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Fumi October Update
October 13, 2019
Dear Friends,
It is wonderful to see you. I always appreciate your continued support and encouragement. I have been doing my best to develop cutting-edge treatments for Wolfram syndrome and save our patients. It has been tough, but your kind words keep me going. I feel incredibly grateful. I would like to share our progress and ideas.
As you know, our clinical trial of dantrolene sodium for adult and pediatric patients with Wolfram syndrome has been going well, but a mild success. To improve the efficacy of dugs targeting endoplasmic reticulum stress (ER stress: a molecular mechanism of Wolfram syndrome), I have been working on two new drugs targeting ER stress. Based on the results of dantrolene trial, I am confident that targeting ER stress is beneficial for patients with Wolfram syndrome. Preclinical data (i.e., data using cell and animal models) and safety profile of both new drugs look very good. Thus, I am planning a multi-center trial using one of these drugs. My goal is to make this happen in the next 12-24 months. I will do my best.
Another priority for me is to develop gene therapy for vision impairment in Wolfram syndrome. We have created adeno-associated virus (AAV: a safe virus utilized for gene therapy) that can deliver normal Wolfram gene (WFS1) and a regeneration factor, MANF, into retinal cells. We got an encouraging result last week showing that one of our new type of viruses can deliver MANF into retinal ganglion cells efficiently in a mouse model of Wolfram syndrome. My goal is to complete preclinical studies on gene therapy as quickly as possible. This is always on my mind. I will keep on doing my best and keep you updated. I am assembling a strong team of eye doctors.
As always, please feel free to contact me with any questions or concerns (urano@wustl.edu). I would like to know what you think and how you feel. Thank you again for your continued support. We will decrease human suffering and change the future of our patients together.
With gratitude,
Fumi Urano
October 13, 2019
Dear Friends,
It is wonderful to see you. I always appreciate your continued support and encouragement. I have been doing my best to develop cutting-edge treatments for Wolfram syndrome and save our patients. It has been tough, but your kind words keep me going. I feel incredibly grateful. I would like to share our progress and ideas.
As you know, our clinical trial of dantrolene sodium for adult and pediatric patients with Wolfram syndrome has been going well, but a mild success. To improve the efficacy of dugs targeting endoplasmic reticulum stress (ER stress: a molecular mechanism of Wolfram syndrome), I have been working on two new drugs targeting ER stress. Based on the results of dantrolene trial, I am confident that targeting ER stress is beneficial for patients with Wolfram syndrome. Preclinical data (i.e., data using cell and animal models) and safety profile of both new drugs look very good. Thus, I am planning a multi-center trial using one of these drugs. My goal is to make this happen in the next 12-24 months. I will do my best.
Another priority for me is to develop gene therapy for vision impairment in Wolfram syndrome. We have created adeno-associated virus (AAV: a safe virus utilized for gene therapy) that can deliver normal Wolfram gene (WFS1) and a regeneration factor, MANF, into retinal cells. We got an encouraging result last week showing that one of our new type of viruses can deliver MANF into retinal ganglion cells efficiently in a mouse model of Wolfram syndrome. My goal is to complete preclinical studies on gene therapy as quickly as possible. This is always on my mind. I will keep on doing my best and keep you updated. I am assembling a strong team of eye doctors.
As always, please feel free to contact me with any questions or concerns (urano@wustl.edu). I would like to know what you think and how you feel. Thank you again for your continued support. We will decrease human suffering and change the future of our patients together.
With gratitude,
Fumi Urano
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