Gout is a form of arthritis that affects tens of millions of people, including more than 45,000 New Zealanders, and is particularly common in Māori and Pacific Islanders, and among men. The painful disease usually affects the fingers and toes, particularly the big toe, with swelling occurring when high levels of uric acid in the blood crystallise in the joints.
Promising new gout treatment
However, a promising new gout treatment, currently labelled BCX4208, developed by Lower Hutt’s Industrial Research Ltd (IRL) and New York's Albert Einstein College of Medicine, may soon be helping alleviate the problem. American biotech firm BioCryst Pharmaceuticals Inc has announced that Phase 2 human clinical trials of the drug will be conducted this year in patients suffering from gout.
The gout trial is currently recruiting 120 patients at 12 hospitals in the United States and is expected to run until September 2010.
Dr Richard Furneaux, who heads IRL’s Industrial Biotechnology research, says BCX4208 has been some 7 years in the making and is a “next-generation purine nucleoside phosphorylase (PNP) inhibitor”. This means it aims to prevent uric acid forming in the blood. The human trial is designed to work out what different doses of BCX4208 (taken by mouth) do to a patient’s uric acid levels in the blood.
The enzyme PNP (purine nucleoside phosphorylase) plays a role in a number of inflammatory diseases, so the researchers are hoping the new drug might eventually be used to treat other conditions as well as gout. Dr Furneaux says the existing 2 treatments for gout have a range of unwanted side effects, but so far BCX4208 seems to be remarkably well tolerated. Existing drugs also work differently in that they try to reduce the build-up of crystallised uric acid in the joint rather than targeting the enzyme responsible.
“BCX4208 is the second drug candidate we have discovered in collaboration with Professor Vern L Schramm, the Ruth Merns Chair in Biochemistry at the Albert Einstein College of Medicine at Yeshiva University in New York City. Our first is in a pivotal Phase 2b human cancer trial for cutaneous T-cell lymphoma, with results expected in 2010,” says Dr Furneaux.
Development of New Zealand’s pharmaceutical industry
The success of the trials in the United States could translate into big money for New Zealand’s biomedical business. Already, the US has invested over $200 million into IRL’s drug research.
“We are hoping that more and more of this will be done here, and we can actually have a pharmaceutical development industry in New Zealand.”
IRL receives milestone payments and royalties on net sales. “Importantly, we are working towards greater capture of the hundreds of millions of dollars of development spent in New Zealand as we expand,” says Dr Furneaux.
IRL also has other drug candidates moving into development under licence to another American company, Pico Pharmaceuticals Inc.
Biotech Learning Hub Organisation profile: IRL
Watch a TV3 news report on the promising new gout treatment.