An oligonucleotide-based drug that may represent a new treatment option for multiple sclerosis has started Phase I trials in the UK.
The antisense drug, ATL1102 (ISIS 107428) was licensed to Australia's Antisense Therapeutics by Isis Pharmaceuticals of the USA. At present, there are only two therapies specifically approved to treat MS - beta interferon products from Schering, Serono and Biogen and Teva Pharmaceuticals' Copaxone (glatiramer acetate) - and these confer only limited benefits.
ATL1102 targets VLA-4 (very late antigen-4), which is expressed on the surface of lymphocytes and binds to the adhesion molecule VCAM-1 on endothelial cells. The hypothesis is that inhibitors of VLA-4 will prevent lymphocytes from being able to cross the blood-brain barrier in the central nervous system and exert the damaging effects on the myelin sheaths surrounding nerve cells that are the hallmark of MS.
Mark Diamond, Antisense Therapeutics' managing director, said: "This trial is the first step in potentially improving the treatment of multiple sclerosis, which is a major unmet medical need. We are hopeful that this antisense drug, the first to be developed for multiple sclerosis, may provide patients with a new treatment option."
The double-blind, placebo-controlled study will evaluate the pharmacokinetic and safety profile of ATL1102 in approximately 40 healthy volunteers. It is being conducted at the Charterhouse Clinical Research Unit of the Ravenscourt Park Hospital (formerly Stamford Hospital) in London.
Under the terms of a five-year collaboration between the two companies, Antisense Therapeutics pays Isis to manufacture oligonucleotide drugs and is responsible for their development and commercialisation. ATL1102 is the Australian firm's lead project, while another oligonucleotide targeting psoriasis (ATL1101) remains in preclinical development.