Cerdelga, a treatment for Gaucher disease type 1 developed at the U-M Medical School and licensed to Genzyme Corp., has been approved by the U.S. Food and Drug Administration.  

Cerdelga represents the first class of chemical entities conceived and developed at U-M to achieve FDA approval. An oral medication, Cerdelga offers an alternative to intravenous enzyme replacement, the other approved Gaucher disease treatment.

 
The strategy of treating Gaucher disease by inhibition of glycolipid synthesis was proposed by the late Norman Radin, Ph.D.,a U-M neurochemist, more than 40 years ago. The work directly leading to Cerdelga began 25 years ago when James Shayman, M.D., a nephrologist trained in lipid biology and pharmacology, sat down with Radin and began their collaboration.

Their shared work continued at least until 1995 after which Radin retired, but research continued by the Shayman group to design, synthesize and test glycolipid synthesis inhibitors. That included a series of "proof of concept studies" in experimental models of lysosomal storage diseases. Patents covering these compounds and related inventions were licensed to Genzyme, a Sanofi company, in 2000 for clinical development.

Gaucher disease is an inherited disorder that affects many of the body's organs and tissues. More than 10,000 patients worldwide are diagnosed with Gaucher disease.

"On a personal level, the success of this research program has been tremendously gratifying," Shayman said. "The FDA approval of Cerdelga provides further motivation for me and my collaborators to bring other drugs through the pipeline."

University of Michigan Health System (UMHS) leadership recently approved a big-data analytic platform purchase from IBM, alongside a product development partnership with IBM Research, targeting clinical decision support tools for critical care.

The approval is for a critical care pilot program, facilitated by the U-M Center for Integrative Research in Critical Care (MCIRCC) that will test the clinical viability of its analytic methodologies coupled with the IBM technology. The volume, variety, and velocity of patient data generated with critical care delivery provides the ideal test environment to push the platform's capabilities. If successful, this clinical decision support tool could transition beyond critical care into comprehensive hospital to home care models.

The goal of this platform is to support scalable translational research and real-time product development to deploy solutions within UMHS and to external customers that are precise, predictive, proactive, and personalized for high impact.  This has the potential to greatly impact the institution's other industry partnerships, grant opportunities, and most importantly patient care.

The proposal was a joint effort between MCIRCC, Medical Center Information Technology, Medical School Information Services, Fast Forward Medical Innovation, Michigan Institute for Clinical and Health Research, the Medical School Office of Research, U-M Hospitals and others.

Questions about this new partnership with IBM? Contact Janene Centurione at jrcentu@umich.edu. 

GlaxoSmithKline (GSK) is inviting U-M investigators to submit proposals for funding by the company's Discovery Partnerships with Academia (DPAc) program.   

Mark your calendars for November 19 as the Fast Forward Medical Innovation team welcomes Dr. Uday Kumar for an event titled "Transforming Medicine Through Innovation and Design Thinking."

Wednesday, November 19
12:00pm-1:00pm
Dow Auditorium, Towsley Center
FREE box lunch

"Design thinking" in biomedical innovation is a problem-solving process that puts the patient first. Dr. Kumar will discuss his experience as a clinician who saw problems in practice, and then developed an innovation framework by analyzing the needs of his patients. It will be an informal Q&A session where he will also dive deeper into the challenges and rewards of being a clinician-inventor, and how he made the leap to becoming an entrepreneur.  

RSVP for this FREE event.

The U-M Michigan Translational Research and Commercialization for Life Sciences (MTRAC) program recently closed its second annual RFP, with 29 new proposals submitted.  

Projects range from a device for stroke emergency treatment to a glaucoma decision support tool to a cancer therapeutic. Project teams will now spend the next few months preparing their "pitches" for presentation to the MTRAC oversight committee in January.

Kickstart, MTRAC's "early-early stage" program has funded 11 projects, and the year one class of MTRAC teams are progressing through their milestones, such as participating in training sessions like the recent "Value Proposition" class offered by FFMI's Commercialization Education program.  

"Project teams are progressing nicely," noted Brad Martin, Commercialization Program Director. "We have a few that will be forming companies in the near future."

Questions about MTRAC? Contact Brad Martin at bradmart@umich.edu.

FFMI is participating in a pilot digital badging initiative designed to build and support the continuing education and professional development of the U-M Medical School's research community.

Digital badges recognize U-M faculty who are actively exploring the commercialization potential of a biomedical technology. In this pilot phase, faculty who successfully complete our i2i: Early Stage Technology Development course will receive a Research Commercialization - Explorer badge issued by the UMMS Office of Research and managed by Credly.

Digital badges complement your formal training by emphasizing a more complete picture of your skill set. They make meaningful achievement and experiences visible to anyone, anywhere, anytime on the web including current employers, instructors, peers, and communities of shared interest.  

You can easily manage your badges digitally by adding them to your online CV or email signature, and by sharing and displaying them with your network through platforms like LinkedIn, Facebook, and Twitter.

Questions about digital badges offered by the U-M Medical School? CLICK HERE for more details or contact ummsdigitalbadges@umich.edu.