Liver specialist on brink of cure

Professor Ed Gane is one of those exceptional New Zealanders we should all know more about.

By Mark Crysell of Sunday

Ed Gane helped set up the national liver transplant unit and pioneered global drug trials that led to a cure for hepatitis C.

And now he stands on the cusp of curing a deadly hereditary disease which has ravaged generations of families.

Ed Gane
Professor Ed Gane (Source: Sunday)

Hereditary amyloidosis is a rare genetic disease that causes liver cells to produce an abnormal protein which accumulates in the body, causing damage to the heart, nervous system and gut.

The first symptoms appear in those aged between 20 and 40 and are fatal within 10 years.

Heartbreakingly there’s a 50/50 chance you can hand it on to your children.

It impacts around 50 New Zealand families and it is very likely Gane knows all of them.

He’s with them as they’re diagnosed, he speaks to their families, helps them through liver and heart transplants and holds their hands as the disease takes its ultimate toll.

Two years ago, Gane was offered the opportunity to conduct the first human trial of an extraordinary new medical technology which allows scientists to target and edit faulty genes in DNA.

CRISPR (clustered regularly interspaced short palindromic repeats) is a natural process found in bacteria, which has evolved to protect them from viruses.

CRISPR gene editing is being hailed as a lifesaving medical breakthrough, but some feel it leaves the door open for scientists to create super humans free of undesirable traits.

“Gene editing brings huge opportunities for medicine, but also big challenges, both morally and ethically,” Gane said.

“New Zealand has incredibly strict, regulatory and ethical guidelines on how we can develop CRISPR. I think this is a huge advance in medicine and it can fill medical needs for people who have untreatable conditions. That’s really where we should be using this new technology.”

Gane’s international standing and our low rate of Covid infections during the early days of the pandemic meant 12 of his amyloid patients were the first in the world to benefit from this new research.

It sounds like something out of science fiction — but it’s real and it’s here.

A three-hour infusion of a drip containing the gene editing treatment binds itself to cells inside the liver which find the gene that produces the abnormal protein.

Molecular scissors then cut out the faulty gene and DNA naturally fuses back together.

Once it’s gone, the liver cells will no longer produce the abnormal protein.

Nobody knew how successful this trial would be, but two years on, early results suggest all participants will be cured of this fatal disease and that means real hope for the children of the people who carry the amyloidosis gene.

“It’s miraculous,” Gane said.

“Not only does it provide a potential cure for everyone living with hereditary amyloidosis, but it could also provide a cure for the tens of millions of people living with other inherited lethal diseases.”

Three other CRISPR trials are underway at New Zealand Clinical Research’s campus, which makes Auckland one of the largest gene-editing centres in the world.

Article/video credit: One News