I got to host the first block of the second morning, which I introduced as “The Translation Block.”  Not talking about language translation here, the term Translation in this context refers to that potentially long and expensive process to get from “good idea” to “Something your doctor can give you a prescription for.”  The four sessions in this block started off with  an excellent lessons learned discussion by Mark Bear http://www.fraxa.org/ra_Bear.aspx which Geraldine captured succinctly in another blog post, so I will add my comments on his session to her post rather than expound here, suffice to say we are very grateful that he came to share his experience with us. 

The second session was a short “recruiting” speech by our own Megan O’Boyle for her afternoon breakout session on the registry questionnaire.  For those of you who haven’t yet heard, our foundation has signed a contract with a registry provider and we are now in the final stages of creating what we hope is a comprehensive questionnaire to capture all medically relevant information about the symptoms of the syndrome to enable the next step in research.  Megan has done a boatload of work over the past year and a half both to find and to secure the right professional registry provider, and now she is hashing through all the options to determine what gets asked.  It made a lot of sense to use this opportunity to capture the input of the researchers attending (since they will be a major user of the summarized data), so Megan led a session to ask them. 

The third session was a presentation by Christine Mueller of the FDA’s Office of Orphan Product Development.  she presented a review of the Orphan Drug Act of 1983 and the incentives it provides to entice pharamceutical companies to promote the translation of therapies for rare diseases like PMS. 

The fourth and last session on Translation was a presentation by Chris Winrow on the challenges associated with Neuroscience Drug discovery.  His data showed that only about 11% of all Neuroscience Drugs survive the development process; there are major hurdles that include the fact that mice are “very close” to humans genetically but “worlds apart” neurologically, and the basic issues that we still face in terms of fundamental lacks of understanding as to how the human brain actually works.  In terms of personal growth I got to learn a whole new vocabulary of keystone concepts in this field:  Toxicity tests to see how much the body can stand; Efficacy measures to quantify the degree of improvement a drug provides in relation to dosage; Affinity which I took to mean how well the drug goes to the place it is supposed to; and about a dozen others.  One of the more interesting concepts was “Subjectivity”, which means “Does the drug make you feel better”.  The example Chris used was a hypothetical sleep aid that might make you go to sleep quicker and sleep more soundly, but if you wake up feeling like crap it would be a bust on the subjectivity scale because no one would buy it (or at least no one would buy it twice).

Lots of food for thought.