medical leap

From cystic fibrosis to diabetes to strep — every medical find requires refinement and improvement over time.

There is a video floating around out there, of an ex Royal Marine being treated for previously untreatable Parkinson’s Disease. I also quite like the ones of deaf people getting cochlear implants switched on for the first time.

Every year, there are more and more biotech advances — and AI could drive improvements even faster.

Medical Advances

A child born with cystic fibrosis in the 1950s could expect to live until age 5 — but those who take a treatment called Trikafta, from adolescence, can now expect to live to 82.5 years old. This is effectively a normal life span.

Trikafta is a triple combination therapy designed for the most common CF mutation, F508del, and developed by Vertex Pharma. The same company is now attempting to develop a next-gen treatment comprised of a once-daily triple CFTR modulator regimen made up of vanzacaftor, tezacaftor and deutivacaftor.

Trikafta did not magically come into existence. It was the process of decades of advancement in medical technology, and this is one of the key things to understand when it comes to early stage biotechs. Trials may be new, but they are designed on the countless failures and incremental steps that came before them.

Yes, most fail. Excluding the collapses due to financial mismanagement, PR blunders, fraud, and incompetence…only 10% of Phase 1 clinical trial candidates get commercialized. And of this 10%, the vast majority never become blockbuster drugs (instead providing a very minor improvement on the previous standard of care). This means even when successful, many drugs do not provide significant financial returns to investors.

Of course, the ones which do can deliver truly impressive sums of money. And while a clinical trial failure can be expensive, in science, a failure is just crossing out one of the possibilities before succeeding. Eventually, the trickle of successes translates into serious medical progress; and while not everyone invested along the entire journey benefits financially, it is worth highlighting that for many biotech investors, there is at least an element of altruism.

medical leap

Yes, sometimes there is one great leap forward. On 11 January 1922, scientists walked into a hospital ward of dying children, all of whom were in comatose diabetic ketoacidosis — essentially hours or at most days away from certain death.

The scientists then started injecting a novel new drug ‘insulin’ into the children. Witnesses described families as already grieving the deaths of their children before they had even happened. But before the final patient had been injected, the first had woken up.

It’s hard to describe the level of human impact this would have had at the time. One of the patients was 13-year-old Leonard Thompson — who suffered only from an abscess at the injection site and a reduction in blood sugar. The child enjoyed the benefit of daily injections of a refined extract from 23 January and lived for 13 more years before dying of pneumonia.

Of the three inventors of this miracle drug, Frederick Banting refused to put his name on the patent — while James Collip and Charles Best sold the patent to the University of Toronto for one solitary dollar.

I mean, money was worth more 100 years ago, but I do like to think the pair spent the cash on a couple of pints and took a week off at least.

This was still just a first step in reality.

The treatment originally required 25 tons of pig parts to generate enough insulin to last a typical patient one year. There wouldn’t be anywhere near enough pigs in the world to manufacture enough insulin for all of today’s diabetics — so further advances were of course needed, and these were expensive.

Now you have the problem of deciding how much insulin should cost. I am not about to go into the ethics of it, this is not my thing. However, it is interesting to note that Novo Nordisk cut the price of its insulin offerings in the US by up to 75% last year, alongside other competitors due to major changes to the Medicaid program.

Then it decided that key drug Levemir — cut by 65% — would stop being sold at the end of 2024, with the alternative now costing three times as much.

An hour in the life

Anyway, there is a point here.

I spent the past week or so — along with my partner and three young sons — ill. We were infected with bacterial Strep A combined with at least one other viral component, and because the symptoms for Strep A and viruses are so similar, it took five days of symptoms being present before we were correctly diagnosed and given antibiotics.

Because if you have a virus, unless you are in a life-or-death medical emergency, there is pretty much nothing the health service will do except let it run its course. That’s just the way it is.

But let’s just picture the scene if you will: it’s 4am on the third day of the illness. You wake up to screaming. It’s the three-year-old, who no doubt has thrown up for the fourth time that night. There are no more clean clothes. Or bedclothes. Your muscles are sore beyond measure, and you haven’t slept more than 30 minutes in a row in more than 72 hours.

The house smells like rotten cats. The cats themselves have chosen to remain outside. You don’t blame them.

You glance to your loving partner, briefly hoping she might decide to take this turn. She only took the last four.

Oh no.

She’s holding the five-year-old. He’s in the bed. He’s currently vomiting. She’s vomiting. Then the three-year-old screams again. And again. Except his voice is gone, so it’s less Nazgul and more Balrog.

Okay. Swing a leg out. Stand up. Legs buckle. Sweat drips. You can do this. Walk around the bed, head to the toddler’s room — then you step in it. What is it? You can guess.

Grab the nearest article of clothing. Throw it on the floor. Rub your foot on the clothing. Go into the three-year-old’s room. He’s vomited. His eyes are sunken and red. You catch a glimpse of yourself in the mirror — and wonder briefly if you’re going to scare your own son. Thankfully, he just wants to be held. But he’s covered in vomit.

Then the ten-year-old walks out. He’s covered in vomit. He’s not crying, but he’s trying bloody hard not to. Decision. Bath time. Ten-year-old in the shower, 90 second time limit Vietnam vet-style. You don’t care what the temperature is. Out, go away. Fend for yourself – come to us when you need Calpol. Good luck, sorry.

We have a parental resource issue and 100 years ago you would have been old enough to get a job down the mine. You’re on your own and we now live in a war zone.

Okay, clean down the bath. Run a new one. The water is barely warm, but apparently might have been fetched directly from Yacumama’s boiling river. Children cry more when they go in. Meanwhile, you can hear your other half in the three-year-old’s room, trying to do some kind of 5D mathematics to force two of your finest suit shirts to make up one fitted sheet.

Okay unplug the bath. Pick up both children, and then promptly drop them again. Look at your arms. You can’t lift them both at the same time anymore. You try again. Nope, not happening. One more time — and feeling like Hercules and most likely looking like Pan, you stagger into the three-year-old’s room.

Throw the youngest into clothes. It doesn’t matter which ones. They’ll be covered in vomit in an hour or so anyway. Sing him to sleep. He needs three choruses of Doe-a-bloody-deer every time. How fast can you sing in a lost voice? Put him down, instantly asleep. You walk out of the room.

The ten-year-old is outside the door, with bleach and a scrubber brush, cleaning the mess you stepped in. You notice the clothing you covered it with was his favourite trousers. Make a mental note to buy him a new mountain bike when the horror show ends. Or a medal of gallantry off eBay.

The five-year-old doesn’t want to sleep. He’s scared. He needs Daddy to sleep in the room next to him. It’s fair enough by this point. The problem is there’s not much space. Daddy can fit in, but the five-year-old needs to remember to use the bucket when he wakes up or Daddy will again be covered in vomit.

‘Can you remember to use the bucket? Promise?’

‘I promise’ comes the swift reply with the first smile in days.  

45 minutes later. You wake to a damp feeling and a smell. The three-year-old is crying. The five-year-old is crying and repeatedly saying sorry. The ten-year-old opens the door, bucket in hand and with an expression resembling a WW1 soldier sometime circa January 1915.

That was just one hour in my past week of hell — and all parents go through something similar eventually.

Of course, the solution was antibiotics (thanks Alexander). But when you consider how the difference between abject misery and total recovery now resides within medication which is as readily available as Tic-Tacs, it makes you really think about how investing in biotechs is not just an exercise in trying to make money.

It really is about developing medical advances. And if one, two or several happen to fail, you can write off the loss against more sensible bets.

Because however bad this one week has been, it’s science that made it temporary.

This article has been prepared for information purposes only by Charles Archer. It does not constitute advice, and no party accepts any liability for either accuracy or for investing decisions made using the information provided.

Further, it is not intended for distribution to, or use by, any person in any country or jurisdiction where such distribution or use would be contrary to local law or regulation.

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