What is Intrathecal clinical trials?
Intrathecal treatment is simple where Elaprase goes through the spinal cord by means of a special port-a cath. The rationale for administering Elaprase intrathecally is that intravenously Elaprase does not cross the blood brain barrier where GAGs (waste matters) are also built up and stored in the brain leading to mental retardation.
Establishing a clinical site in Australia
A few days ago, we sent a letter to Genzyme Australia, the Australian distributor for Elaprase. Our discussion with Genzyme Australia has been to communicate with Shire's Senior Management in the USA about establishing an intrathecal ERT trial site in Australia.
We have been championing this cause and our discussion with Genzyme thus far has been positive and our push is gaining momentum. We have the full support of our specialist doctor that will conduct the trial, along with other MPS-II parents and our media contacts. We have submitted a compelling case and are optimistic of a favourable outcome.
Phase I/II clinical trials
The initial results of the phase I/II intrathecal trials been conducted in the United States and the United Kingdom have been very positive and promising and trials are still ongoing.
There is currently no published data but we expect to have a few more details on those trials from Shire in the coming weeks. We understand it is a clinical trial and different groups of participants will have different results and experiences.
Phase clinical II/III
Phase II/III of the trial is expected to commence later this year and we understand that trials will continue in the United States but will also be extended to other countries that weren’t included in phase I/II trial.
Trials will also continue in the UK. From discussions with our sources, Shire is looking to extend the trial sites to other countries and this is why we have requested Australia is included.
Australia is a developed country with excellent committed Doctors, improving medical facilities, and research centres. Our Government both State and Federal provides significant funding to our hospitals and medical care. Based on this its makes perfect sense to include a trial site in Australia.
Given Australia’s profile, we ask why a major pharmaceutical company such as Shire not included Australia in clinical trials thus far.
The reasons could be quite complex or it could be quite simple and these are some of the possible explanations:
1. Australia does not have as many MPS-II sufferers as compared to the USA or the UK. (18-20)
2. No interested stakeholders in Australia have ever requested a possible clinical trial site to be established i.e. by the Health Minister, the Health Department, doctors, patients or others.
3. The current Health Department guidelines make it almost impossible for MPS-II patients with any sign of central nervous system involvement (CNS) to get access to Elaprase intravenously.
4. These guidelines asked patients to sign an agreement which basically gives the Life Saving Drugs Program permission to remove them from the program at anytime if CNS is determined.
5. Where patients are considered to have CNS involvement they have been removed from the program with no where to turn for treatment. Our son Christian was removed based on CNS involvement early 2012 but we managed to have that decision overturned in May this year.
We eagerly wait to hear from Shire.
We will provide an update as soon as we have more to report