Hope for Haemophiliacs
Despite the controversy and disappointment emerging from the final report of the Penrose Inquiry into contaminated blood, which led to infection and death of hundreds of Scots, including haemophiliacs, new hope is emerging in a small laboratory in Scotland.
Profactor Pharma (PFP), a Scottish biotech start-up based at Biocity near Glasgow with the backing of the Scottish Government’s Investment Bank and Glasgow based Kelvin Capital, is working to produce a safe drug for haemophiliacs called Factor VIII. By improving product availability and lowering cost, it gives new hope for existing and yet to be diagnosed haemophilia A patients around the world.
“Our Factor VIII is what is known as a recombinant biopharmaceutical, and is manufactured without any risk of disease coming from a contaminated blood source” said Dr Ian Garner, co-founder and responsible for the company’s operations. “While we are developing our product for supply to the UK and Europe, we are also very conscious of the need to supply safe Factor VIII to developing countries. Here haemophiliacs continue to face the risk of HIV and Hepatitis C infections from blood derived Factor VIII, or just as bleakly, risk having little or no treatments due to inadequate current supply.”
Factor VIII infusions for Haemophilia A are critical to prevent injury and early death from uncontrolled internal bleeding due to insufficient production fully functional Factor VIII normally made in the liver. It is estimated that 70% of the world’s haemophiliacs currently receive inadequate or no treatment at all, leading to significant suffering and many early deaths. Essentially a ‘male only’ inherited disease carried on the Y chromosome; it affects about 1 in 5,000 male births. In the UK, it is estimated there are about 6,000 haemophiliacs. There is no cure for the disease and it requires lifelong injections of Factor VIII to live a more or less normal life.
“We are grateful for the support of our early funders” said Richard Cruse, PFP’s CEO. “But it’s now important to find additional financing in order to complete our progress to human trials. We see the therapeutic protein Factor VIII project is a starting point for a new biologics industry in Scotland. With an experienced, specialist team of founders and scientists, we are confident this can be achieved here.”
About ProFactor Pharma
PFP was established to develop, commercialise and supply recombinant blood factors for the treatment of haemophilia to a severely undersupplied world market, starting with recombinant human Factor VIII (rhFVIII), and plan to have product to the market in just three years.
Haemophilia is a genetic disorder where the blood does not clot as fast as it should. Haemophiliacs spontaneously bleed into joints, muscles and internal organs causing malformation and pain. Without treatment, haemophiliacs typically die in their teens.
Haemophilia A is treated with infusions of a missing blood protein, Factor VIII, to stop bleeding. This protein is sourced either from processed human plasma or is manufactured using so called recombinant technology. Nevertheless, according to the World Health Organisation (WHO) and the World Federation of Hemophilia (WFH), almost three quarters of the worlds haemophiliacs receive inadequate or no treatment at all.
Average treatment in the western world is around 100,000 international units per year. We estimate that total production of recombinant Factor VIII is around 5BN IUs per year. That’s enough to treat 50,000 patients. But the WFH says, according to their 2013 annual report, that there are 143,000 patients under treatment. And the WHO estimates the total patient population at over half a million.
PFP’s vision is to create a sustainable and profitable business, using advanced biologics production technology, delivering currently undersupplied recombinant human blood factors, including Factor VIII, making safer treatment available to a greater proportion of the world’s haemophiliac population and increasing the opportunity for prophylactic use.