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By inhibiting perforin specifically, there is the potential to find a potent and more selective immunosuppressive therapy with greatly reduced side-effects.
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Why Inhibit Perforin? Perforin is a pore-forming protein secreted by cytotoxic lymphocytes (CTL) or natural killer (NK) cells – immune cells which are critical for killing virus-infected, malignant and foreign cells. Perforin kills target cells by assembling into barrel-shaped pores in the cell membrane, allowing the delivery of toxic components and/or leakage of cellular contents, which promotes apoptosis.While stimulation of CTL and NK cells is essential in the innate immune system, these cells are also implicated in auto-immune disorders and infectious diseases. Current therapies to suppress activity of the CTL and NK cells have a wide range of side-effects due to multiple molecular targets being affected simultaneously. By inhibiting perforin specifically, there is the potential to find a potent and more selective immunosuppressive therapy with greatly reduced side-effects. Applications A small molecule inhibitor of perforin has great potential as a potent and highly selective immunosuppression agent for autoimmune diseases such as transplant rejection and graft- versus host disease and infectious disease, such as malaria. Small molecule perforin inhibitors provide a way to selectively and accurately modulate immune function in a controllable manner.Because pore-forming toxins such as perforin are widely distributed in nature, inhibitors of perforin may also have broader applications in anti-bacterial settings. IP Position PCT/AU2005/000291“Recombinant perforin, expression and uses thereof” filed by Peter Mac in 2005 relating to methods of expressing perforin and screening assays as well as compounds identified by the screening assays and uses thereof. National phase applications filed in several countries including USA, Europe, China, Canada, Australia, Japan, India and New Zealand with benefit of a priority date of March 3rd, 2004. Two new US provisional patent applications have been filed by Peter Mac and UniServices claiming composition of matter of the small molecule and their use as perforin inhibitors.UniServices and Peter Mac have joint intellectual property pending on further small-molecule drug candidates.