The Science: Translational Medicine Platform
The success of our discovery biology program has provided us with a plethora of opportunities for further validation of the more than 150 myelin repair targets identified in our laboratories. At this time we have two development paths before us:
- Validate the most promising novel myelin repair therapeutic targets through contract research organizations.
- Investigate the potential for repositioning of more than 40 targets that are in clinical development by various companies for other indications.
Designed to improve the odds of successful clinical trials, our translational medicine platform will ensure that to the extent possible there is consistent and concordant data in cell cultures, multiple animal models and assays, and in human tissue.
An analysis (Figure 1) of publicly available data conducted by our Drug Discovery Advisory Board of biopharmaceutical experts is helping to prioritize the overwhelming number of targets generated in our funded discovery biology laboratories. The blue area shows those discoveries that are entirely novel. The dark green area shows the overlap of those discoveries with those in the Validated Targets and Pathways Database (VTPD) that are in development, often at a number of competing companies. Source: Validated Targets and Pathways Database
(Figure 2) These are documented programs under investigation by various companies on a subset of Myelin Repair Foundation targets.
Understanding myelination in humans
Though our discovery biology program has successfully isolated many of the triggering mechanisms for myelin repair, our findings have so far been made in animal cell cultures and animal models. Today there are no test systems available for such studies in human brain tissue. Studies to develop these test systems are part of our translational medicine platform and will provide essential criteria for the approval of clinical trials for myelin repair.
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Your gift today will help us reach the first myelin repair Phase I Clinical Trial by 2014.
