GE 'vital' for treating haemophilia

New Zealand must continue to make genetically altered therapies available to patients with haemophilia and other genetic bleeding disorders, the royal commission investigating the technology was told yesterday.

Deon York, of the Haemophilia Foundation, said genetic engineering was keeping alive New Zealand victims of the illness and even allowing them to lead near-normal lives.

Advances in biotechnology offered treatments and possibly an eventual cure.

Two exciting scenarios existed: one for gene therapy aimed at repairing the defective gene in sufferers; and the other using disease-free pigs to produce proteins capable of clotting human blood.

Haemophilia, described as the most expensive disease to treat over a lifetime, is an incurable gene disorder involving blood that does not clot correctly.

In severe cases, patients spontaneously bleed into joints and muscles, and sometimes organs and the nervous system.

It was difficult to treat until the 1970s, when clotting proteins became available from human blood plasma.

The recognition of risks from hepatitis C, which can lead to liver cancer, and HIV, which leads to Aids, left patients using potentially infected plasma to avoid bleeding to death.

Mr York said haemophiliacs now had to worry about variant Creutzfeldt-Jakob disease (vCJD), a human form of mad-cow disease that could be transmitted in blood.

In 1995, families with haemophilia had gained new hope from some "recombinant" or genetically engineered clotting proteins, which were safer than plasma-derived products.

These could be produced in quantities that meant patients could take a normal place in society, but initially were available only for children.

The foundation said there were 1602 haemophiliacs in New Zealand, in three main groups.

These included 118 people, mostly males, with severe haemophilia type A, with deficiencies of blood clotting factor VIII, and 29 with severe haemophilia B, involving a deficiency in factor IX.

Genetically engineered products had made it possible for child sufferers to be treated from birth, and play a normal role in society.

The potential future application of genetic engineering technologies to prevent or treat haemophilia was "enormous."

The foundation said it was inconceivable that New Zealand would choose to avoid such technologies, especially in light of the potential to prevent the disease.

Mr York said New Zealand should be using international models and regulation as a basis for providing re-combinant product, undertaking GE research and making gene therapy available to haemophiliacs.

- NZPA

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