`SAM' begins funding drug trials for muscular dystrophy
by JENNY MONROE
A HILLSBOROUGH based charity set up less than two
years ago to seek treatments for muscular dystrophy has announced
grant funding totalling over £175,000 to evaluate three promising
CMD drug candidates in 2010. Two years ago the McCausland family received the devastating news that
their beautiful little boy Sam -who was then just one year old - has a
progressive muscle wasting condition called Congenital Muscular Dystrophy
(CMD).
Tracy McCausland and her husband William set up the zero cost charity Struggle Against Muscular Dystrophy (SAM) in April 2008. SAM recently announced the funding to evaluate three drug candidates.
Gillian Garrett, Chairperson of SAM, commented: "We believe that a focused investment in science will lead to CMD treatments which will bring hope to the many thousands of families whose lives have been affected by Congenital Muscular Dystrophy. It is now critical that we maintain this level of funding into the subsequent years of these projects."
Tracy said: "At this moment there are no effective
treatments or cures for any of this group of over 40 neuro-muscular
conditions. As a rare disease, the form of muscular dystrophy from which Sam
suffers (merosin deficient congenital muscular dystrophy, MDC1A for short)
has until now been largely ignored in research and drug development."
She added: "I know that none of us know what the future holds for our children, but in Sam's case we do know what lies ahead - and it is a life of suffering and pain unless we can find a treatment or a cure.
"The work we are doing in raising money to fund research through the SAM charity gives me hope. There is great cause for optimism that within the next five years we will at least have an effective treatment to slow the rate at which the muscles are wasting. This will potentially buy us a little more time for the scientists to find a cure - and that dream ignites and sustains the passion I have for raising the vital funds that will help maintain the research.
"I dream that Sam and all the other children who suffer from CMD will have a real chance of having a brighter future than the one currently predicted for them. Please help us to make that dream a reality."
Muscular Dystrophy
- THE three drugs which will be evaluated are an NFKappaB inhibitor, laminin 111, and a muscle specific IgF upregulator.
- A separate effort to identify disease biomarkers specific to merosin deficient CMD (MDC1A) will also be funded by SAM in association with international CMD advocacy group CureCMD. Each application received during this year's grant process was subjected to a rigorous review process that included outside peer review and Scientific and Medical Advisory Board evaluation.
- MD affects thousands of children in the UK, and causes the muscles of the body to gradually waste away. Children become dependent on wheelchairs at a very early age and as the muscles continue to waste they become quadriplegic, suffer from scotiosis (twisting and distortion of the spine) and develop painful muscle contractures. They also lose the ability to breathe and eat without assistance.
Ulster Star
15/01/2010
