John T. Milliken Department of Internal Medicine
Dear Friends, Thank you so much for “coming to see me” today. It is always my pleasure and privilege to see you and talk to you. I feel your support and encouragement, and I am grateful for everything you have done for me, patients, patient organizations, the Snow Foundation, the Ellie White Foundation, Unravel Wolfram […]
I have three goals always on my mind: Raise Awareness, Improve Clinical Care, and Provide Novel Treatments for patients with Wolfram syndrome. As the Rare Disease Day is approaching, I would like to invite you to think about more about rare diseases, including Wolfram syndrome. There are 7,000 rare diseases and disorders that when combined […]
I would like to begin by thanking all of you for your continued support and encouragement. I appreciate it immensely. I am so grateful to have such supportive colleagues all over the world. Our drug-repurposing clinical trial of dantrolene sodium is ongoing. The trial began in January 2017. Twenty-two patients with Wolfram syndrome enrolled. Two […]
I thought a lot about our therapeutic development for Wolfram syndrome in the past several weeks. I feel that drug-repurposing may not be good enough. I firmly believe that we need a breakthrough therapy for Wolfram syndrome. This year, I would like to spend more time for developing regenerative gene therapy, especially for visual impairment. […]
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 Senator Roy Blunt (Missouri) and Director of National Center for Advancing Translational Sciences/NIH Dr. Christopher Austin visited us from Washington DC on August 9th. I had a chance to present our Wolfram syndrome clinical and research program to their teams. My colleague in Internal Medicine, Dr. Dan Ory, and I tried to tell them […]
Another successful Wolfram research clinic was completed this week. I would like to thank all the participants, Dr. Hershey, Dr. Marshall, Mrs. Samantha Ranck, and other physicians and scientists who were involved in the clinic. See you next year! Thank you again. Warmest regards,Fumi Urano
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 […]
I have received many emails and phone calls regarding the ongoing clinical trials for Wolfram syndrome in the US, UK, and Europe. After the discussion with Dr. Barrett’s team in the UK and other regulatory experts, we decided to summarize the gist of the ongoing clinical trials. You can find the information on the following […]
Dear Friends, I would like to update you about the status of our clinical trial of dantrolene sodium in patients with Wolfram syndrome. Dantrolene is a US Food and Drug Administration (FDA)-approved drug currently used for different medical conditions. Based on our data, we received orphan drug designation of dantrolene sodium for the treatment of […]
Our current focus is to bring our potential drugs to our patients. We are interested in drugs that can help patients “regain homeostasis.” In other words, we try to help our patients’ organ systems to keep cellular endoplasmic reticulum states stable. The challenge is that there are not many drugs that can modulate endoplasmic reticulum […]
We have become one of the first 8 members of the NIH’s Rare Disease Data Repository Program. This is one of the important projects at the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) in the USA. I have also become a steering committee member of this program. The purpose of this […]
I tried to make progress in the following three areas in 2016: 1) Improve Clinical Care, 2) Raise Awareness, and 3) Develop Novel Treatments for Wolfram syndrome. I did my best. I know I could not make a breakthrough, but I will keep on trying. My highest priority right now is to work out the […]
I attended the Wolfram UK and WellChild conference in England on Saturday, October 8th, 2016. This was my third time at the conference and met with around 100 patients and their family members. At the conference, Dr. Timothy Barrett (University of Birmingham) announced that his phase 2 randomized clinical trial of his repurposed drug in patients […]
I would like to accomplish the following three goals: #1 Improve Clinical Care, #2 Raise Awareness, and #3 Provide a Cure for Wolfram syndrome. I feel that we have been making steady progress in #1 and #2. #3 is the most challenging one. To achieve #3, we need to first delay the progression of the […]
I had a chance to present my data and concept on regenerative medicine for the treatment of Wolfram syndrome last week at the regenerative medicine symposium in Tokyo, Japan. Five faculty members from Washington University attended this important symposium. I got constructive feedback from the audience. To achieve a cure, we need to develop regenerative […]
Our annual Wolfram syndrome research clinic took place this week. I would like to thank all the participants, staff members, scientists and doctors who were involved in the clinic. Two clinical trials are planned in the US and UK. I will keep you updated about these. I always appreciate your continued support. Take care,Fumi Urano
I attended the 6th Wolfram syndrome workshop in Nantes, France last week. I still remember the first workshop which was held in 2009. Since then, we have made significant progress in the past 7 years. Two potential clinical trial plans in the US and UK were discussed. I presented my ideas and plans about a […]
I have been in Washington DC this week to visit the National Center for Advancing Translational Sciences (NCATS) of National Institutes of Health and attend the World Orphan Drug Congress. My trip has been quite fruitful so far. I had a productive discussion with the drug development team at NCATS. We do have new drug […]
I received many emails regarding the status of the drug development. I am sorry that I have not updated you through this site. I am making steady progress. Our current target is “endoplasmic reticulum (ER) calcium homeostasis.” Based on our preclinical studies, we need to maintain high ER calcium levels to delay the progression of […]
Today is a Rare Disease Day. I am thinking of our patients and families suffering from life-threatening diseases in the world. I am very sorry that I have been slow. I will keep on doing my best to develop and bring novel treatments to our patients. I always appreciate your continued supports. I understand that […]
I just came back from my trip to Denver. I attended the fundraising event hosted by the Ellie White Foundation in Boulder on the 17th. I was impressed and moved by Ellie and her mother’s speeches. I could connect with their friends, families, supporters, and other patients. I would like to thank the White family, […]
The rare disease day is approaching. I would like express my deep appreciation and gratitude to multiple patient organizations supporting my research. I will keep on working with them to improve clinical care, raise awareness, and develop novel treatment for Wolfram syndrome. Snow Foundation (USA) Ellie White Foundation for Rare Genetic Disorders (USA) Worldwide Society […]
Last week, I gave a lecture on Wolfram syndrome at the University of Southern California Medical Center. I received constructive comments and feedback. I gave more than 100 lectures on Wolfram syndrome in the past 10 years. I will keep on doing my best to raise awareness of Wolfram syndrome. I was recently asked to […]
I would like to begin by thanking all the people around the world who keep on supporting me. I think of you all the time. We climb the stairs together hand in hand to bring novel treatments to our patients. Thank you again. I will not give up. I will keep on doing my best. […]
As we approach the end of this year, I would like to thank you again for your continued support, encouragement, and patience. The number of emails and phone calls I receive has been increasing. Three things are always on my mind.1. Raise Awareness of Wolfram Syndrome2. Improve the Clinical Care3. Provide Novel Treatments and A […]
The Snow Foundation has created the Wolfram Syndrome Clinical Trials Fund. I just looked at videos of our patients and was quite moved. I appreciate their continued support. I cannot thank you enough. We are racing against time.https://snowfunds.org/web/snow/pub I hope you have a wonderful weekend. Take care, Fumi Urano
I am pleased to announce that we have launched the NeuroRestorative Therapy team in the Hope Center, Washington University Medical Center. We are a group of neurosurgeons, neurologists, ophthalmologists, bioengineers, and geneticists like myself. The mission of our team is to restore neurological functions in patients suffering from diseases that impact the human nervous system through […]
This year’s Wolfram Syndrome Annual Conference in UK was successful again. I could connect with patients and doctors in UK again. I learned a lot from them. I would like to thank Tracy Lynch, Paul Lynch, Alan Nye, Lottie Nye, Dr. Timothy Barrett, Dr. Patrick Yu Wai Man, Dr. Karen Morrison, and all the patients […]
My friend and colleague, Mrs. Tracy Lynch, posted the following comments on her FB site. Tracy is the Chief Executive of the Wolfram UK, the largest Wolfram syndrome patient organization in the UK. Her daughter has Wolfram. I completely agree with you, Tracy. Kids with special needs are not weird or odd. They only want what everyone […]
The Snow Foundation has been invited to St. Louis Rams vs San Francisco 49 ers game on Sunday November 1st. One of our patients, Raquel, served as an honorary captain of St. Louis Rams. She was in charge of the coin toss and did a wonderful job. I was proud of her accomplishment. I would […]
The Snow Foundation hosted a fundraising event for Wolfram syndrome yesterday in St. Louis. So many people came to the event and supported our quest for a cure for Wolfram syndrome. I would like to thank, Mrs. Stephanie Snow Gebel, Mr. Barclay Gebel, Dr. Saad Naseer, and others who organized this event. I cannot thank you […]
I had an important meeting today. I did my best. I will find out the outcome soon. Thank you for your support, everyone. Warmly,Fumi Urano
This year’s Wolfram Syndrome Annual Conference in UK was successful again. I could connect with patients and doctors in UK again. I learned a lot from them. I would like to thank Tracy Lynch, Paul Lynch, Alan Nye, Lottie Nye, Dr. Timothy Barrett, Dr. Patrick Yu Wai Man, Dr. Karen Morrison, and all the patients […]
Are we making progress? Yes, we are. We have discovered FDA-approved drugs that can potentially delay the progression of Wolfram. We are actively conducting a trial using one of these drugs in one of our mouse models of Wolfram syndrome. The results look promising, but I have been trying to be cautious. Our collaboration with […]
Is headache related to Wolfram syndrome? This is a difficult question because most of us experience headache even though we don’t have Wolfram syndrome or other medical conditions. As far as I know, headache may be related to autonomic dysfunction or neuropathy in Wolfram. Some patients experience sharp and stabbing unilateral pain similar to trigeminal […]
As I mentioned yesterday, the Wolfram Syndrome Annual Conference in the UK was quite successful. The format was well-thought-out. In the morning, doctors gave lectures. In the afternoon, each doctor had an individual room for consultations and answering questions from patients/families. I learned a lot from patients in the UK and appreciated that they shared […]
Recurrent urinary tract infection (UTI) is one of the most common clinical challenges. The bladder dysfunction caused by the central and peripheral neurologic dysfunction is thought to be the underlying cause of UTI. Urine culture is recommended for Wolfram patients with fever or other symptoms, such as headache. Inflammatory conditions associated with UTI may cause […]
I gave a lecture on Wolfram syndrome at the Berrie Diabetes Center, Columbia University Medical Center, in NYC last week. I could connect with patients, doctors, nurses, clinical coordinators, and researchers in New York City. I was impressed by their enthusiasm for developing treatments for Wolfram syndrome. I could establish important collaborations there. Please stay […]
Our Regenerative Medicine 101 event on Thursday was successful. We could raise awareness of regenerative medicine. I would like to thank Dena Ladd at Missouri Cures and Lila Solnica-Krezel and Angela Bowman at Center of Regenerative Medicine at Washington University Medical Center for organizing this important event. Here is my presentation. Please feel free to […]
Three things are always on my mind: 1) Improve Clinical Care, 2) Raise Awareness, and 3) Provide a Cure for Wolfram syndrome. Our current focus is to “repurpose” one of FDA-approved drugs for the treatment of patients with Wolfram syndrome. We have been making steady progress on this, and things are looking good at the […]
As I mentioned repeatedly in my previous blogs, my highest priority right now is to start interventional studies using currently approved drugs. This strategy is called “drug repurposing.” This is an attractive therapeutic option because the development of novel therapeutic compounds takes a lot of time and money, which can be avoided by the use of readily […]
We had a wonderful night at Busch Stadium raising awareness for Wolfram syndrome together with the on Monday, August 17th. Raquel Gebel, our patient with Wolfram syndrome, and I got to throw out the 1st Pitch before the Cardinals took the field. Raquel did a wonderful job. I would like to thank all the people who […]
I have three goals in my mind all the times: 1) Improve Clinical Care, 2) Raise Awareness, 3) Develop Novel Treatments and Provide a Cure for Wolfram syndrome. I believe that our novel treatments for Wolfram syndrome can lead to a cure for other forms of diabetes, including Type 1 diabetes. I have been invited […]
As I mentioned before, our Wolfram syndrome International Registry and Clinical Study has been selected to participate in the NIH Global Rare Disease Registry Program. Fortunately, I have been appointed to serve as a steering committee member for this important initiative. We would like to accomplish the following goals through this program. For patients and […]
My current focus is to delay the progression of Wolfram syndrome using small compounds (i.e., pills), especially repurposing currently available FDA-approved drugs. I am aware that this is not good enough because many of our patients have lost tissues producing insulin, maintaining brain functions, and preserving visual acuity. In collaboration with biomedical engineers and biotechs, […]
Here is a question that I often get. “Should we get our child’s genome (i.e., the entire DNA information) sequenced?” This is a challenging question because my answer to this question may change based on the development of our technology. As of today, my answer is “No.” The contrast between our ability to identify genetic […]
Today’s was the last day for the Wolfram research clinic 2015. I would like to thank all the participants, their families, physicians, researchers, and staff members who have been involved in the clinic. I cannot thank you enough. Our next step is to start an interventional study. I will keep on working. We are racing […]
We had another fundraising event at Manna in Wilmington on July 8th. This was hosted by the Ellie White Foundation. I was gratified to know that the event was successful. My research for the therapeutic development for Wolfram syndrome has been supported by multiple patient organizations. I would like to thank everyone who attended the […]
Our trip to San Francisco this week was quite successful. I flew into San Francisco with Mrs. Stephanie Snow Gebel and Dr. Saad Naseer on Tuesday and had multiple meetings with biotech executives and others. Our fundraising events were quite successful. JT Snow, a renowned former SF Giants player and Stephanie’s brother, helped us a […]