Shire Makes Decision about Intrathecal Clinical Trial for Australia
Our son Christian has hunter syndrome (MPS-II) which is a debilitating and life threatening metabolic genetic condition. He is only expected to live to his early teens without treatment. I am a person of faith so I refuse to allow this disease to take my child. But I also realize that ‘faith without works is death’.
I know all too well the horrible effects of this condition and in late 2007 my determination to see an end to this condition became even more personal when the last of my three brothers passed away from hunter syndrome and its related health problems. I travelled back to Jamaica to bury my brother and as I stood to read the urology at his funeral I could not help but wonder if in several years I would be doing the same for my son. At that time of grief I made my mind up that this would not be the case for my son.
Around about the same time I learnt that Shire the manufacturer of Elaprase was recruiting patients for an IT clinical trial. This trial was to get Elaprase through the spinal cord and into the brain. The weekly intravenous infusion of Elaprase does not cross the blood brain barrier and in the more severe cases leads to mental retardation, aggressive behavior and a diminished range of skills. I was therefore encouraged by the news of the clinical trial and thought there may be some hope for our Christian. I began to investigate the trial but learnt Shire was only recruiting patients in the USA & UK and unfortunately we live in Australia.
The Intrathecal clinical trials have been ongoing for some years now but I never gave up hope that one day my child could benefit from this treatment.
So in July 2013 I once again turned my attention to the clinical trials following the successful reinstatement of Christian Elaprase infusion after 16 months hiatus with the Australian Health Department.
I was grateful to be back on intravenous Elaprase and now wanted to be part of Intrathecal trial with the many other parents who want the same.
Full credit to Shire, they have invested their money in developing these costly trials to help save our MPS children, I am thankful. Yes, they are a big business that need to make a profit but so do all businesses. But what they are doing offers hope and that’s what is important to us.
I decided that since we can’t move to the USA, wouldn’t it be great if the trial could came to us. Of course I knew it would not be that easy.
Getting a trial set up in Australia would be even more difficult due issues such as demography and other governmental issues but the trial in our opinion was well worth the effort. Establishing a clinical trial site requires key people such as a lead investigator (very experienced metabolic doctor) and also a senior neurosurgeon and neurologist. These people are sometimes difficult to find as they are already stretched in a public health care system. But no pain on gain, right! And we wanted a trial urgently and safely.
Like other parents we were concerned about safety of the trial but as I speak to other mothers I have been reassured by those who are on the existing trial of the significant improvements and gains made with their son’s. But besides what do I have to worry about, anything is better than no treatment, because without intrathecal treatment my son will eventually decline cognitively to a point where his quality of life will be nonexistent.
With that being said.
I AM VERY PLEASED TO ADVISE THAT SHIRE HAS DECIDED TO EXTEND IT’S PHASE III INTRATHECAL CLINICAL TRIAL TO AUSTRALIA. THIS IS NOW IT THE FESABILITY STAGE.
This trial could potentially benefit many existing MPS-II sufferers in this country and even children not yet born. At this stage we like to say thank you to our amazing Westmead Children’s Hospital Metabolic doctor, Metabolic doctor at Princess Margaret Hospital, hunter syndrome parents Samantha Ellis and others. Shire Australia and others for helping to get the trial to this stage.
There is still a lot of WORK to be done and we still need everyone to play their part and support this next stage of the TRIAL.
Shire has offered the initial assessment of feasibility to the major hospitals where metabolic doctors practice in each capital city of Australia.
As always there is “red tape” to contend with. We have began to encounter some difficulties. There are willing doctors in different states but they cannot do this trial on their own they need the support of all the various departments in their hospitals.
I therefore implore and ask you to encourage all the relevant authorities at each hospital to please make a concerted effort to investigate the feasibility of establishing a trial site at their hospital and report back to Shire ASAHP.