Royal Children’s Hospital professor’s flight for the kids

I started my first flight lessons in medical school, but flying can be an expensive hobby, and at that time, I was just a medical student, not very much in the way of money, and then soon afterwards, got married and kids, so everything was put on hold. But I’ve always been interested in flying, and the journey started in medical school, and about 20-odd years ago, I started flying again, got my private pilot’s license and then an instrument rating, flew in the US. I use flying as a hobby, seeing the great surroundings of Australia, but I also do outreach clinics, seeing children in Griffith and in Wagga. I fly out, do a clinic for the day and come home. So it’s fun, but also work.

I think many people have passions – I have a passion for my field of neurology, paediatric neurology, and the children I look after, and my other passion is flying. Putting the two together to make a difference is fantastic, in my mind.

AA: What kind of plane do you usually fly, and what do your outreach patients think?

AK: I started off like everyone, flying Cessna 150s and Warriors and Arrows and Diamonds, and then I graduated to a Cirrus.

My first flight around Australia in 2017 was in a Cirrus SR20, a single-engine plane which I think was about 210 horsepower. Now I’m in a SR22 with 315 horsepower at the front – again, single engine, but it’s a Cirrus I’m doing this trip in. It is the same plane [I use for outreach clinics] – Wagga takes me just over one hour to fly, Griffith is one hour 15 minutes, and the plane cruises at 180 knots, so it’s pretty quick, and that’s what I use for my day-to-day flying.

Many of the kids I look after are really happy to talk about flying, and I am happy to show them videos and the plane, and they’re all interested. I do have patients right around Australia who come and see me in Melbourne, and along this trip, I will visit some of my patients – not to treat them, but to say hello – in Cairns and in Busselton, in Townsville, Port Macquarie, all different places. They like to talk about aviation, and I love talking about aviation as well.

AA: What kind of work do you do at the Royal Children’s Hospital?

AK: A neurologist looks after all things from the brain, spinal cord, nerves and muscles.

And if you really think about what kind of conditions can be affected by a disorder in one of those areas, it’s things like epilepsy, obviously easier things like migraine, also developmental problems, cerebral palsy, inflammation in the brain, stroke, neuromuscular problems such as muscular dystrophy and conditions such as spinal muscular atrophy. All those conditions are very serious, severe – many of them lead to early death, unfortunately – and my life is dedicated to looking after kids with neurological problems. Anything you can think of which is severe and serious, that’s what a neurologist looks after.

AA: You’re looking to raise money to support children with rare neurological conditions – can you tell us about what kind of conditions they are?

AK: Let’s start from basics. In essence, it is for children who have rare disorders, which are disorders where the frequency of that condition is less than one in 2,000 – but if you add all the rare disorders together, it actually is about 10 per cent of the population. Now, what we do know is that many of these rare conditions start in childhood, and one third of children who are affected by a rare disorder don’t live to their fifth birthday. Most of these conditions are genetic in type, 70 per cent, and there are gene therapies and precision therapies now available for many conditions that are rare as described, so conditions like spinal muscular atrophy. On our website, there are stories about two children, Axel and Jacob, with spinal muscular atrophy.

This disorder occurs one in 10,000 births, and in the more severe type of SMA, children don’t survive until any longer than two years of age – but with research and progress in treatments, what has happened over the last eight years in spinal muscular atrophy, children have gone from no treatment and talking about death before age two to now having gene therapy that, if administered early, as in Axel, children live normal lives, walk at the right time, and enjoy life, everything that every other child can have.

So, there’s been an incredible explosion of treatments that have made a difference, and I’m hoping that those treatments are given to children as early as possible. Indeed, we have for SMA now a newborn screening program so that treatment is given early – but there are other conditions that are now being researched, and there are gene therapies or other precision medicine treatments that will make a difference. This centre we’re raising funds for is designed that we will have dedicated beds, dedicated infrastructure, nursing staff, medical staff, and this will combine with research, both in Australia and around the world, to make a difference to our children.

AA: Can you tell us more about the centre and what the $4. 5 million will fund?

AK: The $4. 5 million is not really for the treatments themselves. Those can be provided as part of research programs, also as part of government funding for gene therapies such as in spinal muscular atrophy. What we’re raising is $4. 5 million to build a precinct at the Children’s Hospital with dedicated beds, dedicated infrastructure, and dedicated staff to provide gene therapy to a single patient. There is a lot of work that goes into it, obviously, for a good cause, but this is going to be an efficient way to provide treatments in the best way, in dedicated beds. So that’s where the $4. 5 million is going initially, and that will have four dedicated beds as part of the precinct. But we all see that we are just at the leading edge of gene therapies and precision medicine in our lifetime, and I suspect over the next decade, this will be the norm, and the centre will then grow to many more beds.

So, $4. 5 million starts the project and gets dedicated beds for a centre at the Children’s Hospital in Melbourne. It’s not like any other centre cannot provide gene therapy, but what I believe will happen is that once you start this sort of a centre, it will be replicated in all the major centres around Australia, which will help all the children of Australia.

AA: You’re going to be flying for over 50 hours and covering 27,000 kilometres – can you give us an idea of your flight plan?

AK: I have planned it for approximately 21 days, around three weeks, to get right around Australia, and that takes into account one or two rest days and also the weather.

Obviously, I’m flying in winter. The southern part of Australia will be in the midst of winter, whereas the north, we won’t have rain, and I should be OK. In the north of Australia, there’ll be multiple stops – I think each leg, I will be flying two legs a day, at three hours in the morning, three hours in the afternoon, with a fuel stop in the middle. My first stop will be Aldinga or Port Lincoln, then on to Ceduna, and then along the coast to Albany, Busselton, and then up the west coast and just really circumnavigating Australia. Three weeks is what I have planned. It could be more or slightly less, but I think three weeks is a good estimate for the flight.

AA: And you’re hoping as well that this trip will raise awareness of these conditions?

AK: I think the purpose of this is, obviously to raise money, but to, you know, raise awareness of rare conditions, the impacts on our children and families. And part of this is to tell the story of kids like Axel and Jacob and other children. The way I see this is that we have established therapy in spinal muscular atrophy, we’re getting therapies in Duchenne muscular dystrophy, and as many of these rare conditions are genetic in type, it’s not a big jump to say that those treatments will come to fruition at some stage. So, it is to raise awareness, but also to establish the centre, initially at the Children’s but hopefully in other centres around Australia.

Patients like Axel and Jacob really make the story, and once you hear that story from the families, no one can feel that we shouldn’t be doing more, but there are lots of other conditions where there will be a huge positive impact over time. I don’t believe that a lot of people know that rare diseases really affect 10 per cent of the population, and I am sure many of the listeners out there will have a family member or a friend or someone who may very well have been affected by one of these rare diseases, and we can make a difference. We’re in a very lucky time in the world where there’s lots of research, lots of things happening to make a difference, and precision therapies are really the leading-edge care that we can provide, and that’s where we’re at.

To donate to or support Professor Kornberg’s campaign, visit https:// flyforthekids.org.au/