GlaxoSmithKline is linking up with scientists at the University of Dundee to find a treatment for Huntingdon's disease, marking a further push by Britain's biggest drugmaker into the rare diseases area.
The project, still years away from producing a finished drug, will build on the discovery by Dundee's Susann Schweiger of a mechanism that controls production of a disease-causing protein involved in the inherited brain disorder.
Patients with Huntington's typically develop symptoms between the ages of 30 and 50. They suffer severe movement problems, leaving them unable to walk and care for themselves, and die within 10 to 15 years of onset, since there is no cure.
Schweiger believes that by inhibiting the production mechanism it might be possible to prevent a toxic build-up of protein in the brain and thereby provide an effective treatment.
Since identifying the novel target for tackling the disease, the research team has run a pilot screening project looking for small molecules -- or drugs that can be given by mouth -- to inhibit the mechanism.
GSK will now take this work to the next stage by using its large-scale screening facilities to hunt for drug candidates. Schweiger told Reuters she hoped to have substances that are ready for a clinical trial within two to three years.
Treatments for rare or orphan diseases, affecting only a few thousand patients worldwide, can command very high prices and deliver very high profit margins.
The sector has recently attracted growing interest from large pharmaceutical companies, with Sanofi paying $20.1 billion early this year for U.S.-based Genzyme, an early pioneer in the field.
GSK, by contrast, has opted to build its presence through a series of alliances that involve limited capital up front.
It has struck collaborations with unlisted Dutch biotech Prosensa for a Duchenne muscular dystrophy treatment, and is working with Japan's JCR Pharmaceuticals on enzyme deficiency diseases, as well as investing in Italian gene therapy for bubble boy disorder, or ADA-SCID.
It has a separate research project with the University of Dundee into another orphan disease, Recessive Dystrophic Epidermylosis Bullosa, a rare disorder of the skin and mucosal linings which results in lifelong skin blistering.
(Reporting by Ben Hirschler; Editing by David Hulmes)