Monday, 22 October 2018
Private Members' Business
I rise in support of this motion. Haemophilia and bleeding disorders affect far more Australians than people are aware. Everyone is familiar with the story of haemophilia affecting the royal families, and that is the nub of the problem: the genes are the cause of these unfortunate disorders. But there are other bleeding disorders besides haemophilia. There are various forms—one milder form called Christmas disease, another one called Von Willebrand disease, and there are other very rare blood-coagulation-factor disorders that cause problems. There are these pesky little things call platelets which run around and plug up the holes when you cut yourself, and there are disorders of platelets as well. But the coalition government—as governments before us have done and governments in the future will do—administers and delivers probably the best blood product and treatment system in the world for people afflicted with bleeding disorders. I don't know of a better system. We spend about $1.2 billion on the National Blood Agreement, where the federal government kicks in that amount of money and shares the costs with the states around all of the state based haemophilia and other blood-disorder treatment centres.
There is good news. The former speaker mentioned the desire for a cure. The good news is there is a cure on the way. There are cases of gene therapy where the gene for producing factor IX is introduced into the person whose genes don't make it, and they have been cured, basically just with gene therapy.
Like CAR T therapy for lymphomas and leukaemias, it's being researched around the world as we speak. All those doctors, scientists and biochemical wizzes are working on gene therapy for factor VIII haemophilia, which is the more commonly known and severe haemophilia. We also have clever biochemists and scientists who've developed recombinant factor VIII, factor IX and various other replacement therapies so that you don't have to rely on blood products that are pooled from multiple donations, which is what was the case when I was working in the haemophilia centre at Royal Prince Alfred Hospital and Westmead Children's Hospital.
There was one stage in the eighties, before HIV was understood and hepatitis C was understood when, unfortunately, whole cohorts of children with haemophilia got infected because, if your products were pooled from 100 donors, only one of them had to have hepatitis C or the at-that-time-unknown HIV for the person to be infected. It was a tragedy. But recombinant technology has saved that, and gene therapy is on the way.
Parents do have a lifelong struggle. Many of them adjust, but for the child or young adult it really does mean a different life. But the wonders of modern medicine are making huge gains in the quality of life. Instead of big haemorrhages and massive bruises—which you couldn't imagine if you had normal clotting—distorting and damaging joints, in the future, hopefully, those days will be gone. There are other treatments that they can give—sprays into the nose, under the skin or infusions—which will increase the amount of factor VIII.
So there is light at the end of the tunnel for many of these people who, through no fault of their own—it just runs in their family; it's recessive and then it pops up mainly in boys. There is really amazing technology, and Professor Rasko at RPA and other people overseas have been instrumental in this initial trial of gene therapy with factor IX. It's Australian scientists who are really at the cutting edge. The European body that provides certification for these therapies and the FDA in America are both fast-tracking their assessment of this. The Medical Services Advisory Committee in Australia, which is like the PBAC that approves drugs, is looking at these new therapies as we speak.
I thoroughly support this motion because it is a dreadful impost on unlucky people, but we have a great system in Australia.