Thanks to your massive respons, we can announce you that the Bang!-event in Ghent is completely sold out. But don't worry! Ivan De Vadder and co are willing to launch their show a 2nd timein favour of the research against Wolfram syndrome. This time in 'De Roma' in Antwerp.
For more details click here. For tickets click here.
Newsletter from Prof. Urano:
Dear friends,
My highest priority right now is to work out the logistics for the upcoming clinical Wolfram-trial. As our medical center announced in November this year, we plan to start a phase 1b safety clinical trial of dantrolene sodium in patients with Wolfram syndrome.
Dantrolene sodium is an FDA-approved drug utilized for the treatment of muscle stiffness. We found that dantrolene could prevent the destruction of insulin-secreting beta cells in animal models of Wolfram syndrome and in brain cells differentiated from skin samples taken from patients with the illness in 2014 after the 12 years of research. This drug can target the molecular pathways altered in Wolfram syndrome in cell and animal models of Wolfram syndrome. Nobody has ever tested dantrolene in patients with Wolfram syndrome, so our first and most important objective is to make sure it’s safe. We will also closely monitor patients’ vision and brain function, as well as the function of their remaining insulin-secreting beta cells based on the data collected through our research clinic study.
The major question that I get from every patient I see is, “Is there any treatment?” As you are aware, there is currently no treatment that can slow, halt, or reverse the clinical manifestations of Wolfram syndrome including vision impairment and diabetes.
I sincerely hope that this study can help change that. I have been working with Dr. Tim Barrett in the UK to conduct international clinical trials for Wolfram syndrome. In parallel, we keep on developing novel drugs and regenerative therapies for Wolfram syndrome.
As we near the end of this year, I would like to take this opportunity to thank you all for your continued support. You have provided patients with hope and emotional support. Together with you, we can make a difference in the future of our patients. Thank you again for your generous support and continued encouragement. You gave me the power and courage to continue. I cannot thank you enough. I hope you have a wonderful holiday season.
Sincerely,
Fumi Urano, MD
Thanks to all of your donations, we were able to support the Wolfram syndrome project of professor Urano at Washington University in St. Louis. He is the personification of our hope to find a cure on Wolfram syndrome.
Please realise that your donations matter and are very critical in our fight for a bright future for Bertha and Victor and all other Wolfram patients.
Eye Hope Foundation is proud to announce that we have decided to join forces with Stephan Goovaerts and Sophie Recour in our efforts to raise awairness and find a cure for Wolfram Syndrome. The daughter of Stephan and Sophie, Bertha (10), has recently been diagnosed with Wolfram Syndrome and just like us they would like to turn this very painful news into something positive and fight for a cure. They will become a part of the steering commitee of Eye Hope Foundation and will actively be involved in our fundraising efforts.
Please support our fight by donating in the account of the King Baudouin Foundation : IBAN: BE10 0000 0000 0404 - BIC : BPOTBEB1 - Please mention ***016/0630/00037***. All donations of 40€ and more, made in Belgium, are tax deductible. Donations from abroad, are equaly tax deductible when made through 'transnational giving'.
The drug dantrolene is a muscle relaxant approved to treat patients with cerebral palsy, multiple sclerosis and muscle spasticity. Recent research also suggests it can prevent the destruction of insulin-secreting beta cells in animal models of Wolfram syndrome. Researchers at Washington University School of Medicine in St. Louis are beginning a clinical trial to assess the drug as a treatment for patients with Wolfram syndrome.
Washington University of medicine
On saturday the 15th of October, Leen Vincke and Sylvie-Anne Ombregt organised a party to raise money for the research on Wolfram Syndrome. All the people present saw that is was a great success!
Today we can announce you that it was a success, indeed. Leen and Sylvie-Anne gathered a sum of 9.104,00 € !!
We are very grateful to all the people who made this event possible.
Special thanks to Leen and Sylvie-Anne!
Many thanks to the Belgian Driving Association for their financial and fraternizing support in our battle against Wolfram Syndrome!
On the 28th of May 2016 Rotary Damme organised a Golf Charity Cup. We received a nice cheque to the amount of 5000,00 € to support the research on Wolfram Syndrome.
Many thanks to Rotary Club Damme.
The 7th Annual Washington University Wolfram Syndrome Research Clinic Meeting was held in July, bringing together the key opinion leaders and researchers who are leading the fight to bring a cure to Wolfram Syndrome. Here are some exciting highlights of recent findings from the meeting.
Collaboration and Funding is Critical
The effort to understand disease progression of neurological symptoms of Wolfram Syndrome continues, and significant progress has been made in the last year. Researchers now know the appropriate measurements to use in a clinical trial. Several endpoints have been identified, however, visual acuity and the volume of the ventral pons (a structure in the brain) have been shown to change reliably over time and will be used in upcoming trials to determine the efficacy of treatments for Wolfram syndrome. Adding to the challenge of conducting a clinical trial, researchers have also discovered that there is great variability in Wolfram syndrome symptoms and progression. There are also new hypotheses on how Wolfram affects the brain that will be soon tested in models, which may lead to more targeted treatments for the neurological systems.
Progress on these efforts, however, is heavily dependent on funding, with the current NIH grants ending in 2017. Our goal coming out of the 6th International Workshop was to bring researchers together to collaborate, move science forward to treatments and cures. At the 6th International Workshop we had 37 participants, 11 countries, four family associations, and presented 20 talks. Since then, we’ve accomplished the goals of finding a potentially useful animal model, devising new diagnostic guidelines, and planning two clinical trials.
Defining Wolfram has been challenging. A proposed definition of Wolfram syndrome was presented at the conference, and are:
Disease-causing mutations in the WFS1 gene.
Two major criteria (Diabetes mellitus and optic atrophy) or having one major criterion and 2 of: Diabetes insipidus, hearing loss or neurologic deficits.
Most clinical trials have very specific qualifications before they begin, like certain ages, certain sex, at same stage in illness etc. Since Wolfram syndrome is a rare disease and we cannot have specific qualifications like the list above, the trial could face challenges due to the small patient population, variability in the disease progression, and variability in the severity of the symptoms.
Diet Can Be a Factor
Some recent research suggests that nutritional components of a patient’s diet may be significant to Wolfram patients. Although there has been no study for treating Wolfram syndrome in people, there have been studies and recommendations for some other conditions like heart disease. Dr. Urano presented evidence in cells that DHA and Omega-3 fatty acids may help ER stress, which is the problem in Wolfram. DHA and Omega-3 fatty acids are in food and generally believed to be healthy in moderation.
Wolfram patients can get Omega-3 fatty acids, especially DHA from fish: anchovies, bluefish, herring, mackerel, salmon, sardines, sturgeon, lake trout, and tuna. (Fish like mackerel, wild swordfish, tilefish, shark or farm-raised fish have higher levels of mercury, PCB’s or other toxins, and should be limited). Some vegetarian-friendly sources for Omega-3 include: walnuts, pecans, pistachios, eggs, seaweed, parsley, spinach, kale, and beans. We don’t know the ideal dose for Wolfram patients, but a good range would be 500-1000 mg a day of diet plus supplemental omega-3’s.
Another paper has shown that CoQ10 has a possible improvement in vision in one person with Wolfram. The ideal dose is not known, but the recommended range from manufacturers is 20-400 mg. CoQ10 may have some side effects but is probably safe in higher doses for a limited time.
A low salt diet may be also detrimental in cases of diabetes insipidus. A reasonable amount of salt is a teaspoon or 2000 mg a day. Some foods have a lot of salt so it will count towards your 2000 mg daily-recommended limit.
