Last summer, a Swedish ten-year old was given a lifesaving transplant. The little girl had a blockage in the vein that connects her intestines and spleen with her liver. This is a rare condition that can stunt development or can even be fatal. And her parents were presented with some very worrying options for their daughter.
First off: a vein implant to replace the blocked one. Doctors usually cut deep into patient’s leg or neck to remove a vein that can replace the defective one, putting the patient at risk of additional traumatic surgery. Another option is to use man-made replacement veins, but these are prone to dangerous clots and blockages and virtually guarantee that the patient will need a lifelong course of drugs to keep their immune system from attacking it. Hope was wavering.
There was a third option, but it would be the first time such an operation had taken place. After lengthy discussions, the parents opted for this pioneering technique. The surgeons undertook an incredibly tricky procedure. They implanted the girl with a vein that had been grown in the lab using her own cells.
Her parents had an agonising wait. First, doctors took about 3.5 inches of vein from the groin of a 30-year-old deceased donor and removed all of the donor’s cells, leaving just the protein scaffolding of the vein. They then took cells from the bone marrow of the girl and seeded the vein scaffolding with them. For two weeks they watched the cells grow before implanting it to replace the faulty vein.
Thankfully, a year after the transplant, the girl had grown nearly 2.5 inches taller, gained 11 pounds in weight and was throwing somersaults. Her body was showing no signs of rejecting the vein. According to Dr Adam Katz, a director of plastic surgery at the University of Florida, this was, “a validation of what we believe will be a revolution in medicine”.
Regenerative medicine can help heart disease, stroke and cancer
This remarkable transplant will soon become routine. By creating a vein made of the girl’s own cells, this procedure overcame the biggest problem with transplants – rejection by the immune system. Another major problem in transplants is the huge waiting lists. Some patients die before they ever reach the operation theatre.
This ten-year-old is only one example of the remarkable feats happening across the globe. In Baltimore, a team has managed to rebuild an ear for a cancer patient using her own tissue inside her own forearm.
Regenerative medicine will be able to help cure heart disease by successfully growing heart valves from human cells. This means heart valves will be repaired without the need to perform surgery. Researchers are also looking into helping stroke patients in the rehabilitation period, aiming to help patients regain motor and neural functions by using cells to rescue brain tissue at or near the site of the stroke.
During cancer treatments to rid the body of cancerous cells, the healthy cells must also be killed off. This leaves the body weak and in need of new cells. These cells must come from a compatible donor, and this is no easy task. What if you had your own stash of cells? Regenerative medicine can be used to try and help tissues lost to trauma, disease and wear and tear.
What exactly is regenerative medicine?
In general, regenerative medicine refers to the creation of living, functional tissues to repair or replace tissue or organ function that has been lost due to age, disease, damage, or congenital defects. It is a quite a different means of treatment from chemical or biologic drugs, surgery or the transplant of foreign organs. The idea of regenerative medicine is that the body can be encouraged to repair itself, and its basic ingredient is the stem cell.
Pluripotent stem cells are capable of turning into almost any cell in the body and can be derived from human embryos or from bone marrow. Otherwise, thanks to the work of Nobel Prize winners, Sir John Gurdon and Shinya Yamanaka, differentiated ‘adult stem cells’ can be reprogrammed back to the pluripotent state.
These stem cells are the building blocks of regenerative medicine, and they can be used in different ways. First, as we have seen with the ten-year-old girl in Sweden, they can be used to grow body parts in the laboratory, using scaffolds. These scaffolds can be derived from humans or animals, while in the future it may be possible to make body parts with the modern technique of 3D printing. A leader here is Organovo (Nasdaq: ONVO).
The body’s own stem cells can be encouraged to differentiate in situ, while it is possible to inject stem cells into damaged parts of the body where they appear to have ‘paracrine’ effects. What this means is that the stem cells appear to react dynamically with the body, stimulating a positive reaction from those cells in the vicinity. In this respect, the stem cells become more like a drug, stimulating a cure and then clearing the body.
Stem cells are making a big impact on medicines
The business of regenerative medicine, a completely new medical discipline, is experiencing inevitable growing pains but progress is being made. Last year, in a milestone for the industry, Prochymal became the first allogeneic stem cell product to be approved in a Western country – Canada.
Based on stem cells taken from the bone marrow of healthy adult donors, this is approved for treating graft-versus-host disease in children (where newly transplanted donor cells attack the recipient’s body). And it is in clinical trials for Crohn’s disease and diabetes. The evidence from trials so far is that stem cells appear to do no harm to patients, while they are genuinely capable of restoring lost bodily functions.
In the coming years, Gil Van Bokkelen, who is Chairman of the Board of Governors of the USA’s National Centre for Regenerative Medicine believes that stem cell therapy will make a difference in cardiovascular disease, stroke, other neurological conditions and a broad range of other areas. “It is already happening”, he says, “and it will have a big impact in the next several years. You will start to see really exciting things coming to the fore”.
The thing is that this is just one of many tremendously exciting areas where biotechnology is breaking new ground and making serious profits. The fact is that billions are being invested right now in a number of key areas. And it is paying off for investors. The Nasdaq Biotechnology index in the US is up by more than 70% (in terms of pounds sterling) over the past two years. That’s more than twice the return on the S&P 500 over the same period.
Bill Gates believes this “will be to the 21st century what computer technology was to the 20th century.” That’s why he has already invested more than $300m into this – with plans to invest more over the next ten years.
The good news is that there are a clutch of really exciting UK-listed stocks that could really surge during the biotech boom. You can read about three of these great stocks in my latest biotech report – click here to read it now.
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