The report in 1996 by the National CJD Research & Surveillance Unit of a new human transmissible spongiform encephalopathy, variant Creutzfeldt-Jakob Disease (vCJD) linked to the epidemic of Bovine Spongiform Encephalopathy (BSE) in cattle, greatly increased the importance of surveillance and research in this whole field.

First, it became apparent that surveillance was necessary not only to track the incidence of the disease and explore its epidemiology but was also vital to the initiation of public health action (for example, in notifying the blood transfusion service about blood donations made by someone with vCJD). Second, the potential scale of the epidemic of vCJD, which still remains uncertain, has created an imperative for high quality research directed at better understanding the molecular basis and pathogenesis of transmissible spongiform encephalopathies; devising a diagnostic test; and developing effective treatments.

We need to ensure a high level of patient referral to the trial whilst maintaining the public health surveillance function, and delivery of the care package for patients with this disease. The Department of Health has asked the Medical Research Council (MRC) to proceed with the PRION-1 initiative to establish a clinical trial infrastructure for investigating quinacrine and other candidate treatments. In addition, the Department has asked the MRC to offer clinical monitoring within the Prion 1 framework to those patients who have chosen to receive pentosan.

In order to manage the situation in the United Kingdom well, we must make best use of available clinical resources and knowledge. There are eight key principles that we must follow. These are set out below:

A number of organisations, teams and committees enable the eight principles to be adhered to and, in this section of the letter, their roles and responsibilities are set out briefly.

a. Departments of Health and other related bodies in England, Scotland, Wales and Northern Ireland

The Departments of Health, as the central government departments with overall responsibility for public health and health services, are responsible for determining policy, allocating resources, co-ordinating implementation and monitoring progress. In these tasks, they are assisted by the Health Protection Agency (a new body, which incorporates the former functions of the Public Health Laboratory Service) and by local arrangements in the devolved administrations. Other expert committees, including the Spongiform Encephalopathy Advisory Committee (SEAC), and agencies including the Medicines and Healthcare Products Regulatory Agency (MHRA) provide advice and support in specialist areas.

b. National CJD Research & Surveillance Unit – Lead Consultant: Professor Richard Knight

The National CJD Research & Surveillance Unit (NCJDRSU) was established in 1990, and has a high international standing. It is based in the Western General Hospital in Edinburgh and serves the whole of the United Kingdom. The Unit is responsible for monitoring the characteristics of all forms of CJD to identify patterns, trends and risk factors; and, for informing the appropriate body so that prompt, effective action can be taken to protect public health. The Unit has cerebrospinal fluid and genetics laboratories which analyse samples from patients with suspected CJD as an aid to clinical diagnosis. The Unit is responsible for the national pathological surveillance of CJD, involving both histological studies on brain and all other tissues, and protein analysis. The Unit undertakes research on the clinical and diagnostic features of CJD, epidemiology (including the national Case Control Study of risk factors for sporadic and variant CJD, and the investigation of geographically associated cases of CJD), and histopathology and protein analysis of brain and other tissues and fluids. The Unit houses a large bank of brain tissue, cerebrospinal fluid and blood samples which are used for diagnosis and research both within the Unit and with external research groups.

As part of the surveillance activities, staff from the Unit visit each referred patient and their family whenever possible. The Unit provides expert advice, counselling and support for patients with sporadic and variant CJD and their families. The Unit also administers the National Care Package for patients and families with CJD, which co-ordinates care for such patients and their carers throughout the United Kingdom. Staff in the Unit are involved in major programmes of research (much of it in collaboration with other centres in this country and abroad), and co-ordinate the European CJD Surveillance Network. They also provide advice and expert input to the work of government departments, scientific committees and other organisations and agencies. The Unit is a WHO Collaborative Centre for Reference and Research on the Surveillance and Epidemiology of Human Transmissable Spongiform Encephalopathies.

c. National Prion Clinic – Lead Consultant: Professor John Collinge

The National Prion Clinic at the National Hospital for Neurology and Neurosurgery provides expert clinical advice on diagnosis and management, as well as counselling and support for patients and their families, especially those with, or at risk of, inherited prion disease. The National Prion Clinic has close links with patient organisations. The National Prion Clinic is closely linked with the MRC Prion Unit (see below). Staff at both the MRC Prion Unit and the National Prion Clinic advise governments, other bodies and provide expert input into scientific committees. The National Prion Clinic has a key role in leading and co-ordinating the MRC PRION-I clinical trial, and will provide opportunities for patients to participate in clinical research programmes.

d. Medical Research Council (MRC) Prion Unit

The Medical Research Council (MRC) Prion Unit, based at the Institute of Neurology, Queen Square, London, was established to undertake a major multidisciplinary research programme into human prion diseases, and to translate basic research to clinical practice in the NHS. It has high international standing. It has core research programmes covering molecular genetic studies of human prion disease susceptibility, modes of transmission and their characterisation; clinical research to identify early diagnostic markers and longitudinal studies to establish a cohort for subsequent clinical trials; and, the development of human diagnostic tests and therapeutics. In particular, the MRC Prion Unit in collaboration with the MRC Clinical Trials Unit now has a major role in developing protocols for clinical trials of potential treatments for CJD. The Unit provides laboratory diagnostic services to neurologists and neuropathologists in the UK and overseas, including prion gene analysis and analysis of tonsil and brain biopsies and is a WHO Reference Centre for diagnosis and investigation.

e. Consultants in Communicable Disease Control (CCDCs)

Under existing arrangements for local reporting of CJD, (www.doh.gov.uk/cjd/cjdlocal.htm), neurologists inform their local CCDC of all cases of CJD. CCDCs have a responsibility to investigate each patient’s medical history, to report to CJD Incidents Panel, and to advise on infection control measures.

f. CJD Incidents Panel

The CJD Incidents Panel is an expert committee, set up in 2000 by the Chief Medical Officer in England. Its remit is to assist all bodies responsible for the provision and delivery of healthcare to decide on the most appropriate action to take to handle incidents involving potential transmission of CJD and vCJD between patients through clinical interventions

g. The CJD Therapy Advisory Group

The Chief Medical Officer in England has also commissioned a group, under the leadership of Sir Michael Rawlins, the Chairman of the National Institute for Clinical Excellence (NICE), to advise on strategic planning of future CJD drug trials. Its main objectives are to maintain an overview of research likely to lead to the development of therapeutics for human disease and identify potential therapeutics for CJD that may be suitable for clinical trial.

h. CJD Tissue Management Steering Committee

This expert committee aims to facilitate human tissue collections on CJD and related diseases and where necessary to help manage access to scarce resources to support research in this area.

In addition to these formal structures, the two patient support groups – the CJD Support Network and the Human BSE Foundation – provide valuable support and advice to families.