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Here in Europe, two developments are making waves. First, a gene-editing technique is solving a painful condition, and the Dutch have invented an electric car that charges itself.
Because I am your European correspondent for the moment, I have decided to give you a heads-up on some positive developments.
Let’s start with a break-through in medicine: gene silencing. As you probably know, gene therapy is the new black. But now, there is a variant of that idea that promises as much, or maybe even more relief to long-suffering patients.
In 2006, the Nobel Prize for Medicine was won by Craig Mello and Andrew Fire. What they had discovered was something called RNA interference. As far as I understand, this is how it works. A gene is the part of our DNA that comprises the blueprint that makes proteins in our bodies. The problem is that this gene is located deep in the cell’s nucleus, away from its protein-making abilities. So there is a short strand of genetic code called messenger RNA that bridges that gap and carries the instructions.
That is great, normally. But if a gene goes rogue and causes illness, the RNA carries that false message too. Mello and Fire found a way to stop the RNA, something they called gene silencing. Where gene therapy permanently alters the hard copy of the instructions in the DNA, gene silencing leaves the original intact but targets the instructions that it sends out into the cell. It is a ‘tweak’, not a permanent change.
Of course, discovery is lovely and it gets you fame and a Norwegian medal if you are lucky.
To translate an idea into practice, that is where the real gain lies. In the case of gene silencing, it has taken a while, but it looks like we are getting there. First, there was a trial that looked at 94 people across 18 countries who were suffering from porphyria.
Porphyria is an illness that attacks the nervous system and often also the skin. It is very, very painful. Some people call the crippling pain ‘worse than childbirth’. It can cause paralysis and patients have been known to spend half their lives in hospital, where they have to take strong opioids to stay relatively sane.
There is speculation that porphyria was ‘the madness of King George III’, which explains a lot about English history. The cause is toxic proteins and so far there has been no remedy. The trial was into a drug called Givosiran, a gene silencer, and it was spectacularly successful. The therapy cut the number of severe attacks by 74% and 50% of patients were completely clear of attacks that needed hospital treatment.
Where gene therapy permanently alters the hard copy of the instructions in the DNA, gene silencing leaves the original intact but targets the instructions that it sends out into the cell. It is a ‘tweak’, not a permanent change.
At the same time, another gene silencer, Patisiran, has been trialled for patients with amyloidosis, which attacks the autonomic nervous system. It is a horrible disease, that slowly breaks down your body until you die. Again, a rogue gene builds toxic proteins in the body, that then damages organs and nerves. Amyloidosis was usually fatal, but not anymore. Gene silencing has made the untreatable treatable.
Studies published in the New England Journal of Medicine showed that Patisiran ‘halted or reversed’ the disease, something that made the British National Institute for Health and Care Excellence say that it considers gene silencing ‘unique and innovative’.
They are in the process of approving it for the NHS, which means that it will be financed by the government. At the same time, the expectation is that what works for these diseases might also work for Huntington’s and even, maybe, Alzheimer’s and Parkinson’s and other neurodegenerative illnesses. This is why the US Food and Drug Administration said it could ‘transform medicine’.
The second innovation I would like to tell you about is called the Lightyear One. Yes, it sounds slightly Star Trek-ish and in truth, it looks like that as well. But it is a car, an e-car. But this one doesn’t need charging, because it charges itself.
It is made by a group of old students of Dutch university TU Eindhoven. In 2013 they showed its predecessor at the World Solar Challenge in Australia. It was called the Stella, and it had a roof made out of solar panels. It wasn’t bad, but it wasn’t good either. It looked like a car from the old Soviet Union before the Wall fell. It was Spartan, with a body that was held together with duct-tape and very uncomfortable chairs. It was also way too small for a normal family.
That is not the case with the Lightyear One. Yes, it is expensive, around 119,000 euros (190,000 AUD) and you have to put yourself on a waiting list, because the young company is only able to make 1,500 cars a year for the moment.
But it promises a lot: if you look at it, it is almost all solar panel. The roof, the back, the doors, the hood: five square metres of ‘superefficient panels’, as CEO of Lightyear One, Lex Hoefsloot, said during the presentation. It looks good, is roomy, luxurious and more to the point: it can drive 600 to 800 kilometres on a full battery. And you get that full battery not by charging it at a charging station, but by putting the car outside, where it can charge itself. Most cars do nothing for 90% of the time. If you leave them parked in the sun, that time is, literally, money. The Lightyear uses 10 kilowatts per 100 km and that is half as much as most e-cars that now roam the streets. And if you want to charge it, you can do that at a normal power point, you don’t need an official charging station.
One night of charging like that will produce 400 kilometres worth of travel. Another good thing about Lightyear is that they’ve got dozens of jobs on offer.
Sure, you have to move to Holland. To Helmond, to be precise. But wouldn’t that be a challenge in and of itself? Exciting!