May TRAIN E-NEWS
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Innovator Spotlight: Adenoid Cystic Carcinoma Research Foundation
When Jeff Kaufman's wife was diagnosed with adenoid cystic carcinoma, the couple couldn't find an organization to support that was focused solely on this rare disease.
Research Agenda featured as new Innovation Tool
View the Adenoid Cystic Carcinoma Research Foundation's Research Agenda, which covers guiding principles, project categories, specimen donations, biobanks and patient databases, model development, basic research, diagnostic and prognostic biomarkers, drug screening, and clinical trials.
View archive of Webinar on engaging with FDA
Learn from the experiences of senior leaders of two foundations that are pioneers in engaging in product development and in working with the FDA.
fastercures' updates
Global Conference features health issues
View summaries from this year's Milken Institute Global Conference, which included sessions on accelerating medical innovations, investment opportunities in healthcare, and transformative technologies:
• Accelerating Innovation in the Medical Industry
• Your Health: On Your Phone and in the Cloud
• Financial Innovations for Early-Stage Drug Development
• Weight of the World: Strategies to Fight the Global Obesity Epidemic
• Getting to 100: Your How-To Guide
• Getting From Care to Cure
• Cancer Prevention: What Will It Take?
• The Longevity Economy: An Aging Population Creates Worldwide Market Opportunity
• Investment Opportunities in Health Care - Whatever the Supreme Court Decides
• The Human Genome: A User's Guide
New look for FasterCures' Web site
FasterCures has debuted a redesigned Web site so that it is easier for users to find information about our programs, issues, publications, and more.
train organization updates
Michael J. Fox Foundation and Sanofi collaborate on clinical study
Under the terms of the collaboration, MJFF will sponsor a phase I b clinical trial to assess the safety and tolerability of AVE8112 in patients with Parkinson's disease. All data and results generated by the clinical trial will be owned by MJFF and shared with Sanofi. Further development plans will be based upon the results of the study.
MJFF/Sanofi collaboration featured in Economist article on venture philanthropy
A shortfall in R&D within big pharma is inspiring a lot of new partnerships, and charities have been particularly bold.
Organizations collaborate to support IND-enabling drug development activities
The Drug Discovery Center of Innovation (DDCOI), the Foundation Fighting Blindness (FFB), and the National Neurovision Research Institute have completed a strategic Master Services Agreement in which DDCOI will provide pharmaceutical development oversight and strategic expertise to support IND-enabling drug development activities for MitoChem Therapeutics, LLC, which is supported in part by a Small Business Award from FFB.
MMRF and TGen announce molecular profiling and biobanking collaboration for landmark personalized medicine study
Under the agreement, TGen will provide a central hub where patient samples will be analyzed using an unprecedented breadth of genomics platforms. The data to emerge from this work will provide the most comprehensive view of myeloma at the molecular level and will enable the research community to better understand what drives a patient's response to treatment or disease progression and will also generate new leads for targeted drug development.
AVI BioPharma announces top-line results for exon skipping trial
"We are very encouraged by the results of this first placebo-controlled study investigating exon-skipping technology in DMD," said Chris Garabedian, president and CEO of AVI BioPharma. "Eteplirsen represents the first drug candidate for DMD to demonstrate the production of novel dystrophin in a robust and consistent manner and these study results support advancing eteplirsen into a pivotal study." The research was funded in part by nonprofit funder CureDuchenne.
Tuberous Sclerosis Alliance releases white paper assessing scientific progress and research needs
"Our goal with this white paper was to identify key areas of TSC [tuberous sclerosis complex] clinical care and research where significant progress has been made, areas where research is needed, the barriers to progress in TSC research, and the unmet clinical needs of people with TSC," explains TS Alliance President & CEO Kari Luther Rosbeck.
News you can use
patientINFORM brings together publishers of leading medical journals and voluntary health organizations (VHOs) on access to research for patients and caregivers
VHOs identify relevant literature, summarize it on their Web sites, and link to the articles for free. VHOs interested in participating in patientINFORM should contact Margaret Reich at patientinform@gmail.com.
SMARTT program accelerates translation of therapeutic agents
The National Heart Lung and Blood Institute's SMARTT (Science Moving towards Research Translation and Therapy) program supports translation of novel discoveries into successful new therapies for heart, lung, and blood diseases by providing confidential and rapid preclinical development services.
1000 Genomes Project data available on Amazon cloud
The world's largest set of data on human genetic variation, produced by the international 1000 Genomes Project, is now publicly available on the Amazon Web Services cloud.
Broad Institute creates GenomeSpace to make data more accessible to researchers
GenomeSpace is a cloud-based, open-source data management center that offers "a lightweight layer of interoperability." GenomeSpace supports several bioinformatics tools, all integrated to allow easy accessibility, easy conversion, and frictionless sharing.
Research yields clues to personalized cancer therapy
Two research teams searched through their compendia of data to create the most complete picture yet of how mutations can shape a cancer's response to therapy, suggesting that the effectiveness of most anticancer agents depends on the genetic make-up of the cancer against which they are used.
Patent information to be made publicly accessible
The latest effort to provide free access to chemical information should make it easier for chemists to see which bioactive molecules have drawn the attention of the drug industry and to explore new drug targets by designing compounds that are not named in patents.
Wall Street Journal Health Blog features blueprint for 'DIY clinical trials'
Amy Dockser Marcus profiles "citizen scientist" Melanie Swan and her effort to professionalize do-it-yourself science.
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