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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.
Dr 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 www.neuropathyreg.org For further information, visit www.hnf-cure.org
The trial of a new multiple sclerosis (MS) treatment in Sheffield led by Professor Basil Sharrack, Director of the Sheffield MS Research Clinic, and Professor John Snowden, Director of Blood and Marrow Transplantation at the Department of Haematology, is showing promising results. BBC Panorama has reported about the pioneering crossover cancer treatment that has enabled some MS sufferers with paralysis to regain their movement.
The treatment, a stem cell transplant that reboots the immune system, is known as autologous haematopoietic stem cell transplantation (AHSCT). Stem cells harvested from the patient’s own blood are used to rebuild the immune system which in multiple sclerosis mistakenly attacks the protective myelin around nerve cells leading to the debilitating disease.
Profs John Snowden (left) & Basil Sharrack:
a clinical partnership of neurology and haematology
Professor Basil Sharrack said:
“To have a treatment which can potentially reverse disability is really a major achievement.”
The treatment Autologous Haematopoietic Stem Cell Transplantation (AHSCT) is routinely used to treat cancer, but has shown some benefits in patients with active inflammatory MS and is currently the subject of an international clinical trial with Sheffield as the sole UK site. To find out more about the treatment, the Sheffield Teaching Hospitals NHS Foundation Trust has set up a dedicated website AHSCT for MS.
The BBC panorama programme, which had exclusive access to patients who received the treatment, is now available on BBC iplayer. The news was also covered on BBC News Health and on BBC Look North on 18th January 2016, 6:30pm.
To find out more about the scientists involved visit their University of Sheffield profile pages: Professor Basil Sharrack, Consultant Neurologist at the Sheffield Teaching Hospitals and Honorary Professor of Neurology, SITraN, Department of Neuroscience and Professor John Snowden, Consultant Haematologist and Director of Blood and Marrow Transplantation and Honorary Professor of Haemato-oncology & Stem Cell Transplantation.
The Medical Research Council (MRC) has led the way in finding new approaches to link up academic and industry researchers. Three years ago, in a pioneering deal with AstraZeneca (AZ), 15 projects were funded in which groups of academic researchers are investigating alternative uses for compounds that are no longer being developed by the company.
Dr Richard Mead’s group at SITraN were among the first to get involved in the project when it was launched in 2012. They are using a drug originally developed for Alzheimer’s disease by Astra Zeneca – but subsequently abandoned – for a new purpose: to investigate a cell signalling process which they suspect is involved in motor neurone disease (MND). While also bound by company confidentiality agreements, Richard says he’s “hopeful” about what they have discovered so far from studies in mice.
Gaining access to AZ’s resources, expertise and toxicity data for the compound they are using has given the group a great head start, and he is confident that it will allow them to get definitive answers to questions they’ve been chasing for many years:
“With the pharmacological knowledge and support of AZ and the data we’ve generated on this project we’ll be able to say definitively that this pathway isn’t worth pursuing any longer if it doesn’t work. Or conversely, if it does work we’ll have enough data to continue down the line of doing studies in patients.”
Naturally, Astra Zeneca gain from the deal too. The company has exclusive rights to buy back the intellectual property on any drugs that look promising, and access to information they couldn’t get anywhere else, says Richard:
“At the University of Sheffield we are experts in our animal models. Most drug companies wouldn’t have deep knowledge of the preclinical model systems for a disease like MND – it just wouldn’t be worth their while to invest in that over many years. But we can describe in detail how we will execute a study for a particular drug, depending on the pathway that it’s targeting.”
Professor Pam Shaw welcomes the families of our graduates to SITraN.
On Friday 15 January 2016, SITraN and the Department of Neuroscience celebrated the achievements of their latest PhD and MSc graduates. After successfully completing their 3-year laboratory research training, nine candidates were awarded the degree of Doctor of Philosophy: Sufana Almashhadi, Aziza Alrafiah, Khayria Alsomali, Nimah Alsomali, David Baker, Joanna Bury, Johnathan Cooper-Knock, Andreas Damianou, Aida Mohammedeid. Moreover, the Department was proud to present 32 masters students in Translational Neuroscience (18) and Clinical Neurology (14) who graduated in these popular courses established in 2011 and 2012, respectively.
MSc Clinical Neurology (left)and MSc Translational Neuroscience graduates (middle) with their course leader, and some of our PhD graduates (right) celebrating at SITraN.
Prizes for our master students:
Laura Francis was awarded the “Jonathan Stone Prize for Translational Neuroscience” for the overall highest grade in her course. Claire Green won the Department Prize for the highest research project result in Translational Neuroscience.
Both prizes for the MSc in Clinical Neurology went to Jessica Collins, the Department Prize for the highest research project result, as well as the “Irene and Richard Beard Prize for Clinical Neurology” for the overall highest grade in her course.
Our prize winners: Laura Francis (left) with Dr Janine Kirby, Claire Green (middle) and Jessica Collins (right) with Dr Thomas Jenkins and Dr Dan Blackburn.
Building on the success of the master courses, the Department of Neuroscience now offers two further masters degrees in Translational Pathology [Neuroscience] and Genomic Medicine. For more information on postgraduate studies, MSc courses and PhD opportunities, visit the PGR web pages of SITraN and the Department of Neuroscience.
Congratulations to all our students and the very best wishes for their future!
With great pleasure, the Department of Neuroscience is able to announce three successes in the annual promotions round, effective from 1st January 2016, as well as two new appointments, as follows:
Congratulations to our staff promoted and newly appointed in the Department of Neuroscience!