Drug Discovery of the Year Award 2018

A new medicine used to treat an ultra-rare inherited disorder which leaves babies without a fully functioning immune system has been named the British Pharmacological Society’s Drug Discovery of the Year 2018.

The award is made to the researchers from GlaxoSmithKline’s rare diseases division responsible for developing Strimvelis, a medicine used to treat adenosine deaminase (ADA) deficiency; ADA is essential for maintaining healthy white blood cells that fight off infections and a deficiency causes a condition called severe combined immunodeficiency (SCID). People with SCID lack virtually all immune protection from bacteria, viruses, and fungi and are prone to repeated and persistent infections. Children affected usually live in isolation due to their vulnerability to infection which has led to the condition being named ‘bubble baby syndrome’. Without effective treatment, survival is rare beyond two years.

Strimvelis is used in patients with ADA-SCID who cannot be treated by a bone-marrow transplant because they do not have a suitable, matched, related donor. Strimvelis contains cells derived from the patient’s own bone marrow, some of which (CD34+ cells), have been genetically modified to contain a working gene for ADA. Strimvelis is the first CD34 stem cell gene therapy to be licensed anywhere in the world.  The first child to be treated with Strimvelis was in March 2017.  Prior to this, only a single other individual, also from Europe, had ever accessed gene therapy to treat an inherited ailment outside of a clinical trial.

Professor Carole Longson, Director of the Centre for Health Technology Assessment at the National Institute for Health Care Excellence (NICE), said: “Strimvelis represents an important development in the treatment of ADA-SCID, offering the potential to cure the immune aspects of the condition and avoid some of the disadvantages of current treatments. We believe this means that children born with ADA-SCID will now have a better chance of being able to lead as near normal a life as possible, going to school, mixing with friends, free from the constant threat of getting a potentially life-threatening infection.”

David Webb, Society President and Christison Professor of Therapeutics and Clinical Pharmacology at the University of Edinburgh, presented the Drug Discovery of the Year Award to Jonathan Appleby, Chief Scientific Officer for Gene Therapy in GSK Rare Diseases and Project Leader for Strimvelis, at a prize-giving ceremony at the Society’s annual conference, Pharmacology 2017. Jonathan said:

For many of my colleagues in GSK, working on Strimvelis with our collaborators from the Telethon Institute for Gene Therapy in Milan has been the highlight of our professional careers. To see this innovative therapy successfully given to patients in a commercial setting is very rewarding. I truly hope that this achievement will lead to other similar treatments being approved for use in other diseases.

Dr Thomas Blackburn, Chair of the Society’s Industry Sub-committee said:

Strimvelis represents the beginning of tomorrow’s medicines today - an exciting time for personised medicine.

Published: 13 Dec 2017 in Society news