UCL News


UCL and Glaxo to develop drug-antibody treatment for rare disease

24 March 2009


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  • UCL Centre for Amyloidosis & Acute Phase Proteins
  • Pentraxin Therapeutics Ltd, a UCL spin-off company, and GlaxoSmithKline (GSK) are collaborating to develop the world's first dual drug-antibody treatment for the rare and often fatal condition amyloidosis.

    Amyloidosis is a disease caused by the build-up of abnormal proteins (amyloids) in body tissues, which leads to organ failure. The heart, kidneys, liver, and almost any other organ can be affected. Around 500 new cases are diagnosed each year in the UK, and - despite the fact that patients receive the best available therapy - the prognosis for patients is poor and new treatments are urgently needed.

    Pentraxin initially developed the small molecule drug CPHPC. Although early results were promising, it could not benefit patients in the advanced stages of the disease. "Something more dramatic was needed," explained Professor Mark Pepys, the head of Pentraxin and the UCL Centre for Amyloidosis & Acute Phase Proteins, which includes the UK National Amyloidosis Centre. "We then combined CPHPC treatment with an antibody that seeks out the amyloid deposits in the organs in mice. This combination triggered a rapid clearance of the deposits."

    With this new agreement, the research teams from UCL and GSK will work together to convert the mouse antibody into one that can be used in humans in combination with CPHPC. The aim is to find out if the benefits seen in the animal model can be replicated in patients with amyloidosis.

    The collaboration brings together UCL's clinical and science expertise and the development expertise of GSK's Academic Discovery Performance and Biopharm Units.

    "We are delighted to enter into this alliance," said Mike Owen, Senior Vice-President of Biopharmaceutical Research at GSK. "Our biopharmaceutical and clinical development capabilities and Professor Pepys's team's knowledge of the disease provide a synergistic collaboration that will greatly enhance our chances of success."