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 – 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

infographic of an insight into the participation of rare disease patients in research

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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

TREATWolfram Trial Update – November 2019

Liraglutide Research Paper, Estonia – November 2019

Zebrafish Model Update, UK Research – October 2019

Wolfram Syndrome Treatment Pipeline – Updated October 2019

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TREATWolfram Trial Update – August 2019

TREATWolfram Trial Update – April 2019

TREATWolfram Trial update January 2019

TREATWolfram Clinical Trial Update – 26th November 2018

How do faults in the WFS1 gene lead to cell death in Wolfram syndrome? – posted Feb 2020

USA Dantrolene Trial Update from Fumi Urano – 26th September 2018

TREATWOLFRAM clinical trial update – September 2018

TREATWolfram Trial Update from Prof Tim Barrett – 15th July 2018

TREATWolfram Trial Important Update from Prof Tim Barrett – 30 April 2018

Bio Markers update – April 2018

Accelerating clinical trials in Wolfram syndrome: development of efficacy biomarkers and patient relevant outcome measures 1st teleconference meeting report Tuesday 24 April, 2018

USA Dantrolene Trial Update – Dr Fumi Urano, 26 April 2018

TREATWolfram Update from Prof Tim Barrett – 26th February 2018

Biomarkers Research – 31 January 2018
What are biomarkers and why are they important

TREATWolfram Update from Professor Barrett – 27 November 2017

TREATWolfram Update from Prof Tim Barrett – 6 August 2017

USA Dantrolene Safety Trial Update, Dr Fumi Urano – 1st August 2017

TREATWolfram Update from Prof Barrett – 15th June 2017

TREATWolfram Update from Prof Barrett – 9th May 2017

Lay Person Summary for clinical drug trial by Prof Barrett1st March 2017

Research Update Letter24th February 2017

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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