Shire makes positive steps toward a trial site in Australia
About 6 months ago we successfully won our appeal with the Australian Federal health department for the reinstatement our son Christian’s lifesaving treatment after an arduous 16 month appeal process. Once that battle was over I began thinking about the next step in his life. You see I never gave up on him going to college and getting married. Prayer is an essential part of our fight to save my son but also taking practical steps is also just as important. As my other son who at the age of 4 would say “mummy you need a plan”. So that’s it I needed a plan. My plan was to get a treatment for his brain. I knew without his brain being well, life for us as a family would be difficult as raising children with neurologically involvement is very challenging to care for.
Christian is a lovely boy, a smart boy but has many difficulties and his behavior can sometimes be very challenging.
I knew there were clinical studies being conducted overseas for MPS-II with great success and I just could not understand why no studies were being done in Australia. I was perplexed and puzzled as to why? I thought I needed to do something for my son and the other MPS-II patients in this country. I began doing some investigation using the experience we gained from fighting for Christian’s intravenous treatment. Of course this was a different scenarios because the trial was being conducted by an international drug company and we needed to understand how this process worked.
As I began to investigate the process I was being told all the reasons why there wasn’t a clinical trial site in Australia for MPS-II and that there would probably not be one in the foreseeable future. Nonetheless we decided to proceed with determination and purpose to succeed in achieving an extraordinary outcome. I like to think about issues in terms of every problem has a solution.
We decided to approach Shire USA and kindly requested that Australia be considered as a trial site for the upcoming phase III intrathecal trial.
We were very pleased with the initial response we received from Shire and was encouraged by their willingness to seriously consider our request. We were advised it would take considerable time to consider Australia as possible trial site. We were also informed about some of the issues in establishing a site in another country such as Australia that until June this year has very discriminatory rules around treating patient with neurological involvement. Our goal was to ensure Australia provide MPS-II patients with treatment and that the rules didn’t became an issue for a favorable outcome. I believe this has helped with Shire’s decision about considering Australia as a place to do a trial
I am very pleased to SAY that there have been some very pleasing new developments about a trial site in Australia. Steps are being taken to progress the clinical trial site and once complete I believe a trial site in Australia is very possible in the foreseeable future.