New 'wonder drug' for Cystic Fibrosis hailed as the 'closest thing to a cure' for the life-shortening genetic illness... and it has already improved one boy's breathing by 50 per cent

• Kalydeco (Ivacaftor) is a revolutionary blue pill developed in the US and now approved for Australia's Pharmaceuticals Benefits Scheme (PBS)
• CF sufferers take it twice a day, it has been shown to improve lung function and help patients gain weight
• Myles Hobson, 12, was a last-minute inclusion in a trial being undertaken in at the Children's Hospital, Westmead
• His lung function has almost doubled since he began taking the medication just two months ago
• Their mother admits she 'fell apart' when told both her children had CF after watching a close friend die form the disease a few years before 
• Currently the drug can only help those with a G551D mutation of Cystic Fibrosis, work continues on a version for the most common Delta F508
• More than a million Australians have the Cystic Fibrosis gene and most don't know it  

By Frank Coletta for Daily Mail Australia

Published: 20:13 EST, 11 November 2014 | Updated: 04:39 EST, 12 November 2014

Myles Hobson is living, breathing proof, of the benefits of Kalydeco, the 'wonder drug' which is giving real hope to Cystic Fibrosis sufferers.

More than a million people in Australia carry the CF gene and a child is born with the disease every four days. It is the most common, life-shortening genetic illness. There is no cure.

When he began the trial at the Children's Hospital Westmead in Sydney's west, Myles Hobson, 12, was in serious physical trouble - his lung function had dropped in the 40 per cent range.

Myles Hobson performs a lung function test at the Children's Hospital Westmead. His mother Karen insists he has improved dramatically in just two months on the revolutionary drug Kalydeco (Ivafactor)

Myles' lung function is nearing 90 per cent now, after just two months on the trial at the Children's Hospital, almost double what it was a few weeks ago

Regular visits to the CF clinic in hospital are required to keep Myles and other CF patients well enough to do the simple things like attend school

The Hobson family on an all-too-regular visit to hospital. Both Emily (front) and older brother Myles have Cystic Fibrosis

As luck would have it, the only reason the Singleton Heights Public School student in northern New South Wales was able to take part was when one of the 20 participants dropped out.

'I was horrified to think why someone had to drop out,' said his mum Karen.

But the dramatic improvement after just two months in the trial has provided the Hobsons with great hope for the future because their daughter Emily, 8, also has CF.

'It's been a long and amazing few weeks and Tuesday marked two months of Kalydeco (treatment) for Myles,' she said.

'It's a miracle, it's the closest thing we will get to a cure for CF I think, a real breakthrough.

'His health was really bad, I had a little boy who was pale and tiny and he'd miss weeks at a time from school, since he's been taking it he hasn't missed a single day through sickness.' 

'His health was really bad, I had a little boy who was pale and tiny and he'd miss weeks at a time from school, since he's been taking it he hasn't missed a single day through sickness' said Myles' mum Karen Hobson

Infections caused by bacteria in the lungs can lead to lengthy hospital stays for CF patients like Myles Hobson

The range of medications and pieces of medial equipment Myles needs to use to treat his CF

Researchers say that Kalydeco is able to treat the underlying cause of CF by helping 'unlock that gate' and restore the function of the CFTR protein. Other CF treatments have focused on symptoms only

Kalydeco (or Ivacaftor) is a pill taken twice a day for the treatment of CF in people ages 6 and older with the G551D mutation. It's been found to help improve lung function and lower sweat chloride levels and helps patients gain weight

Kalydeco (or Ivacaftor) is a pill taken twice a day for the treatment of CF in those aged six or older and who have the G551D mutation. There are more than 1000 known mutations of the CF gene.

This helps improve lung function and lower sweat chloride levels and helps patients gain weight, all indicators that the drug is working.

The defective gene in Cystic Fibrosis leads to a build-up of thick mucus in the lungs, pancreas and other organs. When the lungs become clogged, it makes it very difficult to breathe but also traps bacteria in the airways. This can lead to infections and inflammation often causing severe lung damage, and eventually, respiratory failure.

Pancreatic problems are ever-present also, with the mucus preventing the release of digestive enzymes that help the body break down food and absorb important nutrients.

CF is considered just as deadly as heart disease and cancer but there are no outwardly visible signs someone is suffering the condition.

Researchers say that the Kalydeco drug is able to treat the underlying cause of CF by helping 'unlock that gate' and restore the function of the CFTR protein.

This allows for a proper flow of salt and fluids on the surface of the lungs. This helps to thin the thick, sticky mucus caused by CF.

Ready for another check-up. Myles puts on a brave face as he gets set to undertake more treatment for CF

Karen looks back at the birth of her second child, Emily, eight years ago and admits what she and husband Andrew were told, broke their hearts.

'They told us to take her home and love her and that we'd probably not have her for long.'

Within a few weeks the couple would learn both their children had Cystic Fibrosis.

'I fell apart. I already knew how serious a condition it is, I had a friend who had CF, we grew up together and she passed away at a young age,' she said.

'But I can't believe how much our lives have changed in two months.

'Myles lung function is now at 87 per cent - that is the highest it has been in his life.'

Just a few months ago, Myles was doing it tough, his lung function had fallen to 47 per cent. But things have turned markedly. His weight gain is also significant.

'It's astronomical - his last weight on Tuesday (at hospital) showed he has put four kilos in just two months,' his mum added.

'That is huge for Myles. He has so much energy and for the first time ever is looking for food in the middle of the night.

'His chest X-ray is the best it has ever been. He is a different little boy and we cannot be happier and more excited for his future.

'A few weeks ago some of the mums got together for a game of netball in 35 degree heat and Myles was playing with us. Normally he'd be looking for a tree to sit under but he was out there running around for more than an hour.' 

'Let's do this!' Myles Hobson has been part of a trial of the Cystic Fibrosis 'wonder drug' Kalydeco and his improvement since taking the little blue pill has stunned his family

The Federal Government recently approved the drug for listing on the Pharmaceutical Benefits Scheme (PBS), helping at least 250 Australians affected by the G551D gene mutation.

'The PBS subsidy of this medicine, which would otherwise cost approximately $300,000 a year per patient, will bring great relief to the patients and the families of people affected by this life threatening condition,' said Health Minister Peter Dutton.

'With this new treatment many patients can experience an improved quality of life with reductions in respiratory and gastrointestinal complications, improved lung function and fewer hospitalisations.'

Sister and brother Emily and Myles Hobson both have Cystic Fibrosis but the advent of Kalydeco treatment in Australia has given them and their family real hope for the future

Myles Hobson leaves hospital after more treatment for his Cystic Fibrosis. By taking Kalydeco twice daily he has dramatically improved his lung function which was seriously low just two months ago

Kalydeco is also being used in a Phase 2 clinical trial targeting the most common Delta F508 mutation, in combination with another oral medication, VX-809.

Myles will remain on the trial with 19 others until May next year. His sister 'a pocket rocket dancer', according to her mum, will be eligible to receive the same medication within the next month.

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