Jason Wood (La Trobe, Liberal Party) Share this | Hansard source

In late October last year I was privileged to meet the Webber family and their incredible young son, Jimmy. Jimmy is 12 years old and suffers from Hunter syndrome, or MPS II. Children with Hunter syndrome miss an important enzyme essential in the breakdown of sugar molecules in the body. Consequently, these molecules remain stored in the body and cause progressive damage. The symptoms are wide ranging, but even in the mildest cases the disease can impact greatly on a person’s physical capabilities.

My initial meeting and discussion with Jimmy’s parents, Tracey and Andrew, centred on how we could obtain access to a drug called Elaprase. Elaprase is the only enzyme replacement therapy available for people suffering from Hunter syndrome. It was approved by the US Food and Drug Administration in July 2006 and the European Commission has granted a marketing authorisation for its use. While the drug is currently being assessed by the Therapeutic Goods Administration in Australia, the detailed process of assessment is too lengthy and involved for someone who needs access to the drug immediately—but I do make the point that the TGA has a very important role in this process.

Jimmy required the drug very quickly as the disease was building pressure on his optic nerve and it needed to be relieved urgently. We appealed strongly and persistently to international biotechnology company Genzyme, who are well known for their provision of charitable access. Their CEO, Mr Dan Brown, astounded me with his willingness to assist. He pushed hard for Jimmy, as he knew what was at stake. The company had to make a decision immediately. By December we were relieved and delighted to be informed of Genzyme’s ultimate decision to purchase Elaprase from another company for young Jimmy. The medication will cost several hundreds of thousands of dollars per year but will help Jimmy live a life he and his family have only dreamed of.

As seems to be commonly cited by many patients enrolled in Elaprase trials, Jimmy already feels more energetic. He is much more positive now. This drug is actually going to help him keep up with his mates and be involved in sport. His mum, Tracey, has told us that even at this early stage Jimmy is much more outgoing than before. He used to stay within himself; now he is beginning to shine.

I have been reminded throughout this journey of what can be achieved when people work together. Testimony to the undeniable strength of family and community is the amazing determination and commitment I have witnessed in the Webbers and the unflinching support extended to them by their family, friends, neighbours and schools in order to achieve this fantastic outcome. Many people have contributed their assistance: Andrew’s parents, Anne and Harry; Tracey’s stepfather, Ted; Tracey’s sisters, Denise, Liz and Narelle, and brother-in-law, Stephen; neighbours Paul and Marion; and Dr Martin Delatycki, who is now administering the drug. I must thank the offices of Tony Abbott, the Minister for Health and Ageing, and of Christopher Pyne in his former role as Assistant Minister for Health and Ageing. I also thank the staff of Beaconsfield Primary School and Beaconsfield Kindergarten.

It goes without saying that Mr Dan Brown and Genzyme have made this dream possible and cannot be thanked enough. Mr Brown’s benevolence, willingness and generosity has given this inspirational little boy access to a drug which will halt the disease process and allow him a second chance to embrace life much more fully. Genzyme have touched and transformed the lives of other Australians as well. For the past four years Genzyme has provided the drug Aldurazyme to four patients with MPS I, which is a variation of MPS II. Just today I had the pleasure of meeting 26-year-old Justin Hannan, who lives with MPS I. He is here in Canberra with his parents to lobby the government to provide funding for Aldurazyme. Justin is already on this drug, which has been kindly donated by Genzyme, and is therefore lobbying for other people with the condition. While the drug has been approved by the TGA, it is never likely to be included on the PBS because MPS I is such a rare disease. I am calling on my own government to strongly consider the appeal to grant funding so people suffering from MPS I are able to get access to the drug Aldurazyme. I am also hoping desperately that the TGA will approve the drug Elaprase and therefore help all people, young and old, in this country who have Hunter syndrome. (Time expired)

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