Snow Foundation January 2017 Newsletter

The Snow Foundation is Making a Difference!

Making a Difference

Many people believe they don’t have what it takes to make a difference. The Snow Foundation (TSF) has never believed that! We have always believed in hope, and where there is hope there is life. TSF would like to thank two dedicated “change-makers” whose fundraising efforts have helped Dr. Urano speed up the start of the clinical trial.

The Ellie White Foundation http://www.elliewhitefoundation.org/
has been working hard the last five years to fund Dr. Urano’s work. Ellie is a courageous young lady who is one of the biggest Wolfram Warriors out there, giving a voice to this disease and trying to make a difference. TSF would also like to thank Mark and Sonita Silberman, the parents of another Wolfram Warrior, Maya. Their commitment to this disease and efforts the last two years to raise the funding to make this clinical trial a reality have been unprecedented. The Snow Foundation and Dr. Urano could not have done this without the White and Silberman families. Their support will make a lasting impact on the lives of those who suffer from this disease.

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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.

I 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

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Clinical Trial to Begin for Wolfram Patients, Birmingham, UK

On December 14th, the UK Medical Research Council awarded £2million ($2.5Million) funding to the Birmingham UK team to lead a clinical trial of sodium valproate in children and adults with Wolfram Syndrome.
The support of The Snow Foundation
was crucial to the success of Dr.

Barrett and his research team being awarded the MRC grant.

Read more about the trial…

Reflections-
Stephanie Snow Gebel

With 2016 behind us and the promise of 2017 ahead, I sit in my front room looking at the beautiful fire, wondering where the time has gone.

My childhood was wonderful. I had two parents who loved me to the moon and back. My dad was one of a kind — handsome, charismatic and so proud of his three children. My mom was beautiful inside and out. My parents were always there for me. They were my cheerleaders when I needed a pick-me-up. They were my lighthouse when I was lost at sea.

I lost my mom to cancer five months before I married Barclay. She was just 54 years old. Talk about devastating! Imagine walking down the aisle on your wedding day, knowing your mom isn’t at the end of it. Then, seven years later, I lost my dad to a staph infection. He had just turned 62.

Read more…