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MAHSC to showcase experimental medicine successes on International Clinical Trials Day

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Manchester Academic Health Science Centre (MAHSC) invites researchers, clinicians and scientists from across its partner organisations to find out more about our groundbreaking  Experimental Medicine (EM) research at the first MAHSC Clinical Research Facilities Experimental Medicine Showcase event on 20th May 2014.

Already, our researchers have shown encouraging results in newborns with rare genetic disorder Lysosomal Acid Lipase Deficiency. Infants with this condition typically die prior to the age of 6 months.  Two of three patients in a recent study have survived beyond their first year, and they continue to receive the novel treatment administered during this first-in-child trial (see further details in text box to the right).

This free event will bring together scientists and clinicians from across the breadth of the partnership's disciplines in order to learn more about our developments and achievements in this exciting field, as well as offering opportunities to explore new and vital translational research collaborations.

Key speakers will include MAHSC Director Ian Jacobs, Chair of the Experimental Medicine Strategy Board Chris Griffiths, and Directors of our Clinical Research Facilities (CRFs) Dr Andrew Wardley and Professor Ian Bruce.Lunch will be provided and there will be an opportunity to network with some of our leading researchers and clinicians from across the MAHSC partnership.

EM trials conducted within our CRFs have not only allowed for the most promising new research to have a direct benefit on the lives of patients, but have also proven invaluable in our collaborations with industry, where they have had a significant impact on the journey towards new disease therapies and medical devices.

MAHSC has been extremely successful in securing Medical Research Council funding for Stratified Medicine research programmes. These consortia-led programmes, in partnership with industry, aim to identify and analyse patients to better direct therapy and to gain a deeper understanding of the differing mechanisms of disease and treatment responses.

In 2012, MAHSC partners were awarded £12.5 million by the National Institute of Health Research (NIHR) to drive our experimental medicine research forward. This money supported three CRFs across the partnership: The Wellcome Trust NIHR CRF, a generic facility based at the

Central Manchester NHS Foundation Trust; a dedicated oncology CRF at the Christie NHS Foundation Trust; and a dedicated Respiratory and Allergy CRF at the University Hospital of South Manchester NHS Foundation Trust.

Closing the event - held to celebrate International Clinical Trials Day - MAHSC's Director, Professor Ian Jacobs, will look to the future of EM as a vital factor in the run-up to the next phase of NIHR funding in 2017, when MAHSC members will compete nationally to secure additional Biomedical Research Units (BRU) and Biomedical Research Centres (BRC).

See the full programme here. Places are limited, so please do not delay in registering your attendance here.

Lysosomal Acid Lipase Deficiency novel treatment trial

The NIHR/Wellcome Trust Manchester Children's CRF recruited patients into an Open Label, Multicentre, Dose Escalation trial. The trial evaluated the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of a new, novel enzyme replacement treatment in children with growth failure and low life expectancy, due to Lysosomal Acid Lipase Deficiency. This is an exceptionally rare, inherited genetic disorder. The first in child Phase I trial has been an outstanding success with two patients living beyond their first year. This first-in-child trial has shown encouraging results with two of three patients at this site achieving the primary endpoint of survival to 12 months of age and showing improvement in other disease-related parameters. These two patients continue to receive treatment on study.