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Professionals Track at MDF Annual Conference a Success

For the first time, the 2016 MDF Annual Conference included a closed, professionals-only track focused on providing updates on basic scientific discovery and efforts focused on therapy development. Over 130 academic, industry and federal agency professionals participated in a variety of sessions from the Drug Development Roundtable to updates on studies and scope for the Myotonic Dystrophy Clinical Research Network and early stage therapies. Read more below.

Drug Development Roundtable (DDR):

The goal of the DDR is to bring together stakeholders in academics and industry in order to address common challenges and opportunities in therapy development for DM. By working together on pre-competitive space activities, such as endpoint development and biomarkers, collective efforts can ease the path to new therapies.

At the 2016 MDF Annual Conference, the DDR considered the topic of what constitutes adequate rationale for launching clinical trials in DM. Ensuring that preclinical data and, when available, clinical data, support a specific target and candidate therapeutic will mean that clinical trials have a better chance to succeed. The DDR also discussed the topics of:

  1. Partnering between industry and academia on early stage drug discovery and development 
  2. The strengths and weaknesses of current mouse models of DM for testing candidate therapeutics 
  3. Preliminary data from a collaboration between Mayo Institute, Optum Labs and the MDF to identify the burden of DM1, including the most common medical procedures and reasons for hospitalization, as well as their associated costs.

Poster Session:

For the first time at the 2016 MDF conference, a poster session was held with 19 presentations made available to both the scientific and patient communities. Poster presentation topics ranged from novel approaches to understanding the mechanisms of DM to efforts to understand the impact of DM on the central nervous system. All MDF fellows in attendance presented posters, as did other academics and corporate researchers. For some MDF fellows, this was their first opportunity to interact with DM patients and their families.


The Myotonic Dystrophy Clinical Research Network (DMCRN) met to review progress in laying the groundwork for highly effective trials and lowering barriers for drug developers. Investigators reviewed progress in establishing effective means of working across the Network sites, building a natural history database and advancing a molecular biomarker panel toward regulatory qualification.

he DMCRN has made excellent progress in recruiting and retaining subjects in the natural history study and in standardizing data collection across sites. Collectively these efforts have helped shape the approach to be used during the next two years of Network operation, where an expansion to eight sites is supporting the collection of natural history data and genetic modifier identification in 500 DM subjects. 

Importantly, inclusion criteria for the new study will be very broad, ensuring involvement of as much of the DM community as possible. Discussions helped shape measurements and standard operating procedures to be used, focusing on keeping patient visits short (3 hours), with 3 visits over the 2-year data collection period. Strategies were discussed to optimize modifier identification from the 500-subject cohort.

Other Professional Track Presentations:

The Professional track focused on key issues in both drug development and patient care. MDF’s plans for development of a BAC transgenic DM1 mouse at Jackson Labs were presented, including the primary goals of having a better phenotypic model that is broadly available and with no intellectual property issues that might impede engagement from the biotechnology and pharmaceutical industries in DM.

Details and a progress report were presented on the development of a new molecular assay suitable for high throughput screening of small molecule libraries. Clinicians also were given advice on engaging with adult DM patients in discussions of sexuality.

A session focused on Early Stage Drug Development provided an update on Pfizer’s program to develop a small molecule drug targeting MBNL1, presented by Pfizer's Dr. Lauren Wood. Pfizer's assay development and initial small molecule screen is expected to be published in early 2017. Wave Life Sciences wasn’t able to present at the Conference but passed along information that they intend to apply their novel oligonucleotide backbone chemistry to DM.





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