UCL and Glaxo to develop drug antibody treatment for rare disease

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.
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