Sheffield Institute for
Translational Neuroscience


SITraN Communications
Rebecca Brown
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Latest News


Promoting the discovery of new treatments for Hereditary Spastic Paraplegia

Hereditary spastic paraplegia (HSP) is a genetically heterogeneous motor neuron disorder that causes significant gait disturbance and disability. There are at least 40 different genes that are implicated in HSP, but many of these are rare, with only a few affected families worldwide. It is estimated that half of HSP cases are due to mutations in the spastin gene (Spast), which encodes a protein that regulates the cytoskeleton.

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Dementia Road Show Events in Yorkshire and Humber

Encouraging health care professionals to become involved in dementia research is the aim of a series of events organised by Division 4 staff of the Yorkshire and Humber Clinical Research Network (CRN).

The Dementia Roadshow Events showcase the exciting dementia research that is taking place throughout the region. Inspired by the Prime Minister's challenge on dementia, the CRN team are building the portfolio of dementia studies in Sheffield and the region, facilitating collaborations between dementia active communities and trusts.

The first Dementia Roadshow Event took place in February 2016 at Rotherham Doncaster and South Humber NHS Foundation Trust and was chaired by Dr Daniel Blackburn Consultant Neurologist and Senior Clinical Lecturer in Neurology at SITraN. Giving an overview of current projects and clinical trials in Sheffield, Dr Blackburn presented his research investigating novel mechanisms of diagnosis of Alzheimer’s disease and other dementias using electroencephalography (EEG) and conversation analysis. There was great interest from clinicians and health care professionals keen to become involved in dementia research. The event gave rise to further plans for supporting new principal investigators in developing their skills within dementia research.

Dr Blackburn said:

"Yorkshire and Humber has recruited the highest number of participants into dementia studies compared to other English research networks. However most participants are recruited from University Hospitals in large urban centres. These roadshow events are attempting to increase access for all people in Yorkshire with dementia to take part in research. This needs local investigators and greater awareness of research into dementia."


Dan dementia.jpgDementia Research Nurse Victoria Murray and Neurologist Dr Daniel
Blackburn presenting dementia research in Sheffield.

There are three further opportunities to attend one of these Dementia Research Engagement events in various locations throughout Yorkshire and Humber (places are limited for some of the events so please book early, lunch/breakfast is available at the events):

  • Wednesday 16th March 2016 between 11.30-1pm (lunch is available from 12.30) - Mayfield Suite at Fulwood House, Old Fulwood Road Sheffield S10 3TH – for further information please contact – *booking is required*.
  • Wednesday 23rd March 2016 between 12.30-2.30 (lunch is available at 12.00) – South West Yorkshire Partnership Foundation Trust Dementia Research Engagement event in Annabel Room Hatfeild Hall, Aberford Road, Stanley, Wakefield, WF3 4JP. For further information please / 01977 605285.

*booking is required*. There are 20 places available for this event.

Drop In Event:

  • Friday 26th February 2016 from 9-12am - Leeds and York Partnership NHS Foundation Trust at the Mount Annexe, 44 Hyde Terrace Leeds, LS2 9LN. (*Drop in event no booking required*). Breakfast available.

Details of a Principal Investigator Workshop will follow.

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PVC Dame Pam Shaw gives prestigious Croonian Lecture

Our new Pro-Vice-Chancellor for the Faculty of Medicine, Dentistry and Health, Professor Dame Pam Shaw, has been selected to give the prestigious Croonian Lecture at The Royal College of Physicians in London on Wednesday, 10 February 2016.


Pam pic.jpg

Pro-Vice-Chancellor for the Faculty of Medicine, Dentistry & Health,
Professor Dame Pam Shaw

An internationally acclaimed neurologist, Professor Shaw has devoted her career to improving the lives of people affected by  motor neuron disease (MND). She will be talking about her translational neuroscience approach to  developing new effective treatments for MND at the Sheffield Institute for Translational Neuroscience (SITraN), a research institute devoted to MND and related neurodegenerative diseases, which Professor Shaw founded and directs.

Professor Shaw said:

"It is a great honour to be invited by the President of the Royal College of Physicians to give the Croonian Lecture, as part of the annual Advanced Medicine Conference on 10th February 2016.  This prestigious lecture was endowed by William Croone, an eminent and inspiring 17th century physician-scientist.  Interestingly, he was particularly intrigued in his work by the motor system and the 'nature and laws of muscular motion'.

During my lecture, I will describe how we can harness the exciting developments in neuroscience and translate these insights into benefits for patients and families facing the devastating neurological illness of motor neurone disease (MND).  I will be highlighting the progress being made by the talented medical and scientific teams within SITraN at our University."

The Croonian Lectures are given at the invitation of the Royal Society and the Royal College of Physicians. They are a legacy of William Croone (1633 –1684), an English physician and one of the original Fellows of the Royal Society and the Royal College of Physicians. William Croone pursued research in several important subjects of his day, including respiration, muscular motion, and generation, with widespread influence.

William CroonePortrait of William Croone, painted by Mary Beale 1860.

The Croonian Lecture is given on 10th February at 12:05pm as part of the conference in Advanced Medicine, 8-11 February 2016 at the Royal College of Physicians in London. The RCP will be live streaming parts of this conference – for more details follow @RCPLondon #RCPAdvMed on Twitter.



Getting involved in dementia research

The new website of the South Yorkshire Dementia Research Advisory Group (SYDEM-RAG) is now up and running and provides a wealth of information on dementia research in the wider Sheffield area and how members of the public can get involved. It will serve as a platform to bring researchers and the public together to inform dementia research and improve the lives of people affected by the disease. The website covers the latest news on dementia research in Sheffield, details on projects, as well as local events and researcher profiles.

SYDEMRAG websiteSouth Yorkshire Dementia Research Advisory Group launches new website


The SYDEM RAG was set up in September 2013 to enhance patient and public involvement in dementia research. The group includes people with dementia, carers and family members who are passionate about improving the care of people living with dementia and help researchers better understand the disease from the patient and carer perspective. For dementia researchers, the group is an extremely valuable resource, as they benefit from the input and feedback from the group to develop their projects based on the needs of people affected by dementia. 

The group meets quarterly at SITraN and always welcomes new members. To join the group , access the group as a researcher or to promote dementia news, events and research projects on the website please contact

SYDEM RAG members

Members of the SYDEM RAG at their quarterly meeting at SITraN.





Accelerating the development of treatments for Charcot-Marie-Tooth Disease

Acetylon Pharmaceuticals, Inc., joins the long-standing collaboration between SITraN researcher Dr Andy Grierson and the Hereditary Neuropathy Foundation (HNF). The aim of the collaboration is to develop treatments for Charcot-Marie-Tooth disease (CMT), the most common inherited disorder of the peripheral nervous system which affects close to 3 million people worldwide. Acetylon, a leader in the development of selective histone deacetylase (HDAC) inhibitors for the treatment of cancer and other critical human diseases, will provide a therapeutic compound that will be tested in a preclinical zebrafish model of CMT developed by Dr Grierson at the University of Sheffield for its potential in the treatment of CMT.

P1080757.JPGDr Grierson’s drug screening research for CMT is supported by the Hereditary Neuropathy Foundation (HNF) as part of their Therapeutic Research In Accelerated Discovery (TRIAD) programme, which fosters collaboration among academics, government and industry to accelerate potential treatments for CMT. The zebrafish model of CMT2A, developed by Dr Grierson, at the Bateson Centre is used as a platform to test therapeutic compounds that may lead to potential treatments for people affected by CMT2A, the second most common form of CMT.

Dr Grierson said “The zebrafish model offers a unique opportunity for preclinical testing in CMT2A, the most common form of axonal CMT. With support from HNF we have refined and validated this model. Through this new partnership we will discover whether HDAC6 inhibition is a viable therapeutic approach”.

Charcot-Marie-Tooth (CMT) is a progressive disease. Early signs include high arched feet, curled toes, and claw-like hands. Many of these signs begin subtly and may go undiagnosed for years, leading to legs and arms becoming deformed and difficult to use. Severe, chronic pain is common, and there is no cure. To date, over 80 mutated genes associated with CMT have been identified, with more being discovered each year. The most common form of CMT is CMT1A, for which a treatment is currently being tested in a Phase 3 clinical trial. CMT2A, caused by a mutation in MFN2, is the second most common form of CMT.

The Hereditary Neuropathy Foundation (HNF) is a non-profit organization whose mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth disease (CMT) and related inherited neuropathies. The charity supports patients and families with critical information to improve quality of life, and supports research that will lead to treatments and cures.

“HNF recognizes the sense of urgency to get treatments to patients and families as quickly as possible”, said Sean Ekins, Chief Science Officer of HNF. “Whenever possible, we try to connect companies with researchers who are pushing the envelope to find potential treatments for CMT. Whether it is a focus on drug development, high throughput screens with FDA approved drugs, or novel compounds that could lead to new targets, we are constantly searching for ways to help accelerate this process.”  

HNF and partner organization Hannah’s Hope Fund co-sponsor the Global Registry for Inherited Neuropathies (GRIN) to collect clinical and genetic information on patients diagnosed with the various forms of inherited neuropathies in order to advance therapy development for these debilitating disorders. To join the patient registry, visit For further information, visit

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