MAHSC to showcase experimental medicine successes on International Clinical Trials Day
Manchester Academic Health Science
Centre (MAHSC) invites researchers, clinicians and scientists from
across its partner organisations to find out more about our
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
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
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).
full programme here. Places are limited, so please do not delay
your attendance here.
Lysosomal Acid Lipase Deficiency novel treatment
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.