The phase II study, led by Professor Peter Horby of the University of Oxford on behalf of the International Severe Acute Respiratory and Emerging Infection Consortium (ISARIC), is the second drug trial to be funded through the Wellcome Trust’s £3.2 million Ebola therapeutics platform.
TKM-Ebola-Guinea, developed and manufactured by Tekmira Pharmaceuticals, is a synthetic small interfering RNA (siRNA) therapeutic designed specifically to target the strain of the Ebola virus responsible for the present outbreak in Guinea, Liberia and Sierra Leone. It works by blocking certain genes of the virus, thereby reducing viral replication.
An earlier version of the TKM-Ebola drug (targeted at a different strain) has been tested in healthy human volunteers. The efficacy of the Guinea version will now be evaluated in patients with a confirmed diagnosis of Ebola virus infection in a single-arm study called RAPIDE-TKM (Phase II Rapid Assessment of Potential Interventions & Drugs for Ebola-TKM). Results of the trial are expected in the second half of 2015.
The RAPIDE-TKM study will be led by the University of Oxford in partnership with the Sierra Leone College of Medicine and Allied Health Sciences, the Sierra Leone Ministry for Health, the WHO-based Special Programme for Research and Training in Tropical Diseases (TDR), the UK Department for International Development (DFID), Public Health England and GOAL Global.
Professor Peter Horby, Associate Professor of Infectious Diseases and Global Health at University of Oxford, who is leading the trial, said: "There are still around 10 new cases of Ebola being diagnosed every day in Sierra Leone. It’s therefore essential that we push forward with clinical trials while we still have a realistic chance of getting answers about which, if any, of the candidate treatments can save lives in this, and in future outbreaks."
Colonel Professor Foday Sahr, Commanding Officer of the joint medical unit in Sierra Leone and a principal investigator on the study, said: "The start of this trial is a very important event for Sierra Leone and the other countries affected by Ebola, it is an exciting development because research is essential in this outbreak so that we can determine what might work for these cases. This clinical trial is a highly collaborative effort and we are working closely with international partners so that together we can get the evidence that is needed. The team working with the patients in the treatment centres demonstrate this partnership in action because they are a mix of local and overseas staff who are working side by side to deliver these trial treatments to the patients."
Dr Jeremy Farrar, Director of the Wellcome Trust, which is funding the trial, added: "The recent surge in new Ebola infections in Guinea and Sierra Leone should serve as a stark warning that this epidemic is far from over. Almost a year on from the first confirmed case, we’ve reached a crucial stage where several large scale trials are gathering steam, but we still don’t have any proven treatments. It’s therefore heartening to see this latest trial of TKM-Ebola getting underway after so much hard work from the research team and partner agencies. We’re very proud to be supporting their important work."
The Wellcome Trust Ebola therapeutics platform was set up in September 2014 to enable multiple partners to quickly establish clinical trials at existing Ebola treatment centres. TKM-Ebola-Guinea is the second candidate drug to be evaluated through the platform. A previous trial of the antiviral brincidofovir was abandoned earlier this year following a sharp fall in the number of Ebola cases at the study site in Liberia.
About Oxford University's Medical Sciences Division
Oxford University's Medical Sciences Division is one of the largest biomedical research centres in Europe, with over 2,500 people involved in research and more than 2,800 students. The University is rated the best in the world for medicine, and it is home to the UK's top-ranked medical school. From the genetic and molecular basis of disease to the latest advances in neuroscience, Oxford is at the forefront of medical research. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. A great strength of Oxford medicine is its long-standing network of clinical research units in Asia and Africa, enabling world-leading research on the most pressing global health challenges such as malaria, TB, HIV/AIDS and flu. Oxford is also renowned for its large-scale studies which examine the role of factors such as smoking, alcohol and diet on cancer, heart disease and other conditions.
GOAL is an international humanitarian agency that operates in 15 countries worldwide. Founded in 1977, it has been working in Sierra Leone for 16 years, delivering a range of health, livelihoods, child protection, psychosocial support, water, sanitation and hygiene programmes in Western Area and Kenema District. They have been responding to the Ebola emergency since the summer of 2014. Apart from managing an Ebola Treatment Unit in Port Loko, they are working with affected communities, survivors and supporting health facilities across Freetown, Kenema, Bo and Bombali Districts, as well as leading a social mobilisation consortium with national coverage.
Tekmira Pharmaceuticals Corporation
Tekmira Pharmaceuticals Corporation is a biopharmaceutical company focused on advancing novel RNAi therapeutics and providing its leading lipid nanoparticle (LNP) delivery technology to pharmaceutical and biotechnology partners. Tekmira has been working in the field of nucleic acid delivery for over a decade, and has broad intellectual property covering its delivery technology. Tekmira is based in Vancouver, Canada and Seattle, USA.
About the Wellcome Trust
The Wellcome Trust is a global charitable foundation dedicated to improving health. We provide more than £700 million a year to support bright minds in science, the humanities and the social sciences, as well as education, public engagement and the application of research to medicine. Our investment portfolio gives us the independence to support such transformative work as the sequencing and understanding of the human genome, research that established front-line drugs for malaria, and Wellcome Collection, our free venue for the incurably curious that explores medicine, life and art.