Dr. Urano’s August update

Fumihiko “Fumi” Urano, MD

Dr. Fumihiko “Fumi” Urano, MD

Dear Friends,

I hope you had a wonderful summer season. I would like to update you about our progress.

Our clinical trial of dantrolene sodium in patients with Wolfram syndrome is ongoing. We have been monitoring the safety, tolerability, and efficacy of the drug. Some patients have been taking dantrolene sodium for more than 6 months, and we have started getting the safety, tolerability, and efficacy data. We plan to publish the data once we collect the information from 20 patients. I have started preparing for the next phase of this trial. As I mentioned in my previous blog, we are considering the following possibilities.

  1. A longer duration, More participants, Multi-center
  2. Include the placebo arm. I understand that nobody wants to take the dummy drug for a long period of time. So I have been getting advice from medical officers conducting clinical trials for rare diseases.

In addition to dantrolene sodium, my team has been looking into two new drugs for Wolfram. Unlike dantrolene sodium, these drugs are new. So we need to carefully collect more data from mouse models of Wolfram and healthy human subjects.

  1. The first drug is an endoplasmic reticulum (ER) calcium stabilizers which we discovered together with NIH/NCATS. This drug is for delaying/halting the progression of the disease. Pre-clinical studies in mouse models of Wolfram are ongoing.
  2. The second drug is a chemical chaperone which reduces ER stress. We have started collaboration with a biotech company on this new drug.

I am aware that we need to find a way to improve visual acuity. I am trying to secure funds for testing our new regenerative gene therapies for optic nerve degeneration using a novel neurotrophic factor in combination with gene transfer technology. I have applied for multiple grants and am quite hopeful about the outcome.

Thank you for your continued support. I cannot thank you enough.

Take care,

Fumi Urano, MD

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Eye Problems in Wolfram Syndrome

Professor Patrick Yu-Wai-ManProfessor Patrick Yu-Wai-Man

Affiliations

  1. John van Geest Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge
  2. MRC Mitochondrial Biology Unit, University of Cambridge
  3. Cambridge Eye Unit, Addenbrooke’s Hospital, Cambridge University Hospitals
  4. Moorfields Eye Hospital and UCL Institute of Ophthalmology, London

I am an eye doctor (ophthalmologist) with a particular interest in genetic eye diseases. I look after patients with Wolfram syndrome in my specialist clinic and I also lead a research group that is investigating ways of slowing or preventing loss of vision in patients affected with this relatively rare genetic disorder.

How is visual information sent from the eye to the brain?

visual info to brain

The eye is a very sensitive camera that converts an image from the outside world into an electrical signal. At the back of the eye is the optic nerve, which is similar to a high-speed “broadband cable” that allows this electrical signal to be sent quickly to the vision centres at the back of the brain to be decoded. The figure above illustrates how visual information gets from the eye to the brain via the connecting optic nerve.

What is optic atrophy?

The majority of patients with Wolfram syndrome will develop optic atrophy. Optic atrophy means that the optic nerve has been damaged and it looks pale in colour when the eye doctor looks at the back of the eye with the appropriate equipment. Because the optic nerve is damaged, less visual information is sent from the eye to the brain, and this also happens more slowly with transmission errors. As a result, patients with Wolfram syndrome start to struggle with their central vision and they find it increasingly difficult to read small print and make out people’s faces (as in the example shown below). Visual difficulties usually start in childhood and they tend to get progressively worse with time.

optic atrophy

What other eye problems can you get in Wolfram syndrome?

  1. Diabetes is very common in Wolfram syndrome, but fortunately diabetic eye complications tend to be rare. Nevertheless, patients with Wolfram syndrome need to take particular care that their blood sugar levels are well controlled to avoid further diabetic eye complications in addition to optic atrophy.
  2. A small group of patients with Wolfram syndrome can develop cataracts at a young age. If the eye doctor spots that a cataract is present and vision is getting worse because of it, the option of cataract surgery can be discussed.

What treatments can we offer at the moment?

  1. Unfortunately, there is currently no proven treatment to stop the damage to the optic nerve and loss of vision. There is a lot of research being carried out at the moment to look for drugs that can protect the optic nerve. Gene therapy is also being considered, but this strategy is still an early stage of development and so far, studies have only been carried out in mice.
  2. As there are no effective treatments yet for the optic atrophy in Wolfram syndrome, visual rehabilitation is very important and children, especially, must be provided with the right level of support at school.

How frequently should an eye check-up be carried out?

All patients with Wolfram syndrome should ideally have an annual check-up. Drops will usually be put in the eyes to dilate the pupils and make it easier to have a careful look at the back of the eye for any changes since the patient’s last visit.

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Washington University Wolfram Research Clinic completes its 8th consecutive year!

Washington-University-School-of-MedicineWashington-University-Wolfram-Study-group

Dear Wolfram Families and Supporters:

The Washington University Wolfram Research Clinic (or ‘natural history) study just finished its 8th consecutive year of assessing patients with Wolfram Syndrome. This work began in 2010, supported in part by the Snow Foundation. For the past 5 years, it has been supported by a National Institutes of Health grant in Dr. Hershey’s lab (Tracking Neurodegeneration in Wolfram Syndrome; Hershey, Principal Investigator). The focus of this grant is on understanding the neurological changes that may occur over time in Wolfram Syndrome, including the function of the brain (e.g. vision, balance) and the structure of the brain (e.g. the volume or integrity of different regions of the brain).

This year, we focused on assessing new patients, and those who had only been seen once or twice before. We hosted 9 families from all over the country and performed 106 exams and 8 MRIs. This clinic was smaller than previous years due to limitations in funding and the ongoing Dantrolene safety study. However, this focus allowed us to increase our understanding of how symptoms change over a 2 year time period, which are critical data for planning and implementing clinical trials. Altogether, over the past 8 years, we have assessed 40 unique individuals with Wolfram Syndrome and their families, some up to 7 times.

Just weeks before the clinic, we got the good news that our request to extend this work for another 5 years was reviewed very favorably! We received a score which should ensure that we get the funding we need, but will only know for sure this fall. In anticipation, we are going to start planning for next summer’s research clinic soon and hope to open it to anyone who has been seen in the past or any new patients that come to our attention. We will work together with Dr. Urano on any ongoing drug safety or efficacy studies to make sure that families do not have to choose between studies and that each study can support the other’s goals. 

As always, we will keep you up to date with our WU Wolfram Research Clinic newsletters, the Snow Foundation newsletters, our website (http://hersheylab.wustl.edu) and emails or calls. Please contact any of us at any time if we can provide more information or assistance!  We would love to hear from you!

Thank you!

Tamara Hershey, Ph. D.

Professor & Principal Investigator,
WU Wolfram Research Clinic

314 362-5593

tammy@wustl.edu

Bess Marshall, MD

Pediatric Endocrinologist & Research Clinic Medical Director

314 454-6051
Marshall@kids.wustl.edu

Samantha Ranck, MSW

Research Clinic Coordinator

314 362-6514
rancks@npg.wustl.edu

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Dr. Urano’s June Wolfram Research Update

Fumihiko “Fumi” Urano, MDDear Friends,

It’s a spectacular Saturday morning in Saint Louis as I write this to you. I always appreciate your continued support, encouragement, and kind words.

I have received many questions regarding our ongoing clinical trial, as well as questions related to our next step lately.

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

  1. A longer duration, More participants, Multi-center
  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 acid 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.

I think we are making progress, and need to speed up as we are racing against time. Thank you again for your support.

Take care,

Fumi Urano, MD

Dr. Barrett meets with Wolfram Associations across the globe

Dear Friends and colleagues,

I have been fortunate to visit the French Wolfram Association meeting in April in Paris; and the Spanish Wolfram association meeting in May in Almeria.

I was humbled by the friendship and community spirit of these families; and they were very tolerant of my poor French and Spanish language skills.

On the research front, we have just published the online mutation database of genetic variants in the Wolfram gene. This was work undertaken by Dr Dewi Astuti, and is a freely available database to support scientists worldwide. The database is available at: https://lovd.euro-wabb.org

Regarding the Phase II clinical trial, we are negotiating manufacture of the investigational medicinal product and placebo. We hope to complete this in the next 4 weeks and are still aiming to begin recruitment in the UK in November.

Thank you for all your interest and support, and I will try to provide regular updates on our progress

Sincerely,

Prof Tim Barrett

My Fight Continues – Pat Gibilisco – Pt. 3

Pat GibiliscoPart 3: My Fight Continues – Pat Gibilisco

Money was the only reason my child was not able to receive the drug that would potentially save her life.  The Snow Foundation, or I should say Stephanie Gebel, worked so hard to raise money for all children affected with WS.  She had to beg people, year after year, to continue to support the WS Foundation.  I can tell you that was not an easy task! She had some support from the other families with WS, but not enough.  Some families felt it was just too hard to ask for contributions. For 18 years, I have done everything I could to fight for all WS children.  I repeatedly asked my family and friends to help me save my daughter.  They were so generous in their efforts to help me find a cure for WS. Now I have to say that as much as I so appreciated their love, support and financial assistance, it was just too late.  The father from Utah also lost his five children afflicted with WS.  

Why does money always stands in the way of a cure?  Could we have had more help from our WS group?  Absolutely.  I can tell you that the Snow Foundation has fought for ALL the WS children.  Stephanie Gebel has fought to save her child’s life and every other family’s child.  Dr. Fumi, Dr. Naseer, Dr. Hershey, Dr. Hoekel, Dr. Marshall, Dr. White, Dr. Paciorkowski, Samantha, Beth, Cris, and all the other medical professionals and staff have fought hard to save our children.

Am I mad?  Yes.  Am I bitter? No.  Would I have done anything differently? No. It all comes down to money.  Now it is too late for a drug to save my daughter.  We started this fight 18 years ago, and it was ending for my daughter. No more clinical trials and no more clinics. My daughter is now totally blind and almost deaf, and she has bowel and kidney problems, seizures, choking episodes, heat intolerance, poor balance, Diabetes, Diabetes Insipidus, short-term memory loss, and many more issues that she deals with daily.  All the while though, she has such a smile on her face and soul. Lauren and all the other past and present WS children are the real heroes.  They are amazing kids.

For a fourth time, I will cry.  It will be at her funeral.  It will happen. Nothing can stop the clock from ticking.  I can tell you I will NEVER quit until a cure is found. I will continue the fight so no other family has to go through the heartache I have.  Please, I beg you, continue to fight the fight.  Do not let money come between your child and a cure.  We must save all the WS children.  I don’t want another family to watch their child slowly die from the inside out.  It is brutal to watch.

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The Journey Continues – Pat Gibilisco – Pt. 2

Pat GibiliscoPart 2: The Journey Continues

Dr. Permutt and Jon Wasson, who helped discover the WS gene and named it WSF1, were leading the first-ever  WS clinic in St. Louis.  Dr. Hershey and many other fabulous doctors from Washington University in St. Louis and other areas of the United States, started the first database of WS families and symptoms. Dr. Permutt talked with us about his mission to find a cure for WS, and he was so overwhelmed with emotion that we were willing to help him with his dream, even though it was a financial burden on us.  For so many years, he studied only mice afflicted with WS.  Now he had actual patients with this terrible disease, and our united efforts to help him find a cure.  

We heard there wasn’t going to be a second clinic because Dr. Permutt’s grant did not come through for a second year.  Again, our hopes and dreams were crushed.  But soon we learned there was a new family diagnosed with WS right in St. Louis.  We discovered we had a mama bear ready to take on the mission.  Stephanie Snow came at us full speed.  She quickly set up fundraisers to get the money we needed, and through her hard work, the Snow Foundation was able to fund the second WS clinic.  

It was during the second clinic that we discovered Dr. Permutt had cancer.  He had said nothing to us so we were very surprised.  After the clinic, we learned he had died.  I felt all our hopes and dreams also died.  We had worked so hard and for so long to have the possibility of a cure within our reach, and I was completely devastated.  For only the second time since my daughter’s diagnosis, I broke down and cried.  

We thought we were back at ground zero, but we soon learned that Dr. Hershey had come up with a three-year grant.  With that grant and funding from the Snow Foundation, we were able to hold four more research clinics.  We also learned that Dr. Fumi Urano, who attended our first clinic, would take over Dr. Permutt’s role and research.  We were again headed in the right direction and had hope.  But the year after Dr. Permutt died, the clinic was a little somber.  One day Jon Wasson asked to meet with us.  He told us this would be his last clinic; he had cancer and was going to die. This was almost too much to bear. Again, we mourned the loss of another WS pioneer.

At the sixth research clinic, we learned that Dr. Fumi found a drug he believed would at least stop the progression of symptoms.  This was exciting news!  The only problem was that it had taken seven years to reach this point, and my daughter’s condition had worsened over that time.  They were ready to start the first human safety trials on the drug that could possibly stop the symptoms from progressing. The drug would target the optic nerve loss, the beta cells of the pancreas, and balance.  My daughter was already blind, and she had no beta cells left in her pancreas; the balance wasn’t enough to get her into the clinic. I told them that if the drug couldn’t help my daughter, we would step aside so it could help someone else.  What they didn’t know was that on the nine-hour drive home, I cried most of the way.  This was everything I had hoped for, but it was too late.  It was only the third time I cried since my daughter’s diagnosis.  I had failed her.

Continue to part 3>

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My Wolfram Syndrome Journey – Pat Gibilisco – Pt. 1

Pat GibiliscoPart I: The Beginning

My journey started 28 years ago. My daughter was two years old when she was diagnosed with Type 1 Diabetes. We were able to manage it and still live a great life. When she was 12, she started to have other health issues, specifically, problems with vision and extreme thirst. Our ophthalmologist suspected Wolfram Syndrome (WS). He noticed the pale optic nerve, and he suspected she also had Diabetes Insipidus. Our endocrinologist suspected Lauren had either a brain tumor or WS. Of course, not knowing what WS was, all I heard was brain tumor. Lauren underwent an MRI that determined she didn’t have a brain tumor, and we were ecstatic, until we learned what WS really was. I felt it was a death sentence.

Like any parent, I searched the Internet for information. Unfortunately, there wasn’t a lot of it, and what was available was so very scary. I was determined to find help for my daughter. I finally found Dr. Barrett from the UK. He told me he knew of only one other WS family in the United States. I was so excited to hear from another parent who understood my fears and questions. His family has eight children, five of them with WS. I rushed home every night hoping I had an email from him. Although he faced such a huge burden of knowing five of his children had WS, he was the most positive and spiritual person I had ever met. I could no longer avoid WS, and I decided we had to do something.

Fortunately he knew how to set up a website, and I had all the research material. Together, we started the first-ever WS family support website. My life continued to be consumed by WS, and I was on a mission to find a cure for my daughter. I called every doctor who had written articles on WS. I finally found Dr. Permutt, who had completed some research with WS. Dr. Permutt had started a small research project on WS, but due to lack of money, he abandoned it. I told him if he ever started one up again, please let me know. Nothing was going to stop me from searching for a cure.

I connected with three new WS mothers who were just as determined as I was. We researched and tried to contact anyone with information about WS that could help us. We were willing to be guinea pigs, if needed. We had nothing to lose. Meanwhile, Dr. Permutt told me he was going to St. Louis to start a small WS research clinic, although he had to start with mice. For the next few years, he kept me updated on his mice trials. Then one day he called to tell me he was starting the very first WS Registry. I was the first to sign up! It was headed by Jon Wasson. Dr. Permutt also scraped together a little money to start a research clinic, but he had no funding for participants. Money, however, was not going to stand in the way of a cure for my daughter. Seven other families and I agreed to come to St. Louis using our own funds to pay for gas or airfare, and hotel stay. We had families from New York, Pennsylvania, North Carolina, California, Nebraska and even Italy.

To be continued… Part 2 >

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Jack Snow – His Cause Continues

Strong of Heart, Profiles of Notre Dame Athletes

(written by Randy Covitz)

Jack Snow’s daughter Stephanie pleaded and finally persuaded him to be in the birthing room when she was about to deliver her daughter Raquel.

Tears filled Snow’s eyes when he held Raquel, his seventh grandchild, with the steady hands that caught 60 passes as an All-America wide receiver at Notre Dame in 1964 and 340 more passes and 45 touchdowns in an 11-year National Football League career with the Los Angeles Rams.

Snow would never live to see Raquel grow up, nor would he know that she would be diagnosed at age 4 with a rare disease known as Wolfram syndrome. He died on Jan. 9, 2006, of a staph infection at age 62. It was nine months and a day after Raquel was born.

But the legacy and memory of Jack Snow could be the catalyst that leads to more effective treatments, if not a cure, for millions of patients who suffer from diabetes and related illnesses, including Wolfram syndrome.

In 2011, Stephanie Snow Gebel and her brother, former Major League Baseball star J.T. Snow, established the Snow Foundation to raise awareness and funding to fight Wolfram.

Wolfram presents itself as Type I diabetes and ultimately leads to vision loss, hearing loss and cognitive decline. There are about 25,000 known cases of Wolfram in the world, and 60 percent of the patients die by their 30th birthday.

“From the time of diagnosis, they decline a great deal within five to eight years,” said Stephanie Snow Gebel, who with husband Barclay has four children, including Raquel, now 10.

“Raquel is legally blind now … she can’t see five feet in front of her. She has to go on medicine to control her bladder. She gets awful headaches and choking episodes.”

During Jack Snow’s playing career, he represented several charitable causes, including the Susan G. Komen (breast cancer), the Epilepsy Foundation and, most dear to his heart, the Juvenile Diabetes Research Foundation. His other daughter, Michelle, has a son, Jacob, who was first afflicted with diabetes as a young child.

“He was an out-of-the-norm athlete,” Stephanie said of her father, who teamed with Heisman Trophy-winning quarterback John Huarte in 1964 and left Notre Dame as the school’s all-time leader in single-season receptions, receiving yards and touchdowns. “He would go out and publicly speak and give his fee to the Rams’ charitable foundation. He was always doing things for people, and he never expected anything in return.”

Unlike the many charities Jack Snow endorsed, the Snow Foundation is one of a kind. It’s the only organization in the world dedicated to conquering the lesser-known Wolfram syndrome.

The Snow Foundation has raised close to $1.5 million for research, but it is about $2 million shy of the necessary funds to complete drug trials.

“We’re learning that lesser known diseases don’t get a lot of attention and have a hard time with fund raising,” said J.T. Snow, a former Gold Glove first baseman for the San Francisco Giants. “We’ve reached out to a lot of people. We got this thing rolling. We need to raise a lot more money to make sure people like Raquel are going to be taken care of.

“We’re kind of bummed out that my dad’s not around because he would have taken this and would have gotten a lot of attention for it. He would get people to buy in and to get into their checkbooks and help us raise money for these doctors who are studying Wolfram syndrome.”

Three-generations-Papa-Jack-Stephanie-and-tiny-Raquel

Three generations…Papa Jack, Stephanie and tiny Raquel…

As analyst for the Rams’ radio broadcasts after his professional football career ended, Jack Snow accompanied the club when it moved to St. Louis in 1995. In a stroke of good fortune, the doctor who began researching and discovered Wolfram syndrome was Dr. Alan Permutt of Washington University in St. Louis.

Permutt, a diabetes victim himself, treated Raquel for nearly two years until his death in 2012. His work is being carried on at Washington University by Dr. Fumihiko Urano, whom Stephanie convinced to relocate his family from Japan to St. Louis.

“In focusing on a rare disease like Wolfram, we have uncovered a lot about common problems—that’s what is so exciting about it,” said Dr. Saad Naseer, chief executive officer and chief medical officer of The Snow Foundation.

“It’s diabetes, vision loss, hearing loss and brain deterioration. Those four collectively affect hundreds of millions of people. You probably know 10-12 diabetics personally. You know people whose vision and hearing deteriorate or have Alzheimer’s or Parkinson’s or some sort of other brain deterioration.

“The research we’re doing is applicable to all those people. If we find a new treatment for diabetes, it’s not just for Wolfram syndrome patients, it’s for every diabetic. The same goes for any treatments we develop for vision loss, hearing loss or brain deterioration.”

The doctors at Washington University recently received encouraging data from its first testing of mice.

“We actually prevented diabetes in all of the mice we gave our test compound to,” Naseer said. “We’re close, but the problem is we need a minimum of $2 million to complete all these studies in mice.”

To help come up with more funds, the Snow Foundation established a website, thesnowfoundation.org, to raise awareness and has staged events including golf tournaments, celebrity bartending functions and other activities to raise funds.

Stephanie and J.T. also reached out to the Notre Dame family, including former Fighting Irish quarterback Rick Mirer, whom Gebel calls “her little Notre Dame angel,” and Brian Murphy, an NFL player agent and Notre Dame graduate whose wife Lauren is a diabetic.

Mirer, who operates the Mirer Family Foundation that provides help for youngsters with health and educational needs, said Raquel’s illness reminded him of former Notre Dame coach Ara Parseghian’s three grandchildren who died from a rare genetic disorder.

“I ran with this because I had a lot of sympathy for Stephanie’s situation, and I wanted to introduce her to people who may be able to help,” Mirer said. “The Notre Dame family rallied around Ara’s situation and made an impact, hopefully eliminating a lot of other kids having to go through that.

“It’s been a little harder because she’s not Ara Parseghian. Eventually, we’ll get the right people aware of it and she’ll get the help she needs.”

Mirer hopes members of Notre Dame’s Monogram Club respond to the cause— and Murphy, who represents current and former NFL players from Notre Dame, believes they’ll step up to the plate, similar to how Jack Snow contributed his time and resources to worthwhile causes.

“This is going to sound corny,” Murphy said, “but this is what they teach you during your four years under the Golden Dome, that part of our purpose here on earth is to make our community better and to help those less fortunate and to give and to give and to give.

“It’s an old philosophy that you give what you get. Jack Snow was actively involved in all these charities, and now it’s time to help his family and a lot of people are anxious to give back to him because he helped so much.”

Once the doctors at Washington University complete the mice studies, the Snow Foundation will have sufficient data to approach a pharmaceutical company and ask it to take over the project.

“Our foundation is the Little Engine That Could,” Stephanie Snow Gebel said. “Keep your eye on us. We’re getting positive hits on the diabetic rounds with the drug we’re working on and positive hits on a drug we’re working on with vision.

“It’s going to be revolutionary. I think there’s a reason my child got this disease. I am a fighter, I am a momma bear, I’m not going to sit back and watch her deteriorate if I can do something about it. I’ve got my dad’s personality. I’m a mini-Jack Snow … ”

In all, Jack Snow had three children and eight grandchildren. In this quest to find a cure for Wolfram, the family still derives inspiration from Jack nearly 10 years after his death.

“He’s watching, and he’s helping out best he can,” said J.T. Snow. “We know that because little things here and there pop up and let us know that he’s paying attention, both he and my mom, Merry Carole, who we lost way too early (to cancer in 1998).

“We see the No. 84 a lot, his number with the Rams. Things that remind us of him, things a family would know. You go to the grocery store, and your grocery bill is like $84 … or a lot of times we travel, and we’re leaving out of Gate 84. Just random things like that let you know my mom and dad are watching.”

“Our foundation is the Little Engine That Could.”

Clinical Trial Update by Fumihiko Urano, M.D.

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 Wolfram syndrome from US FDA and European Medicines Agency (EMA) in 2016. We also ​received the​ green light from ​the ​FDA and Washington University Medical Center to proceed to a pilot clinical trial (phase 1b) to assess the safety and efficacy of dantrolene on patient’s vision, brain functions, and diabetes.

Thanks to the generous support from the Snow Foundation in collaboration with Ellie White Foundation and other private donors, I was able to commence the clinical trial and slowly started recruiting patients. I would like to recruit at least 24 patients and hope to secure more funds to accommodate all the patients who are eligible for this trial. Stephanie Gebel​ has told me that she will do her best to make this happen. ​I was just informed ​​this week that we will receive grant funding from the National Institutes of Health (NIH) to support this trial.

​have not forgotten​ about our patients in other countries. Dr. Tim Barrett in the UK and I are closely working ​to conduct international clinical trials for Wolfram syndrome. In parallel, we keep on developing novel drugs and regenerative therapies for Wolfram syndrome. Dantrolene could be a band-aid for Wolfram, but cannot provide a cure. Thus, we need pipelines for developing breakthrough treatments.

Thank you for your continued support.

Sincerely,

Fumi Urano, MD