05:51am Wednesday 16 October 2019

Trials could lead to new treatments for rare inherited disease

Writing in the New England Journal of Medicine (NEJM) Professor Paul Morgan, a leading research specialist in Clinical Immunology and Cardiff University’s Dean of Medicine, considers the effectiveness of three new treatments, currently under trial, for Hereditary Angioedema.

Hereditary Angioedema is a rare disease, estimated to affect between one and five thousand people in England and Wales.  Sufferers present with painless, recurrent swelling, usually of the hands, feet, arms, legs or face, and sometimes involving the throat.

Current treatment works by restoring levels of C1 inhibitor – low in the patient’s body.  C1 inhibitor is made from blood plasma, and concerns have been raised about the safety of this treatment because of the possible transmission of viruses like Hepatitis C, and also because of difficulties with administering the treatment, especially in emergency cases.

“The preferred treatment for Hereditary Angioedema should restore normal C1-inhibitor levels in sufferers” writes Professor Morgan.  

“However, because of concerns about the possibility of the transmission of viruses, and the fact that C1 inhibitor is not an ideal treatment for patients to administer, there is a desperate need to find alternative approaches.”

In his article, Professor Morgan considers the effectiveness of three new treatments which could help provide more effective options for the routine and emergency treatment of the disease.

The first is the creation of a new formulation of C1 inhibitor called Cinryze, which is treated to eliminate the risk of the spread of viruses. The second is Rhucin, a small molecule enzyme inhibitor that replaces some of the functions of C1 inhibitor, and the third is Ecallantide a small protein that also mimics C1 inhibitor. In trials, each of these has proved effective in acute attacks.

Professor Morgan adds: “The availability of three new drugs suggest that new ways of treating Hereditary Angioedema could soon be on their way.

“Each of the three new agents helps reduce the duration of attacks as well as the risk of laryngeal obstruction – which will save lives.

“These trials provide an important step forward; however, more work is needed to confirm the safety and stability of the drugs and ease of administration. These trials move us closer to creating better treatments for a small but vulnerable group of patients.”



Professor Morgan’s editorial is published today (5th August) in the New England Journal of Medicine (NEJM): www.nejm.org

A copy of Professor Morgan’s editorial and copies of the three individual research papers are available on request.

Further information, please contact:
Chris Jones
Public Relations
Cardiff University
Tel: 029 20 874731
E-mail: jonesc83@cardiff.ac.uk

School of Medicine
Cardiff University’s School of Medicine is a significant contributor to healthcare in Wales, a major provider of professional staff for the National Health Service and an international centre of excellence for research, delivering substantial health benefits locally and internationally.  The school’s 800 staff include 500 research and academic staff who teach more than 2,000 students, including 1,110 postgraduate students.
The School is based at the Heath Park Campus, a site it shares with the University Hospital of Wales, the third largest university hospital in the UK.  The School has an all-Wales role, contributing greatly to promoting, enhancing and protecting the nation’s health.

A key partner in this role is the National Health Service (NHS) in Wales, with which the School is linked at all levels. This mutual dependency is illustrated by the teaching of medical undergraduates in more than 150 hospitals located in all of Wales’ health authorities. The medical curriculum followed at the School enables students to acquire and apply knowledge, skills, judgement and attitudes appropriate to delivering a high standard of professional care. Around 300 new doctors currently graduate from the School every year and the Welsh Assembly Government has invested substantially in new teaching facilities to increase this number further

Cardiff University
Cardiff University is recognised in independent government assessments as one of Britain’s leading teaching and research universities and is a member of the Russell Group of the UK’s most research intensive universities.  Among its academic staff are two Nobel Laureates, including the winner of the 2007 Nobel Prize for Medicine, Professor Sir Martin Evans.

Founded by Royal Charter in 1883, today the University combines impressive modern facilities and a dynamic approach to teaching and research. The University’s breadth of expertise in research and research-led teaching encompasses: the humanities; the natural, physical, health, life and social sciences; engineering and technology; preparation for a wide range of professions; and a longstanding commitment to lifelong learning.

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