Medical breakthroughs underpinned by animal research

The use of animals in biomedical research helps researchers better understand the biological processes that are central to our health. This is essential for developing safe and effective ways of preventing or treating disease.

For over a century, research using animals has advanced the scientific understanding of human health, and the impact of this research is so vast that it can be difficult to measure. However, some key recent examples of lifesaving treatments that were developed thanks to animal research are worth highlighting.

COVID-19 vaccine trials

Professor Sarah Gilbert and her team at the University of Oxford spearheaded a vaccine trial in which they used a safe version of an adenovirus. An adenovirus is a virus that can cause a common cold-like illness.

Previous work funded by the Medical Research Council (MRC) through the UK Vaccine Network used this adenovirus (known as ChAdOx1) by Professor Gilbert in the production of vaccines against the Middle East Respiratory Syndrome coronavirus.

Engineering a spike protein

The team engineered ChAdOx1 to make a specific coronavirus protein, known as the spike protein, from the SARS-CoV-2 virus. As a result, our immune system should in theory be able to recognise the spike protein as ‘foreign’ and form antibodies against it. And then attack the SARS-CoV-2 virus and stop it from causing an infection.

It is hoped that long lasting immunity can be provided through vaccination by ‘bluffing’ the body in this way, and by slipping in parts of the virus that do not harm, but induce the release of antibodies.

The vaccine testing involved animal trials in ferrets and non-human primates at the Public Health England (PHE) laboratories. The team also collaborated with researchers at the BBSRC funded Pirbright Institute to study the effect of this vaccine in pigs.

Vaccinating millions of people worldwide

Under normal circumstances, animal work must be completed before human trials can start. But because similar vaccines have worked safely in trials for other diseases, the work was accelerated and happened in parallel. It led to the approval by the Medical and Healthcare products Regulatory Agency on 30 December 2020.

This vaccine, commonly known as the Oxford AstraZeneca vaccine, has now been administered to millions of people worldwide.

Professor Alain Townsend’s team at the MRC Human Immunology Unit worked in collaboration with:

  • MRC Weatherall Institute of Molecular Medicine
  • Radcliffe Department of Medicine
  • University of Oxford
  • the Biotechnology and Biological Sciences Research Council’s (BBSRC) Pirbright Institute.

Further vaccine development

They have shown that a new potential vaccine against COVID-19, named RBD-SpyVLP, produces a strong antibody response in mice and pigs. It provides vital information for the further development of the vaccine.

Investing in the research and development of the second generation of COVID-19 vaccines is important because they will help fill gaps in efficacy against novel variants. It also addresses issues around production and distribution such as the requirement for cold chain supply logistics.

Find out more about the Oxford-produced RBD-SpyVLP vaccine candidate.

Llama antibody has ‘significant potential’ as COVID-19 treatment

A unique antibody produced by llamas could be developed as a new frontline treatment against COVID-19 and could be taken by patients as a simple nasal spray.

The laboratory research is led by scientists at the Rosalind Franklin Institute. The research was funded by:

  • MRC
  • Engineering and Physical Sciences Research Council (EPSRC)
  • EPA Cephalosporin Fund
  • Wellcome.

The research has shown that nanobodies (a smaller, simple form of antibody generated by llamas and camels) can effectively target the SARS-CoV-2 virus that causes COVID-19. It is the first step towards developing a new type of treatment against COVID-19.

Preparing for human clinical studies

The scientists are hoping to progress this work from the animal setting to prepare for clinical studies in humans.

Human antibodies have been an important treatment for serious cases during the pandemic, but typically need to be administered by infusion through a needle in hospital.

However, nanobodies have several potential advantages over human antibodies:

  • they are cheaper to produce
  • it is possible to deliver them directly to the airways through a nebuliser or nasal spray, so they could be self-administered at home rather than needing an injection.

This could have benefits in terms of ease of use by patients, but it also gets the treatment directly to the site of infection in the respiratory tract.

Gene therapy treatment for treating blindness

Inherited eye conditions are currently untreatable because they are caused by mutations in our DNA, which form defective copies of key genes required for normal vision. Gene therapy aims to deliver healthy copies of these defective genes directly to the retina, to correct these genetic mistakes.

MRC has been funding research into gene therapy for inherited eye diseases since 2004. Animal research in mice and dogs has been vital for establishing the necessary proof-of-concept for ocular gene therapy.

Developing a new, efficient technique

In 2011, with MRC funding, a team of scientists at the UCL Institute of Ophthalmology developed a new technique for improving the efficiency of this gene therapy. The results of which were confirmed in mouse models, a special strain of mice to study a particular human disease or condition.

Once the safety and efficacy of this approach was established in mice, the work rapidly progressed to two clinical trials. The first patients receiving this ground-breaking treatment have benefited from significant vision restoration, with more patients now in clinical trials. As well as the benefit to patients, this work is now widely regarded as a landmark for the entire gene therapy field.

Last updated: 28 April 2022

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