New on TRAIN Central Station
Partnering for Cures connects hundreds of decision-makers from across the medical research system who are motivated by the same mission: to reduce the time and cost of getting new therapies from discovery to patients. We are excited to be in new cities this year and look forward to meeting new health innovators! Join us in Boston on July 12 or in San Francisco on Nov. 14.
News from FasterCures
Margaret Anderson says it’s time for a “health citizenship” movement in Science Translational Medicine
In a recent issue of the journal Science Translational Medicine, FasterCures' executive director, Margaret Anderson, introduces the concept of health citizenship. Anderson explains that FasterCures sees health citizenship as a two-way street: Individual citizens have a responsibility to engage in their own heath and in the biomedical research system, and “the system” has a responsibility to facilitate and value participant engagement.
What can the biomedical R&D ecosystem learn from the vast literature and learnings from outside the space about how to change organizational culture and behavior to build a more effective, efficient enterprise? Join us for the discussion Wednesday, March 29 at 2:00 p.m. ET.
Kim McCleary takes a look at recent developments in patient-centricity. These reflections come five years after Leonard Kish, a health IT strategy consultant, predicted that patient-centricity would be "the blockbuster drug of the century." The list highlights: climbing the adoption curve, progress toward objective evaluation, alignment on value, federal policy and growing respect for patient data.
FasterCures expands on findings from the “Expanding the Science of Patient Input: The Power of Language” report with “Four Steps to Advance the Conversation about Patient Engagement.” If we want to build the science of patient input, our language needs to be clear and easy to understand.
FasterCures kicked of 2017 by giving Webinar listeners a snapshot of the organization’s popular annual “Top Medical Research Issues and Trends to Watch in 2017” list and welcomed a trio of distinguished FasterCures senior fellows to offer their own expert insights. Access the session recording and summary on our website.
TRAIN Organization Updates
John Walsh, a dedicated patient advocate who co-founded and led the Alpha-1 Foundation and COPD Foundation, passed away this month. Walsh led the Alpha-1 Foundation for more than 20 years, and under his leadership the organization became internationally recognized for its commitment to people affected by Alpha–1 and driving for the cure by investing over $65 million in research and programs worldwide.
23andMe and The Michael J. Fox Foundation (MJFF) seek proposals for collaborative Parkinson’s disease research using the 23andMe research database and its genotyped cohort. Researchers may propose analyses to conduct with 23andMe scientists. Sharing of complementary data resources and application of new methodologies are highly encouraged.
The Chordoma Foundation Medical Advisory Board has recommended a new path for patients with advanced or recurrent chordoma to follow when seeking treatment, based on the availability of clinical trials testing new therapies.
The FDA announced that it has approved Spinraza (nusinersen) to treat spinal muscular atrophy (SMA), making it the first-ever FDA-approved therapy for SMA. Cure SMA’s efforts were vital to the approval of Spinraza by providing the first research funding needed to begin investigation into this therapeutic approach.
Exonics Therapeutics launches with funding from CureDuchenne Ventures to advance therapy for Duchenne muscular dystrophy
CureDuchenne Ventures, LLC, a subsidiary of the nonprofit CureDuchenne, has made a commitment of $5 million in initial seed funding that will allow Exonics Therapeutics to advance its preclinical research into developing gene editing technologies to permanently correct a majority of mutations causing Duchenne muscular dystrophy and other neuromuscular diseases.
News You Can Use
The FDA is considering establishing an "Office of Patient Affairs," to support and coordinate patient engagement across the agency. The FDA will collect comments on the proposal until June 7.
Vol-PACT Phase II is a three-year research partnership designed to develop new methods for analyzing digital images to more accurately measure cancer response and progression. The project is expected to review data from 12 different Phase II/III trials from multiple cancer types and therapies.