Modern medicine, for all of its sophisticated drugs, complex gadgets, and amazing surgical procedures, rarely cures anything. It treats. It manages. It postpones the inevitable.
But return a patient to normal, optimal health?
Seldom…
So when an innovation comes along that can effect a complete and permanent remission of disease or restore damaged organs to a pristine state, it should cause your keenest investing instincts to perk up and pay attention…
A $1.8 Billion Start That's Headed Straight Up
That innovation is here, in the form of a revolutionary approach to curing disease and healing injury, and it's taking the research establishment by storm.
Biotech and pharmaceutical companies around the world are using it to create whole new classes of drugs and diagnostic tools that actually promise to cure heretofore incurable diseases – and create unimaginable wealth in the process.
It's a total game changer. And we can profit from it.
The idea is simply this: with current technologies, it's possible to design drugs, such as cell therapy products, that can be altered or optimally combined to fit the genetic and morphological profile of each individual patient, often recruiting his or her own immune system into the fight.
It's the opposite of the traditional one-drug-fits-all approach, and it's called personalized or regenerative medicine.
Instead of the old model, it treats every sick individual as a unique instance of patient meets pathogen.
It holds the promise of miraculous treatments for severe spinal injuries, joint and bone repair, and organ replacement, and may provide real-life "magic bullets" for diseases like cancer, multiple sclerosis, lupus, and inflammatory bowel disease (Crohn's and ulcerative colitis).
This is not science fiction or fanciful speculation.
In fact, some of these new, experimental medications have already proven completely curative in early trials against some cancers. And according to the Alliance of Regenerative and Personalized Medicine (ARM), current cell therapy products alone generated over $900 million in revenue with 160,000 patients receiving treatments.
If you add in other types of regenerative medicine products, that number doubles.
There are currently more than 2,500 clinical trials underway that use personalized medicine. About 400 of them are taking place in the labs of biotech and pharmaceutical companies.
And that's just the start.
Personalized Medicine in Action
Personalized medicine generally falls into three categories:
- Rejuvenation. This is not about taking years off of your appearance. Instead, it means enhancing the body's ability to heal itself by repairing and replacing damaged tissue. You see this process at work when you have a skin cut or a broken bone. But researchers are currently finding ways to exploit and boost the body's ability to do the same thing with heart, lung, and nerve tissue.
- Replacement. This means replacing unhealthy tissue with healthy tissue – from a donor or the patient themselves. This includes organ transplants, which have been going on for a long time, but researchers are currently enhancing the science by investigating new ways to overcome organ rejection by the immune system.
- Regeneration. In this type of personalized medicine, potent stem cells or bioengineered cell products are delivered to diseased tissues or organs, where they can rebuild tissue and restore function. Often, a patient's own cells are removed, re-engineered to do a specific job, and returned to the patient.
Certain illnesses have proven stubbornly resistant to medications designed to keep them under long-term control. That's because they're amazingly complex and actually differ in pathology – the way they function – from person to person.
For example, IBD (Crohn's disease and ulcerative colitis) depend not only upon a patient's particular genetic make-up, but also upon the local environment in the gut – what's called the microbiome, or the sum total of organisms that live in the intestines – and how these two factors interplay with each other.
To treat an individual, it may well be necessary to understand how the various bacterial, viral, and fungal populations in his or her lower digestive tract are distributed, how they interact with various types of cells in the intestinal lining, and how they affect vulnerable locations in the genome.
New medications may be crucial to altering those populations, cells, or genome loci in very particular, individualized ways.
And certainly, new diagnostic tools will be essential to determining which medications are needed, in what combination, and at which doses.
Immunotherapy is one of many approaches to personalized medicine that has recently shown particular promise.
At the University of Pennsylvania, researchers are collecting the blood of leukemia patients and reengineering the genomes of their T-cells – powerful attack dogs of the human immune system – to recognize and hunt down cancer cells.
They then re-introduce the supercharged blood back into the patient from which it was taken, sometimes with astonishingly effective results. This technique is called CART or CTL019, and the FDA has designated it as breakthrough therapy, which will help speed it through the pipeline and into clinical use.
The only question left for us is how to take advantage of the opportunity in this exciting new space…
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About the Author
Ernie Tremblay has more than 25 years of experience in following and analyzing the latest developments in health, medicine, and related technologies. He understands the FDA approval process, as well as the "hard science" behind new, experimental drugs and the market demand for them - and has a comprehensive grasp of the complex dynamics that determine whether a new drug will be a breakthrough winner, or just another casualty of the FDA approval process.
Mr. Tremblay, This research sounds very interesting but as a pediatrician I would have to ask if Organovo Holdings, Co. is using embryonic stem cells for their research or adult stem cells? In other words, what are "functional human tissues?" Can you answer that question for me? Thanks, Jim Pearson
Jim, let me look into this for you. It's a good question.
Dr. Pearson, I have a query into Organovo about the use of embryonic stem cells in their 3-D bioprinting ink, but I haven't received a reply. However, what I can say more generally is that bioprinting seems to be moving toward the use of cells harvested from the adult patient, him/herself, as there is no rejection problem with them.
why is xoma on the downturn got sold out today should i buy back
John, unfortunately I'm constrained by law from giving individual investment advice, so I can't really say whether you should or should not get back into your XOMA position.
Your reader asked a good question. With the recent downturn of XOMA, should he buy more, sell or what? Did something change with the FDA approval process? If you cannot advise when to get into and out of a position, as many other services provide, what good is your investment advice?
Dr. McShane,
Thanks for reading Money Morning. We do provide the kind of advice you describe in our paid subscription services, but not in our free articles.