It’s the biggest thing in medicine for decades…
It’s at least as important as Fleming’s discovery of the first antibiotic, penicillin, in 1928 or Köhler and Milstein’s first monoclonal antibody in 1975.
It has relieved golfer Jack Nicklaus from chronic back pain, saved singer Peter Gabriel’s wife from cancer and even persuaded the Pope to address a conference on the subject.
It has even got Wall Street analysts tearing up their spread sheets and big pharma quaking in its boots.
This is nothing short of a medical revolution.
It has hugely exciting implications for human health, and, for the handful of investors who have spotted this wave, it offers mouth-watering profits.
I am talking about Regenerative Medicine, an umbrella term that covers every method of restoring the human body to normal healthy function.
Until now medical treatment has either involved giving you pills that make you feel better while the body is performing its own repairs, patching you up through surgery, or countering the likes of diabetes or haemophilia through regular infusions in a sort of running battle against the enemy.
Despite great progress over the past few decades, none of these approaches do no more than either help you to lead a relatively normal life or stumble along for a few extra years.
Regenerative medicine is nothing like this…
Instead it corrects the underlying problem – for good. It goes right to the source of the problem, makes the necessary repairs and leaves you with a body that is as good as new. It is a once and for all treatment that will last forever.
In 2012 when I spotted the coming opportunity in biotechnology I would never have dared write that sentence.
At that time regenerative medicine sounded like a great idea, but was it really possible? Could we correct problems at source? Would there be nasty side effects? Would these ‘cures’ last forever – and thus justify their sky high cost – or would the patient enjoy only a temporary reprieve?
We are starting to answer these questions. Numerous trials have already been conducted. The Alliance for Regenerative Medicine counts 946 clinical trials that are under way, with 82 in the definitive Stage III. The evidence is mounting up fast and what it shows is highly encouraging.
Regenerative Medicine, which includes Gene and Cell therapy and Tissue Engineering, is safe. It does not seem to have severe side effects.
But most of all, it is effective. And, although the permanence of any cure can only be measured by the passage of further time, many patients who have been treated in these trials are today enjoying consistent respite from their diseases.
Let me give you an example…
At a conference I attended in Oxford, Dr Amit Nathwani described how gene therapy can cure haemophilia, a condition in which the blood fails to coagulate and thus prevent bleeding. Most of us never have to worry about this. If we cut our finger the blood soon forms clots and the bleeding stops.
Haemophiliacs are not so lucky. Two or three times each week they need an injection of a clotting factor. Nobody likes having injections or indeed having to administer them, and the treatment costs the NHS £140,000 per year. But at least haemophiliacs in this country do get this treatment. For the 80% of haemophiliacs who live in the developing world there is no treatment.
Haemophilia is caused by a mutation of the gene responsible for coagulation. That is the root of the problem. Correct this faulty gene and everything else falls neatly into place. And this is precisely what has been done. In 2016 seven men with severe haemophilia A were given a single injection of a virus that carried into their cells the mechanism to correct their mutated gene. The results were stunning.
Six of the seven participants were cured. They no longer needed regular injections and there were no significant adverse reactions. It now remains to be seen how long the effect will last but, given that the problem has been repaired at source and given that similar experiments conducted on monkeys and dogs have recorded an effect of at least ten years, there is every reason to think that these haemophiliacs will be haemophiliacs no longer.
Within a few years every haemophiliac in the developed world could be offered this gene therapy, lifting a huge burden off health services. But it won’t stop there…
Diabetics also need regular injections, in their case of insulin. Could gene therapy relieve them too?
Quite possibly. The results of a research project announced in January described how gene therapy had been used to restore normal insulin levels in laboratory mice. A team from the Pittsburgh School of Medicine engineered a viral vector so that it delivered two proteins to the mouse pancreas. These proteins reprogrammed alpha cells to become insulin-producing beta cells.
There are numerous other targets…
Gene therapy could restore normal heart function and it could provide an answer to the most intractable diseases of all, those affecting the Central Nervous System.
But far and away the biggest target for this new wave of therapies is cancer. We are now well past the phase of considering this a good idea. Trials have been conducted, results have been published, safety and efficacy has been established and the first drugs – so-called CAR-T therapies – have been approved for rare blood cancers.
Cancer is a tough target. Cancer cells are heterogeneous, they change over time and when clumped in solid tumours they have ways of fending off attack by the immune system. So while gene therapy has worked extraordinarily well for some patients it has not helped others and the challenge now is to match the correct drug with specific sub-groups of cancer.
Despite this inconsistency and the high c.$475,000 price of CAR-T treatments this is now being recommended and paid for by health authorities. Even the NHS, which is duly cautious about splashing large sums of cash on new drugs, is joining the party. Only last week NHS England chief executive Simon Stevens indicated that CAR-T will soon be available to patients on the NHS.
To quote another speaker at the Oxford Conference gene and cell therapy ‘has now taken hold.’ It is becoming mainstream and one more crucial piece has been added to the jigsaw. In the years that I have been following this subject I have several times heard it said that none of this scientific advance will really matter until Big Pharma decides to take it seriously.
Several times I have heard that Big Pharma will not take it seriously without the requisite data. Promise and scientific theory is not enough. Before committing billions to this new venture executives want to see validated trial results. These they now have, and the penny has dropped. Big Pharma is buying a seat at the table. Gilead paid $11.9bn for Kite Pharma, Celgene splashed out $9bn for Juno Theraputics and for $8.7bn Novartis is acquiring AveXis, which is working on a gene therapy for spinal muscular atrophy. Other deals are expected to follow.
So what you should be doing about this? As I described here recently, investing in drug trials is not a smart thing to do.
And nor is speculating in companies that might get taken over, although of course you might get lucky.
Nor should you back Big Pharma, for one good reason. Selling cures is no better business than selling shoes that never wear out. The patient will never need to be a customer again. The Big Pharma model has been based upon selling repeat prescription drugs, many of which don’t work and, better still, leave the patient in need of yet more treatment. Regenerative medicine threatens this model.
The key is to identify companies that are benefiting from this new medical revolution without having to get lucky. Some of these stocks have already made gains of over 200% but there is much more to come.
We are only at the start of this story, a story that few people are even aware of… and I’ll be here to show you how to profit as this story unfolds.