September 22, 2017
It is nice to “meet” you again. Thank you for your generous and continued support for our therapeutic development for Wolfram syndrome. With the support from the Snow Foundation, multiple patient organizations and supporters around the world, and federal grants, I could maintain the Wolfram syndrome program to study the disease, which led to fundamental laboratory studies that uncovered the molecular genetic defect, and ultimately to the identification of a therapeutic target that is now being tested in patients. Our first clinical trial of a re-purposed drug, dantrolene sodium, in patients with Wolfram syndrome is ongoing. We have been monitoring the safety, tolerability, and efficacy of dantrolene sodium in 21 patients who have qualified for the study. You can find the information about the inclusion and exclusion criteria on the following website. We have both male and female participants in pediatric and adult populations. https://clinicaltrials.gov/ct2/show/NCT028292680
The planned duration of oral dantrolene sodium administration in this study is 6 months with an optional extension phase up to 24 month. All the participants are required to come to our clinic 9 times in the first 6 months to determine the appropriate dose and ensure the safety. After the first 6 months, participants come to our clinic every 6 months up to 24 months. As of today (September 22, 2017), 20 participants are taking dantrolene sodium and one participant has left the study due to personal reasons. 9 out of 20 patients have been taking dantrolene sodium for more than 6 months. In addition to safety and tolerability, we have been assessing our participants’ visual acuity, remaining beta cell functions (i.e., their ability to produce insulin from their own pancreases), and neurological functions every 6 months. We plan to publish the data once we collect the information from these 20 participants after the 6-month administration of dantrolene sodium.
On a different note, Senator Roy Blunt and the Director of National Center for Advancing Translational Sciences (NCATS), Dr. Christopher Austin (https://ncats.nih.gov/), visited our medical center last month. I had a chance to present our medical center’s efforts on rare disease therapies. I am glad to tell you that our presentations were perceived really well. Stephanie and I met with Dr. Austin a few years ago at the NCATS headquarter in Bethesda, close to Washington DC, and that was the beginning of my collaboration with the drug development team at NCATS. We will keep on working together for developing rare disease therapies.
Thank you for being with me. I plan to update you about our two new drugs and regenerative gene therapy for retinal degeneration in my next blog. I hope you will have a wonderful fall season. See you soon.
Fumi Urano, MD