This is what becomes of the broken-hearted

Before we get into today’s note – a quick reminder.

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In the meantime, onto today’s letter…

We’ve all seen nature documentaries where a lizard loses and later regrows its tail.

Still, to this day, when I watch them I don’t get why people can’t just regrow their body parts after damage.

I mean, I get why they can’t – that’s not how human biology works. But I don’t get why we haven’t discovered a method for doing it yet.

If we could simply regrow our damaged body parts and then transplant them, it would solve so many health problems. From knee and hip injuries to respiratory problems and heart disease.

Alligators can regrow their teeth up to 50 times. They have stem cells, then a baby tooth then an adult tooth on top. If they lose their adult tooth, the baby tooth grows into an adult tooth and the stem cells grow into a baby tooth.

Back in 2013, scientists thought we might be able to adapt this alligator method into an injection for humans. Lose your tooth or get tooth decay? Just get a stem cell injection and regrow it.

It turned out to be a little more complicated than that. But, almost five years later, it seems the stem cell injection method is almost ready to market.

From a King’s College press release in November:

“Early in the year we showed that we can stimulate natural tooth repair by activating resident tooth stem cells. This approach, which is simple and cost effective, was used in teeth that had the vital layer (dental pulp) of the tooth exposed to the outer environment,” explains Professor Sharpe Paul Sharpe, Director of the Centre of Craniofacial & Regenerative Biology at King’s College London.

“Now we have demonstrated that this same approach can be used in situations of shallow dentine damage where differentiated cells (not stem cells) required activation. This type of damage to the tooth is the most common case that dentists see in the dental practice.”

Regrowing teeth is great. And I welcome the day when I can get new teeth with a simple injection. But the real body parts we want to be able to regrow are the life-supporting ones.

Heart disease is the leading cause of death worldwide. If we could regrow our broken hearts, it would be such a game changer it would mark a new era in humanity.

You can probably see where I’m going with this now can’t you?

Reprogrammed skin cells are being used to regrow broken hearts in Japan

In a first-of-its-kind trial, Japan has given the go-ahead for doctors to treat heart disease patients with reprogrammed stem cells.

This is a true breakthrough. And I feel like it’s more than your average headline-grabbing biotech story for a number or reasons.

Firstly, these stem cells are induced pluripotent stem cells (iPS). What that means is they are normal cells – like skin or blood cells – that have been regressed to an embryotic state. These cells can then be reprogrammed to form any other type of cell.

So, in this case, you can take skin cells from the patient and turn them into healthy heart cells. The patient provides the cells themselves. There is no need to seek out foetuses or donors. So no ethical problems.

Secondly, because the cells come from the patient’s own body, there is no risk of rejection. And no need for lifelong courses of anti-rejection drugs.

In this case, a thin sheet of heart cells is grown and grafted on to the patient’s heart. This graft then causes the damaged heard to regenerate healthy tissue.

Thirdly, this graft doesn’t need a scaffolding to fix it to the heart. So there is no foreign material for the patient’s immune system to deal with.

Japan’s accelerated system is the way forward

And finally, the trial is ready to be rolled out on a large scale if it proves successful.

Unlike in many parts of the world, Japan has decided that treatments like this could be so life changing to so many people that it is willing to truly fast-track them.

As Nature reports:

If the treatment proves to be safe, and shows some signs of working, it can be approved for sale under the accelerated system. This allows researchers to bypass expensive large-scale clinical trials aimed at proving efficacy, and instead to use small pilot trials to show that the therapy is safe and demonstrates an indication of efficacy.

So basically, if it’s proven to work on a relatively small scale, it can be rolled out commercially almost straight away.

Other commentators have been quick to point out Japan’s method has “insufficient oversight”. From that same Nature article:

Some researchers say the bar for approving therapies for commercial use is too low. Even if the cells are found to be safe, there are risks associated with any surgery, and patients could give up other therapies for a treatment that might not work. Ethicists and regulators say that the benefits of any new therapy must outweigh the risks.

However, I think this way of doing things is much more ethical. At least for a treatment like this.

If I was dying of heart disease – or any other condition for that matter – I would want the chance to try a promising treatment like this as soon as it was showing promise. I would want to be given that choice.

The alternative of waiting 5-10 years or more for huge trials to take place before I was even given the option of trying it seems much more unethical to me.

And what final point is why I think this is a standout breakthrough, and much more than your usual biotech headline-grabber.

It’s not going to be decades before it might become available. It’s all happening now.

What do you think about this new treatment, and Japan’s accelerated programme? Let me know: harry@southbankresearch.com.

Until next time,

Harry Hamburg
Editor, Exponential Investor

Category: Genetics and Biotechnology

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