The use of stem cells for what looks like magical fixes of damaged or diseased human tissue is becoming big business.
There are more than 700 companies globally with a regenerative medicine focus. Just 2% of them are in Australia but this side of medicine is just getting started.
The global market is valued at $16.4 billion and is expected to grow at 23.2% a year to reach $67.5 billion in 2020, according to analysts at Frost & Sullivan.
Stem cells are now being used to treat wounds which won’t heal and injuries but the potential is enormous, including helping nerves to grow.
In the works, and coming soon after clinical trials are complete and authorities give the okay, are stem cell therapies for the big killers cancer and heart disease.
“Factors that are driving stem cell manufacturing in the short term include ageing populations in need of alternative medicine,” says Rhenu Bhuller, Frost and Sullivan’s senior vice president of healthcare.
Australia is well ahead in creating the infrastructure needed for regenerative medicine research and has had some successes, including the work of scientists IVF pioneer Professor Alan Trounson and prominent stem cell researcher Professor Martin Pera.
The ASX-listed Mesoblast is making strong progress in commercialising stem cell therapies. The Australian biotech is working on treatments for inflammatory ailments, cardiovascular disease and back pain.
And the $153 million Australian Regenerative Medicine Institute, opened at Monash University in 2009, is supplementing the efforts of other research organisations including Stem Cells Australia, the Hudson Institute of Medical Research and the Walter and Eliza Hall Institute.
In October Australian researchers announced a major advancement, successfully growing a functional mini human kidney in a dish.
The breakthrough isn’t the same as growing a replacement organ — yet — but the feat has the potential to save billions of dollars and a lot of time in the testing of drugs for humans.
New drugs often fall over when it comes to human testing. With replica organs, grown in the laboratory, this process can be shortened and a drug proved or disproved before human trials are needed.
The savings can been enormous and will free resources for work on creating more drugs and procedures.
“It’s exciting science,” Melissa Little, the head of the research team, told Business Insider. “It’s just amazing to get such a complex structure in a dish.”
The kidney isn’t a replacement part but the potential to bioengineer a larger organ is there.
“That really is an amazingly hard task,” she says. “A human kidney is larger than a softball … and we have something in a dish which is 6 mm across.”
The process is also a way of advancing very personalised medicine, something which would take enormous computing power to map an individual’s cells and to work out where the problems are and how to fix them.
However, using the same process as growing the kidney, researchers could create one using an individual’s stem cells. This produces a little organ which doctors can use to model a treatments for an individual.
And one day, rejection-free organ transplants could be possible.
Martin Pera, program leader at Stem Cells Australia, a seven year research project funded by the Australian Research Council, is working on understanding what stem cells are and how they decide to give rise to another stem cell.
“It’s really around understanding decisions at a molecular level,” says Professor Pera. “It’s about the basic background on what we need to know to use stem cells effectively. We are always looking at ways we can apply our discovery either in research or down the track in regenerative medicine.”
Over ten years, the science has gone from discovering human embryonic stem cells to using derivatives in clinical trials. These are being used to treat macular degeneration, a very common cause of blindness, spinal cord injury, type one diabetes and, probably soon, Parkinson’s Disease.
“I think it’s pretty remarkable going in a short time from a fundamental discovery to these potential applications in patients,” Pera says.
“Now these are early stage trials and they are just really looking at the safety of the treatment but nevertheless they are big milestone along the way to developing cell therapies for some conditions which are really difficult to treat.”
Similar to the use of the mini kidney’s, stem cells are being used as a platform for basic research, disease modelling and drug discovery.
“We’ve learned to make beating cardiac muscle cells from embryonic stem cells,” Pera says. “Now, with an amazing development which occurred around 2006, we now know how to take an adult cell from a patient with a disease and turn it into something very like an embryonic stem cells … and turn into any cell type. That is enabling us to study the development of disease and to use human cells to develop and screen new medicines.”
Drug development is a very expensive and a big part of that cost is clinical trials and human testing. Millions of dollars can be spent developing a drug only to have it fail at the last post.
“They fail because the test we did on animals in the laboratory didn’t predict what might go wrong,” he says. “Now we have for the first time in the laboratory human cells we can test drugs on and weed out the ineffective compounds early in the development.”