Dr. Urano's Blog

Goodbye and Hello New Year

Ready to Say Goodbye

Thank you for reading my blog. I always feel your support. I appreciate it immensely. As I am ready to say goodbye to 2017, I would like to update you about our progress.

Our drug-repurposing clinical trial of dantrolene sodium is ongoing. The trial began in January, and nineteen patients with Wolfram syndrome from the US and Canada are now involved. Participation requires periodic testing and monitoring at the adult and pediatric clinics at Washington University Medical Center in St. Louis. The trial is focused on the safety, tolerability, and efficacy of the drug (visual acuity, neurological functions and remaining beta cell functions). At this stage my team has found few side effects and has identified safe dosage levels. More tests and data are necessary before any conclusions can be reached. I plan to announce our next step early next year.

I am aware that we need a breakthrough therapy for Wolfram syndrome. In theory, drugs that target endoplasmic reticulum (a cellular compartment damaged in Wolfram patients), such as dantrolene sodium, can delay the progression and may improve functions of remaining beta cells and brain cells, but these drugs cannot reverse symptoms. We need something that can reverse symptoms, such as visual impairment, and we need to tap into new technologies. We are developing a regenerative gene therapy, especially for visual impairment. Our strategy is to introduce a regeneration factor into a type of retinal cells using a gene transfer technology. This is clearly not simple and requires a lot of time and efforts, but we are making progress.

As we rise into 2018, I feel that something new and wonderful will happen to us next year. I feel that we will go into a new stage. Thank you again for your continued support. Happy New Year to everyone.

Sincerely,

Fumi Urano