Research

Amyloidosis is a rare disease caused by the build-up of one or other of the body’s own proteins in the form of insoluble abnormal fibres, called amyloid fibrils. The amyloid deposits progressively damage the structure and function of the affected parts of the body and can lead to life-threatening organ failure.

The UK NHS National Amyloidosis Centre at the Royal Free Hospital is the only centre in the UK specialising in amyloidosis and one of the world’s leading centres for amyloid research.

The Royal Free Charity is the only charity in the UK directly supporting the national amyloidosis centre.

Research into HIV is creating breakthrough treatments which have transformed the lives of HIV diagnosed patients over recent years. Through the Ian Charleson Day Centre (ICDC) patients receive support and testing while advanced research translates into advanced diagnosis and care.

Over 3,000 patients regularly attend the ICDC. The centre runs specialist consultant-led clinics, including in viral hepatitis, TB (tuberculosis), cardiology, renal disease, neurology, rheumatology, chronic fatigue, lipid disorders, AIN (anal intraepithelial neoplasia), gynaecology and psychiatry.

The centre has access to a range of clinical trials including those for new antivirals, primary HIV infections and questionnaire-based studies with a dedicated research team.

You can support the centre by taking part in HIV clinical trials to help find the treatments for the future.

Myeloma is a cancer that affects cells in the bone marrow called plasma cells. As the cancerous plasma cells fill the bone marrow, the body is not able to make enough normal blood cells. This can lead to anaemia, bleeding problems and infections.

Through the support of the Royal Free Charity, Dr Andrea Knight is working in collaboration with a team from the Czech Myeloma Group in Brno, analysing bone marrow samples from patients at different stages of Multiple Myeloma. This is ground-breaking research into this disease, which remains incurable, and it is hoped that the study will lead to a novel tumour immunotherapy.

The myeloma practice at the Royal Free Hospital sees approximately 30 newly diagnosed patients each year and follows over 100 patients through treatment at any one time.

A neuroendocrine tumour (NET) is a rare tumour that can develop in many different organs of the body. It affects neuroendocrine cells, which release hormones into the bloodstream. The cause of neuroendocrine tumours is not fully understood.

For many people with NETs, the progression of the cancer is slow but for some people it may grow rapidly and spread to other parts of the body, causing a range of debilitating symptoms. 

The NET unit at the Royal Free Hospital is internationally renowned for the treatment and care of neuroendocrine tumour patients. The first UK centre to achieve European Centre of Excellence status, it receives patient referrals from across the UK as well as abroad.

As well as delivering patient care, the NETs team has a strong research programme aimed at increasing understanding of the condition and supporting the development of better personalised treatments for patients. The team’s research is supported by funding from the Royal Free Charity thanks to the generosity of supporters and donors, as well as other funding sources.

The research programme incorporates both laboratory and clinical research, with projects including:

• genomic studies looking at targeting specific aspects of tumour cell biology which can be used for earlier diagnosis and therapy
• advances in molecular PET imaging
• fibrosis related to intestinal carcinoid tumours
• carcinoid heart disease
• radionuclide targeting of neuroendocrine tumours
• impact of the microbiome on tumour growth and implications for therapy

A research project made possible by the Miranda Filmer Fund, and led by leading neuroendocrine specialist Professor Martyn Caplin, is advancing the use of genomic profiling to treat neuroendocrine tumours (NETs).

This study explores why some cells within a patient are more aggressive, why their change their structure, and how they respond to treatment. The aim is to increase understanding of which treatments work better on which patients.

There are different types of neuroendocrine tumours, NETs and NENs. This research is focussed on the most aggressive form which sadly affected Miranda Filmer, one of our amazing, courageous and inspirational patients. 

Read more about our neuroendocrine cancer research study, supported by the Miranda Filmer fund.

Started in November 2014, this UCL project is led by Dr Steve Pereira and sponsored by charitable funds, including Pancreatic Cancer UK, Gemma Fund, Nicki’s Smile as well our charity fund Fiorina. Its aims are to identify patients with early pancreatic cancer by testing symptomatic ​‘high risk‘ patients with a panel of blood biomarkers. Validation of a diagnostic biomarkers panel combined with a ​‘symptoms tool’ for early detection of pancreatic cancer could potentially save hundreds of lives in the UK every year.

Research findings so far into identifying early biomarkers for pancreatic cancer are promising. The project has also found that some early symptoms which patients have reported are often not recognised as possible pancreatic cancer in its early stages. The study aims to adopt these promising biomarkers for pancreatic cancer into routine clinical practice as well as alternative diagnostic strategies.

Our aim is to reduce the delay between the appearance of symptoms and a confirmed diagnosis of pancreatic cancer. This project brings together subject experts from London and Liverpool to join the dots between symptoms, early markers of the disease and access to treatment. We should be able to identify and treat patients quickly and more efficiently to give them the best chance against pancreatic cancer.

Our findings will also provide primary care physicians (such as GPs) with a rapid diagnostic pathway for patients with pancreatic cancer.

Scleroderma, also called systemic sclerosis, is a complex and serious rheumatic disease that affects much more than the joints and muscles. Unfortunately, many patients develop scarring and inflammation in the blood vessels, lungs, kidney, and intestinal tract. This leads to problems that can be life threatening including pulmonary fibrosis, pulmonary hypertension, and renal failure.

Fortunately, research efforts have improved the outcome for patients and work led by the Royal Free London and University College London has underpinned the progress.

The Royal Free Charity has provided generous support for research, patient care and education in scleroderma. Our unit cares for well over 1,000 patients with this rare disease and is the largest centre in the UK and one of the leading centres internationally. There are strong links with research scientists at UCL and with many key clinical services at RFL including plastic surgery, cardiology, pulmonary hypertension, respiratory medicine, nephrology, dermatology and gastroenterology.

Highlights and projects that are being undertaken include:

• Stem cell transplantation: For some patients at risk of severe internal organ disease autologous stem cell transplantation has emerged as a treatment. Unfortunately, it is very intensive and sometimes recipients do not survive the treatment to gain the long-term benefit. Recently we have hosted a visiting fellow form Utrecht, a lead centre in Europe with unrivalled experience and expertise. This allowed us to optimise our patient assessment and approach to align it with the best international standards.
• Better prediction of life-threatening complications: One of the challenges of systemic sclerosis is that development of major heart, lung or kidney complications is hard to predict. We have worked on integrating routinely collected clinical information for lung and heart tests and developed new blood tests (called biomarkers) to predict complications before they develop, or at an earlier stage when treatment may be more effective. One key project called MODERNISE Scleroderma will develop new ways of harnessing and integrating data to improve risk prediction for patients.
• Development of new medical and surgical therapies: The Royal Free Centre, linked to the new NIHR Royal Free Clinical Research Facility and the ongoing research laboratories, including collaborations across UCL, is at the frontline in testing new treatments. Globally, three drugs have been approved for systemic sclerosis or scleroderma associated lung fibrosis in the past two years and Royal Free Hospital has been involved in key trials of all these agents. New drugs are emerging, but additional research funding can help to unlock the full potential and permit exciting scientific studies that will unravel the mechanisms of systemic sclerosis and lead to the next trails to try and achieve the benefit of stem cell transplantation without its associated risk. In addition, we have the largest programme for ​“autologous fat transfer” led by Professor Peter Butler, that represents a uniquely effective treatment for facial fibrosis that has not previously been treatable.
• Unravelling the gut in scleroderma: We are very grateful for donations to the Royal Free Charity that have specifically supported research into the intestinal complications. This has already led to new research and better understanding of patients at most risk and we also are gaining insight into new disease mechanisms and moving to test effectiveness of emerging drugs. Gut complications impact quality of life and can be life-threatening and represents a major unmet need.

Donated funds have helped cover the relatively modest costs of small pilot studies looking at the possible factors underpinning the link between fibroblast activation and tissue damage that we think may underlie progression of fibrosis in the skin and lungs. We have been able to undertake some new work that hopefully will allow us to secure a larger research grant to support laboratory studies.