October 13th, 2009

Well, it’s been a very long time since the last update and a handful of things have happened since. We continue to move toward an FDA approval for CTX treatment. The two companies who are working on this, Sigma Tau and Manchester Pharmaceuticals are in various stages of review by the FDA for their individual efforts toward accomplishing the goal. No firm dates have been set yet but I fully expect to see something solid by the end of the year with a hope that manufacturing and distribution of the newly approved medication in production within the first quarter of 2010. As we’ve learned through all this, patience is paramount and any number of factors can hold things up but the most important thing to note is that the medication in its current state will continue to be available until the new medication is fully available.

I am also participating in a steering committee for developing a Health Management Program for CTX patients. The program is being developed by Centric Health Resources. Centric Health Resources is a company that strives to deliver prescribed therapies and specialized health management services to individuals affected by orphan diseases. The basic goal of the program is to develop several components to a comprehensive treatment protocol to manage patients and their treatment regimen as well as to develop literature for physicians to distribute to patients that have references to resources available to patients and/or their caregivers. This is greatly simplifying it however. Please visit their site to learn more about them.

Lastly, Dr. Andrea Debarber who works as an Assistant Professor in th BioAnalytical Shared Resource lab in the OHSU Department of Physiology and Pharmacology continues to make amazing progress in her research of developing a newborn screening test for CTX. In order to continue her research she relies on grant funds from various organizations. One of the(if not the) biggest grant providers for disease research grant money is the National Institutes of Health(NIH). Recently the NIH announced an expansion of the Rare Diseases Clinical Research Network, which consists of a second phase which includes funding for 19 additional research consortia. OHSU is one of the research consortia included in the expansion which designates 117 Million dollars over the next 5 years to be distributed for research. Several of OHSU’s research projects will benefit from this grant money one of which is the CTX newborn screening test research. This is phenomenal news for us, and of course the researchers as this has been one of the many goals that we’ve had as part of our bringing increased attention to CTX from a patient advocacy perspective.

The NIH press release can be seen here

That’s about all for now, more to come as additional developments occur.

Posted in Updates.