Sometimes in science, persistence is rewarded. For more than 30 years Mark Pepys, professor of medicine at University College London, has concentrated on research into amyloidosis, an incurable disease that causes organ failure in tens of thousands of people a year worldwide.
Amyloidosis is caused by the build up of abnormal “amyloid” proteins in body tissues. Prof Pepys has long believed that the key to understanding the disease is a related blood protein called SAP, which sticks to amyloid fibres and stops enzymes removing them.
The FT has covered his work several times. My predecessor David Fishlock described in 1990 Prof Pepys’s discovery of a way to image SAP and amyloid fibres. I wrote in 1995 and 2002 about progress in developing a drug called CPHPC, which aimed to clear the destructive amyloid deposits from patients by removing the protective SAP from their blood.
Prof Pepys was working then in collaboration with Roche. But the Swiss pharmaceutical giant eventually pulled out.
“While we had promising early results [with CPHPC] they were not enough to benefit patients with advanced disease,” he says. “Something more dramatic is needed.”
That something turns out to a combination of CPHPC with an antibody – a molecular guidance system designed to seek out amyloid deposits in vital organs.
Now Prof Pepys has reached an agreement with another big pharmaceutical group, UK-based GlaxoSmithKline, to collaborate on producing a treatment for amyloidosis based on the CPHPC-antibody combination.
Although the details of this week’s deal are confidential, Prof Pepys says GSK will spend many millions of pounds working with UCL scientists, to convert their successful animal tests into an antibody-based drug for human use.
Mike Owen, GSK’s head of biopharmaceutical research, said it was realistic to aim for clinical trials within two years.
Symptoms of amyloidosis are very variable because the heart, kidneys, liver and almost any other organ can be affected. Every year 500 to 1,000 new cases are diagnosed in the UK, mainly in middle-aged and older people; their prognosis is poor.
Although amyloidosis is a rare disease, there are enough sufferers – an estimated 80,000 in the industrialised world – to bring in considerable revenues from an effective drug.
Prof Pepys points out that antibody-based medicines can sell for tens of thousands of pounds per patient, particularly if they treat a previously untreatable disease.
While staff at GSK’s research centre in Stevenage will look after the clinical development of the new medicine, “Prof Pepys will remain closely involved in the project,” says Pauline Williams, the company’s head of academic liaison. “It was critical for him to feel that he was not handing everything over to us.”