-centered solutions and creating meaningful collaborations. FasterCures is excited to convene P4C 2017 in two biomedical innovation hubs, Boston and San Francisco. Register to attend and make connections with decision-makers from across diseases who are motivated by the same mission -- to reduce the time and cost of getting new therapies from discovery to patients.
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Science and Technology
Lab-created organoids yield insights into how the brain works
Clusters of human brain cells grown in a lab from skin stem cells are allowing scientists to learn how the brain grows. Scientists studying the clusters -- also called minibrains, organoids or spheroids -- report in Nature that a genetic mutation linked to both autism and epilepsy prevented developing cells from migrating normally.
National Public Radio (4/26) 
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Artificial womb succeeds in lamb study
Artificial womb succeeds in lamb study.
(Christopher Furlong/Getty Images)
An artificial womb developed at Children's Hospital of Philadelphia allowed premature lambs to survive and mature normally outside the mother's womb for about a month, according to a report in Nature Communications. The device, which may allow very premature babies to survive, is made up of a clear plastic bag filled with synthetic amniotic fluid and is attached to an external machine that provides nutrition and oxygen and removes carbon dioxide.
The Washington Post (tiered subscription model)/The Associated Press (4/26),  National Public Radio (4/25), (4/25) 
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Cheap generic could prevent thousands of maternal deaths
Tranexamic acid can stop postpartum hemorrhage, which kills some 100,000 women every year and forces emergency hysterectomies in low- and middle-income countries, according to the results of a six-year study in The Lancet. The drug was invented in the 1950s, costs less than $2 per dose, does not need to be refrigerated and reduced the rate of maternal bleeding deaths by a third when given within three hours.
The New York Times (free-article access for SmartBrief readers) (4/26) 
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Finance and Economics
Heavy hitters fund development of monoclonal antibody combo
The Bill & Melinda Gates Foundation, GV (formerly Google Ventures) and Alexandria Venture Investments led a Series D financing round that raised $45.5 million for Arsanis, which is moving an experimental treatment for Staphylococcus aureus pneumonia into Phase II testing. The drug, ASN100, combines two monoclonal antibodies, and the company said it will use the fresh funds to study other monoclonal antibodies for multi-drug resistant Escherichia coli and Klebsiella pneumoniae, Streptococcus pneumoniae and respiratory syncytial virus.
Xconomy (4/26) 
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CDC awards $3M grant to UNC, Duke for Zika virus diagnostics
The University of North Carolina at Chapel Hill and Duke University received more than $3 million from the CDC to develop better diagnostics for the Zika virus, which can be difficult to differentiate from dengue and similar viruses. Researchers will work with the CDC "to understand the finer points of the human antibody response, particularly humans that are experiencing Zika or multiple flavivirus infections sequentially over the course of their lives," said Matt Collins, whose virology lab at UNC will participate in the research.
WCHL-FM (Chapel Hill, N.C.) (4/25),  WUNC-FM (Chapel Hill, N.C.) (4/25) 
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Cancer research center in Va. awarded $15M grant
The University of Virginia Cancer Center has been awarded a $15 million grant from the National Cancer Institute to develop new treatments for cancer. The funds will go toward more research, recruitment of faculty and clinical trials.
WVIR-TV (Charlottesville, Va.) (4/26) 
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Forge Therapeutics raises $15M
A large financing round has raised $15 million for San Diego-based Forge Therapeutics. The proceeds will be used to advance the company's gram-negative antibiotics program and will allow it to move out of its incubator space at Johnson & Johnson's JLABS.
Chemical & Engineering News (4/25) 
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Policy and Regulation
FDA aims to broaden participation in oncology clinical trials
The FDA is developing guidance meant to encourage sponsors of clinical trials for cancer treatments to enroll a more-representative population of participants, including children and people with HIV, say Richard Pazdur, director of the FDA's Oncology Center of Excellence, and Julia Ann Beaver, associate director of the FDA's Office of Hematology and Oncology Products. The FDA is working with stakeholders including Friends of Cancer Research and the American Society of Clinical Oncology to develop the guidelines, Beaver said.
Bloomberg BNA (free content) (4/21) 
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Society and Ethics
Medical breakthroughs unlikely without publicly funded research
Basic science research is key to discovering new drugs, but the biopharmaceutical industry already invests heavily in research and development and is unlikely to make up the difference if Congress complies with President Donald Trump's request to slash the NIH's budget, writes John LaMattina. "We are in a time of tremendous possibilities in the area of biomedical research. What is needed is more money devoted to basic research, not less," writes LaMattina, the former president of Pfizer Global Research and Development.
Forbes (4/27) 
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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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