Scientists are collaborating with community health centers to recruit a participant pool for the Precision Medicine Initiative that reflects the nation's geographic, ethnic, racial and socioeconomic diversity. Study leaders hope to recruit 1 million participants, who will be asked to share genetic, medical, environmental exposure and lifestyle data with researchers.
You can now view the general schedule and topics for this year's Partnering for Cures meeting, which brings together hundreds of 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. More details will be added throughout the summer. Register today.
Volunteers working on the Open Insulin Project are trying to develop new ways to make insulin in an effort to boost worldwide supplies and accessibility, and the data they generate will be openly available. Success is a longshot, says former FDA Center for Drug Evaluation and Research Director Carl Peck, as the so-called biohackers lack the funding needed to bring a new drug to market.
Memorial Sloan Kettering Cancer Center and Medidata are conducting a study of 40 patients with multiple myeloma that involves using wearable monitors to track participants' activity and sleep patterns, and using apps to conduct quality-of-life surveys. "This is a first exercise and the hope is that we'd be able to take learnings from this exercise to the deployment of mobile health technologies," said Kara Dennis, managing director of mobile health at Medidata.
Scientists with Sichuan University's West China Hospital in August will launch a trial of a therapy that uses CRISPR-edited genes in patients with lung cancer previously treated with chemotherapy and radiation. The researchers will collect T cells from three study participants, eliminate a gene from the cells that encodes a protein named PD-1 using CRISPR-Cas9 technology and then reintroduce the modified cells.
Pfizer Venture Investments participated in a $17 million Series A funding round for startup Vtesse, a spinoff from the orphan-drug incubator Cydan Development. The company is developing a compound licensed from NIH for treating Niemann-Pick Type C1 disease.
Merck and Amgen recently invested through their venture capital arms in Akili Interactive Labs, a startup creating mobile video games designed to diagnose and treat various cognitive disorders. Akili intends to test its clinical-stage products on neurodegenerative diseases, such as Parkinson's disease and multiple sclerosis, to track disease progression and potentially help treat early-stage cases.
The NIH has awarded a $5.9 million grant to the University of California, San Diego, for its postdoctoral research and career development program. The funds will go toward training and supporting 15 young scientists.
Tech titans including Google, Apple, IBM and Microsoft are gathering, analyzing and storing people's health information, and the digitization of patient data promises to radically improve health care, but that promise will not be met if corporations continue to retain data in closed-loop systems, write Eric Topol and John Wilbanks, a FasterCures senior fellow. "[F]or such data to truly empower patients, customers must be able to easily send the information to their health provider, genetic counselor or any analyst they want," Topol and Wilbanks write.
The ALS Ice Bucket Challenge that encouraged people to dump cold water on their heads to raise money for the ALS Association was a success, raising $220 million worldwide for organizations without seeming to take away from other charitable giving, writes James Surowiecki. The campaign was a successful use of social media to reach younger audiences, but one that no other charity seems able to duplicate.
As more patients with pediatric diseases live longer, they are becoming their own advocates, and advocacy groups founded by parents are considering adult issues such as insurance coverage for assisted living, transportation and reproductive services. Some parent-run groups are at odds with patient-run groups as concerns diverge.
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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