Dr. Urano's Blog

Clinical Trial Update: Next Phase

I have received many questions regarding our ongoing clinical trials, as well as questions related to our next step lately. We started the patient registry, and then moved onto the research clinic. Now, we are conducting trials. I think we are making progress, and need to speed up as we are racing against time.

Although we don’t have a concrete plan yet, we consider the following possibilities.

  1. A longer duration (2-3 years), More participants (> 200), Multi-center, Including a placebo arm (dummy drug)
  2. Compare the efficacy of dantrolene sodium, valproic acid, and dantrolene sodium plus valproic acid

We are also developing breakthrough treatments for Wolfram syndrome as dantrolene and valporic acids are old drugs originally developed for other medical conditions.

Here are our new plans.

  1. We have been developing a new drug designed for Wolfram syndrome  (ER stress stabilizers) together with NIH/NCATS to delay/halt the progression of the disease. Pre-clinical studies in mouse models of Wolfram have been designed. We are seeking funds to complete these studies.
  2. Regenerative therapies using a novel neurotrophic factor in combination with gene transfer technology for visual impairment have been designed. We are seeking funds for testing these new therapies in mouse models of Wolfram syndrome.

Thank you for your continued support. I hope you will have a wonderful Memorial Day weekend.

Take care,

Fumi Urano