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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:

Monoclonal antibodies: Antibodies are fast becoming some of the world’s most successful drugs, treating a wide range of diseases ranging from arthritis to cancer. MRC-funded discovery science involving animal research has underpinned this rapidly burgeoning field from the very beginning, from César Milstein and Georges Köhler’s isolation of mouse monoclonal antibodies in 1975, to Sir Greg Winter’s work in the late 1980s to develop humanised monoclonal antibodies.

The early experiments that Milstein and Köhler conducted involved the use of spleen cells from a mouse immunised with sheep red blood cells. Their monoclonal antibody (mAb) production involved animals being given antigen injections and blood being collected. Later improvements, developed by MRC funded scientists Michael Neuberger and Greg Winter, led to new types of antibodies (such as chimeric recombinant mAbs, CDR grafted mAbs and phage display synthetic mAbs) which have helped reduce the need for mice in the production of these antibodies. However, mice have not altogether disappeared from the process; transgenic mice are now crucial for producing monoclonal antibodies, with one of the first such transgenic mice developed by Neuberger.

These antibodies subsequently progressed through clinical trials to establish safety and efficacy and then were licensed for the treatment of various diseases such as rheumatoid arthritis, and even led to the development of home pregnancy kits. Today, monoclonal antibodies are amongst the most widely used drugs for treating cancer.

Gene therapy for treating ‘Bubble boy’ disease: Severe combined immunodeficiency (SCID) is the name given to several rare inherited diseases where children are born with defective immune systems. It is also known as the ‘Bubble boy disease’ because affected children are extremely vulnerable to infectious diseases, and some of them have become famous for living in a sterile environment. In the most severe forms, children are unable to fight off even mild infections and, without treatment, will usually die within the first year of life. Children with SCID are usually treated with expensive weekly injections of enzyme replacement therapy for life. In 2010, MRC-funded scientists at the UCL Institute of Child Health developed a mouse model (a special strain of mice to study a particular human disease or condition) for this rare disease and confirmed that a new gene therapy approach delivered improvements in safety and efficacy. Crucially, data obtained from this mouse model provided the confidence for regulatory approval for human trials, allowing the work to proceed to human clinical studies (phase 1 and 2) and the successful treatment of 20 children with this life-threatening disease, permanently freeing them from weekly injections.

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

For more examples of how animal research benefits human health – everything from Alzheimer’s disease and asthma inhalers to transplants and Parkinson’s disease – visit the human health section of the Understanding Animal Research website.