It was great to see so many of you involved in the PSFs at the US and UK meetings this fall! Congratulations to Kim, Tracey, and all the hard-‐working and dedicated PSF Board Members for putting on two great events!
As those of you who attended the meetings or watched the talks on-line (see link below) may know, all of us in the NIH Proteus group are very excited about new avenues of research that have opened to us since we identified the genetic cause of Proteus syndrome. We are very hopeful that this discovery will improve our understanding of why patients with Proteus syndrome have overgrowth and that this knowledge may lead to better treatments for the condition.
Dr. Biesecker and Julie Sapp recently met with a drug company to start talking about a possible drug treatment for Proteus syndrome. Because of confidentiality agreements, we cannot share lots of concrete details at this time, but we left the meeting quite encouraged about the potential to start a trial of a new drug in patients with Proteus syndrome and we are optimistic that this trial could have promising results.
One thing that we talked about at the PSF meetings was how the research here at NIH could evolve as we orient ourselves around the goal of trying new treatments for Proteus syndrome. There are still many experiments we need to do and many procedures we need to put in place before we can start seeing patients as part of a drug trial, but we are working very hard on these problems and hope that we can provide more information soon. In the meantime, it is a great idea to be sure that Kim and/or Tracey have up-‐to-‐date contact information for your family so that they can keep in touch with you about our progress. As well, an important goal for the foundations is to help identify as many patients as possible who might be eligible for the next phase of the research. It is often said that there is emotional power in numbers, but in this case it is emotional and scientific power that we need to generate!
Thank you again to the PSF Board and Membership for a great set of meetings and for all you do to partner with us in our research!