A global effort led by the World Health Organization to control or eradicate neglected tropical diseases by 2020 has made progress since its founding in 2007, according to a report presented at the Global Partners' Meeting. The approach involves preventive chemotherapy, innovative disease management, vector control, veterinary public health, and provision of water and sanitation.
FasterCures recognizes a knowledge gap exists between what people understand about the R&D system and what the system asks them to do. A relatively small number of participants are highly engaged with the R&D system, while their peers haven't been informed about the complex data infrastructure supporting their health care. To help bridge the gap, FasterCures is developing a tool to help people understand what makes up their health data, how they are collected and shared, and who uses them. This project is funded through the Eugene Washington PCORI Engagement Award program. Learn more.
This year, we have been inspired by our locations to change up the way we conduct these meetings. Each meeting will be a carefully curated and highly interactive experience. In Boston, look for sessions on partnering locally for productivity and how the federal landscape affects the ecosystem. In San Francisco, we will explore how the tech industry is influencing life sciences and why medical research needs more data scientists. Register to attend today!
Sequencing the RNA of skeletal muscle biopsies from patients with undiagnosed disorders revealed disease-causing genetic mutations in 17 of 50 patients, researchers reported in Science Translational Medicine. The researchers identified disease-causing mutations in the dystrophin gene in patients with Duchenne muscular dystrophy, and severe collagen VI-related myopathy intron mutation that had been missed in DNA sequencing.
University of Wisconsin-Madison researchers are developing edible probiotic therapies, such as a pill or liquid that contains a customizable CRISPR message, to possibly treat infectious diseases caused by pathogens such as Clostridium difficile. The pill would contain bacteriophage that will deliver the CRISPR message to pathogens such as C. diff., resulting in the bacteria destroying its own DNA.
Chemist Martin Burke proposed at the recent American Chemical Society meeting that his colleagues embark on a mission to synthesize most of the world's known organic natural products made by microbes, plants and animals -- an undertaking Burke estimates could take 20 years and cost $1 billion. Identifying, isolating and testing natural products is slow and complicated, but Burke and his colleagues developed a machine that simplifies the process.
Researchers report in Science Advances that they corrected gene mutations that cause Duchenne muscular dystrophy using CRISPR-Cpf1 in mice and patient-derived stem cells. Function was at least partially restored in cardiomyocyte cultures, and the results show the potential of CRISPR-Cpf1 to correct disease-causing genetic mutations, experts said.
Two researchers are proposing that scientists receive equal shares of national research funds, then allocate half of the grant money they receive to colleagues they cite in their papers. The system "is not based on committees' judgments, but on the wisdom of the crowd," said ecologist Marten Scheffer, who is advocating for the system along with computer scientist Johan Bollen, who pioneered the idea of self-organized fund allocation.
The Bill & Melinda Gates Foundation, drugmakers and leaders of Western countries vowed continued support for the global effort to eradicate most neglected tropical diseases. With a "broader, deeper bench of investors," the goal of treating 90% of those who need it by 2030 is attainable, Bill Gates said.
Ohio will put $20 million toward research to fight opioid addiction, Republican Gov. John Kasich said in his State of the State speech. The money will come from Ohio's Third Frontier program and will go toward research to develop new ways to ease opioid withdrawal and relieve pain.
A group of economists proposed developing a system under which drug developers could purchase "exchange-traded FDA binary options" that would pay a specified sum if an experimental treatment fails to pass the FDA approval process. The options would "allow medical R&D investors to better share the pipeline risk associated with FDA approval with broader capital markets," the economists wrote.
Life science innovations increasingly come not from established drugmakers but from Apple, Google spinoff Alphabet and other big technology companies, writes John Nosta. The trend might be a wake-up call for Big Pharma, Nosta writes.
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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