2012 Progress Report
Siatskas, of Monash University, was awarded a project grant in 2011, supported
by the Trish Foundation to examine novel treatments combining immune
regeneration and gene therapy approaches.
A rational approach for the
treatment and cure of autoimmune diseases such as MS requires incorporation of
three fundamental processes: suppression of the inflammatory response,
restoration of self-tolerance and regeneration of the target cells or tissues.
Current clinical practice at best addresses the first problem but there are no
clear strategies for the other two. To
overcome this, new therapies are desperately needed.
Dr Siatskas has
previously established a method of suppressing the inflammatory response to
tackle the first problem. This method was able to reduce clinical symptoms in a
laboratory model of MS. Dr Siatskas is now investigating a new approach to the
second problem - restoring self-tolerance. His approach uses a gene therapy
method that directly targets the self-reacting cells in the thymus. The thymus
is the organ where immune cells are trained to recognize the difference between
‘self’ cells and dangerous cells. Targeting treatment to the thymus could
prevent the release into the bloodstream of the rogue immune cells that attack
showed that treatment in the thymus prior to the onset of the disease reduced
inflammation and neurological damage. He is also investigating what happens
when treatment is started after clinical symptoms begin. These experiments are
important since they will more accurately mirror the treatment situation seen
in people with MS. While these experiments showed a slowing of symptoms, they
were not a cure, since some rogue immune cells had already entered the
bloodstream prior to treatment.
are an excellent first step since they show retraining at the thymus to restore
self-tolerance is possible. They also show that treatment strategies to
specifically target the rogue immune cells circulating in the bloodstream will
also be required.