Clinical Trial & Research Updates
Here you will find news updates, papers and links from the Clinical Trial Team in Birmingham, USA and other Researchers globally.
USA – Amylyx Pharmaceuticals – April 2024
Amylyx Pharmaceuticals announces interim data from ongoing Phase 2 Helios Clinical Trial, demonstrating improvements in Pancreatic function and Glycaemic control with AMX0035 in people with Wolfram Syndrome.
Italy – San Raffaele Myocarditis & Cardiomyopathies – March 2024
Our team here in the UK say, “It is known that complex and non complex cardiac disease can be seen in WS. Certainly the presentation with fever would point to this and I would be reluctant to ascribe this to WS (particularly as a single case).” They also said, “..bear in mind this is only one person out of the 00s of people with Wolfram who have been described in the literature; so more likely to be a co-incidence than a true association.”
UK – TREATWolfram Update from Prof Timothy Barrett – March 2024
USA – Research Update from Dr Fumihiko Urano – February 2024
WS Treatment Pipeline – January
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WS Treatment Pipeline – November 2023
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UK – TREATWolfram Trial Update – Prof Barrett – November/December
USA – Research Updates – Dr Urano – November/December
WS Treatment Pipeline- Sept 2023
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UK – Research Updates – Prof Barrett – August
USA – Research Updates – Dr Urano – August
Spain – The UAB to study gene therapy for Wolfram syndrome – August.
The original article can be found on the 2021 tab.
UK/Europe – What can drug repurposing mean to patients with rare diseases? – July
UK – NIHR investment will tackle barriers in rare disease research – July
UK – WFS1-Associated Optic Neuropathy: Genotype-Phenotype Correlations and Disease Progression – Prof Yu Wai Man – Updated July
Italy – Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1 – Prof. Lorenzo Piemonti – Updated July
UK – TREATWolfram Trial Update – Prof Barrett – May
USA – Research updates – Dr Urano – May
International – 8th International Symposium – April
France – Research update – Dr Cecile Delettre – March
UK – TREATWolfram Trial Update – Prof Barrett – March
USA – Research updates – Dr Urano – February
Italy – MCT1-dependent energetic failure and neuroinflammation underlie optic nerve degeneration in Wolfram syndrome mice – February
WS Treatment Pipeline – uploaded Dec 2022
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USA – Research updates – Dr Fumi Urano – Nov 2022
UK – TREATWolfram Trial Update – Nov 2022
USA – Amylyx pharma, pipeline. We plan to start a trial with them very soon. October 2022
USA – Dantrolene trial results phase Ib/IIa. We are planning a phase II/III trial next year. (Published June 2021, added Oct 2022)
USA – Wolfram iPSC-based therapeutic development – September 2022
UK – TREATWolfram Trial Update- Prof Tim Barrett – August 2022
USA Research Updates – Dr Fumi Urano – August 2022
UK NIHR-supported researcher Professor Yu Wai Man appointed Chair of Ophthalmology in Cambridge – July 2022
USA Research Updates – Dr Fumi Urano – May 2022
UK Trial Updates – Prof Barrett and Dr Wright – June 2022
UK Trial Updates – Prof Barrett – March 2022
USA Research Updates – Dr Fumi Urano – February 2022
An Insight into the participation of Rare Disease patients in research – Infographic provided by EURODIS and Rare Barometer Voices
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WS Research Pipeline – Uploaded January 2022
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USA – Trial and Research Updates – Dr Fumi Urano – 30th November 2021
UK – TREATWolfram Update – Prof Tim Barrett – 30th November 2021
UK – TREATWolfram Update (Adults) – Dr Ben Wright – 18th November 2021
Recruitment to the trial has now closed. People who have been recruited are continuing to be monitored with both face-to-face visits at the Queen Elizabeth hospital and as telephone contacts. We are immensely grateful to all the people and their carers and/or families who have been or are involved.
UK – A mutant wfs1 zebrafish model of Wolfram syndrome manifesting visual dysfunction and developmental delay – October 2021
The team at Cambridge, UK are now working on the next phase – developing this zebrafish model further to fluorescently label various retinal cell types.
USA – How a cheap antidepressant emerged as a promising Covid-19 treatment – September 2021
Some of you may have heard of research that was being done in USA, looking at the treatment of Covid with Luvox. Luvox is an antidepressant that Dr. Angela Reiersen at Wash U (works with Fumi et al) found was particularly effective for people with Wolfram Syndrome who have anxiety/depression. She made a remarkable connection between the anti-inflammatory action of Luvox and it’s possible mechanisms of action in Wolfram Syndrome, and then extrapolated that to people with Covid…
Realising that the anti-inflammatory mechanism could help people with Covid. It looks like she was right; the article below talks about this and there are several other published articles that took off after she made the connection.
Once again, this ultra rare disease is helping to solve some other really big problems.
Spain – The UAB to study gene therapy for Wolfram syndrome – August
UK – Therapeutic Advances in Rare Disease – August 2021
A recently published review from Dr Patrick Yu Wai Man’s team at Cambridge University Hospital.
USA – Trial and Research Updates from Dr Fumi Urano – August 2021
Spain Gene Therapy to get a treatment for Wolfram Syndrome – Spanish WS Association – August 2021
We are proud to announce that, together with researchers from the Autonomous University of Barcelona (UAB), we have started the first gene therapy project for Wolfram Syndrome
UK TREATWolfram Trial Update – Prof Barrett – June 2021
USA Trial and Research Updates from Dr Fumi Urano – May 2021
Acknowledgments also to the White Foundation for their help in referring patients to Dr Urano’s research. They were unfortunately omitted from the blog sent out in the newsletter.
Portugal – Wolfram syndrome: a Portuguese Research – May 2021
USA Psychiatric Diagnoses and Treatment in Wolfram Syndrome
UK TREATWolfram Trial – update from Prof Tim Barrett – February 2021
Estonian Research February 2021 – Liraglutide, 7,8-DHF and their co-treatment prevents loss of vision and cognitive decline in a Wolfram syndrome rat model.
UK TREATWolfram Trial – Adult’s update from Dr Ben Wright – February 2021
Due to continued Coronavirus pressure this year on delivery of adult Queen Elizabeth hospital medical services, we were requested to pause further recruitment to the TreatWolfram study. This was disappointing, but understandable. We remain committed to the safe delivery of this trial, and will seek to re-open recruitment again as soon as permitted to do so. People currently enrolled to the trial will continue to be supported by the trial team. Whilst some members of the trial team have been redeployed to other hospital roles during this time they will continue to respond to any trial questions raised but may take longer to do so. Their contact email is NeurologyResearchTeam@uhb.nhs.uk
USA Trial and Research Updates from Dr Fumi Urano – February 2021
Adult Trial Recruitment Webinar – 9th November 2020
Virtual Tour of I-Stem Research Labs, France – October 2020.
For those interested in research you might like to watch this short video from the I-Stem Research Centre in France.
I-Stem is the largest French laboratory for research and development dedicated to human pluripotent stem cells, of embryonic origin or obtained by reprogramming gene. https://www.istem.eu/en/
The video has subtitles for the researchers speaking in French, so apologies to those who are VI.
USA Trial Update from Dr Fumi Urano – September 2020
TREATWolframTrial Update from Prof Tim Barrett – September 2020
Wolfram Syndrome Treatment Pipeline – Updated September 2020
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Two new red bars added.
Wolfram Syndrome Treatment Pipeline – Updated August 2020
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TREATWolfram Update from Prof Barrett – 15th June 2020
TREATWolfram Adult Recruitment Update from Dr Wright – 12th June 2020
USA update from Dr Fumi Urano – 25th April 2020
An easy read version of the medical research paper below.
USA Research Paper – Diabetes reversed in mice with genetically edited stem cells derived from patients – April 2020
TREATWolfram Trial Update – March 2020
USA Update from Dr Fumi Urano – February 2020
Zebrafish Model Update, UK Research (linked information on 2018 trials page) – February 2020
Wolfram Syndrome Treatment Pipeline – Updated October 2019
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How do faults in the WFS1 gene lead to cell death in Wolfram syndrome? – posted Feb 2020
BiP/GRP78 binding protein/glucose-regulated protein 78
BSA bovine serum albumin
cAMP cyclic adenosine monophosphate
CHOP C/EBP homologous protein
CISD2 CDGSH iron sulphur domain protein 2
CL control
DIDMOAD diabetes insipidus, diabetes mellitus, optic atrophy, & deafness
DKA diabetic ketoacidosis
DMEM Dulbecco’s modified Eagle medium
DNA deoxyribonucleic acid
DSMB Data Safety Monitoring Board
ECL electrochemiluminescence
ER endoplasmic reticulum
ERIS endoplasmic reticulum intermembrane small protein
ETDRS Early Treatment Diabetic Retinopathy Study
GABA gamma amino-butyric acid
GFP green fluorescence protein
GRP94 glucose-regulated protein 94
HZ heterozygous
ICD International Classification of Disease
ip intraperitoneal
IRE1 Inositol requiring 1
KO knockout
LDH Lactate dehydrogenase
MAA Marketing authorisation application
MRI Magnetic resonance Image
PBMC Peripheral blood mononuclear cells
PCR polymerase chain reaction
PERK protein kinase like ER kinase
PIP Paediatric Investigation Plan
RNA ribonucleic acid
SERCA sarco-endoplasmic reticulum calcium ATPase
shRNA short hairpin RNA
T1DM Type 1 diabetes
UPR unfolded protein response
WFS1 Wolfram Syndrome 1
WT wild type
WS Treatment Pipeline – January 2024
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WS Treatment Pipeline – November 2023
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WS Treatment Pipeline – September 2023
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WS Treatment Pipeline – uploaded Dec 2022
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WS Treatment Pipeline – Uploaded January 2022
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Wolfram Syndrome Treatment Pipeline – Updated September 2020
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Two new red bars added.
Wolfram Syndrome Treatment Pipeline – Updated August 2020
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Wolfram Syndrome Treatment Pipeline – Updated October 2019
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Contact & Company Info
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Phone: 01903 211358
Email: admin@wolframsyndrome.co.uk
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