Regenerative medicine and stem cells: Delivering on expectations or overhyped prospects?
Hear me out, buddy! Stem cell therapy ain't as easy as it sounds. This fancy schmancy medical approach aims to swap or fix screwed-up cells, or regrow tissue and organs to restore normal bodily function. That's a big freakin' difference from most traditional drugs that merely treat symptoms.
Now, guess what? Regenerative medicine has the potential to totally transform the medical field, making stem cells and biocompatible materials the hallmark of this revolution. But hold onto your socks – the number of regenerative treatments smack dab in the middle of medical practice is disappointingly low, according to a panel of badass commissioners who recently spilled the tea in The Lancet.
You know Prof. Giulio Cossu from the Division of Cell and Matrix Biology & Regenerative Medicine at the University of Manchester, right? Well, he and his crew think that only a handful of breakthroughs have made it to patients so far, and private clinics are capitalizing on people's desperation for treatments by offering bogus therapies that ain't been proven yet.
So, why in the heck are so many promising new therapies falling flat? And what needs to happen for society to start reaping the insane potential of regenerative medicine?
What Freakin' is Regenerative Medicine?
According to the commissioners, regenerative medicine is all about repairing or replacing human cells, or regenerating tissue or organs to restore normal function. That focus on "normal function" sets this approach to medical treatments apart from many commonly used drugs, which tend to treat symptoms but not the underlying issues.
"Cell therapies and regenerative medicine, with their potential to improve the health of patients, represent a structural shift in healthcare by focusing on the underlying causes of disease by repairing, replacing, or regenerating damaged cells in the body," the authors explain.
Now, imagine a person with type 1 diabetes who can't produce insulin. Instead of daily insulin injections, regenerative medicine aims to regenerate the islets of Langerhans, allowing the individual to make insulin and say so long to shots. While we ain't quite there yet, there are some areas of regenerative medicine that are well established in medical practice.
Early Succeesses
Transfusing blood is commonplace in most clinical settings these days, and the transplantation of bone marrow gave patients with radiation damage or blood cancers a chance to make new, healthy blood cells using donor bone marrow stem cells.
When a patient suffers severe burn or scald injuries and lacks enough undamaged skin for skin graft treatment, skin cells are isolated from a small biopsy, expanded in a specialized laboratory, and transplanted onto the burn wound to speed up healing.
But despite these successes, regenerative medicine treatments haven't entered mainstream medical practice in most areas of medicine. According to the report in The Lancet, "the potential exists to substantially reduce the burden of disease for some common conditions (e.g., stroke, heart disease, progressive neurological conditions, autoimmune diseases, and trauma)."
So, what the heck is holding back these developments?
From Research to Medical Practice
An army of scientists worldwide is working on new regenerative medicine solutions to common diseases and injuries. In the past year alone, Medical News Today reported on some wicked cool advancements in stem cell and regenerative medicine research – like a chip technology that can change one cell type into another, and a growth factor that might reverse osteoporosis.
Yet the number of approved cellular and gene therapy products on the FDA website is surprisingly short: it has only 15 entries. According to the authors of the report published in The Lancet:
"Cell therapy has produced clinically extraordinary results, having saved hundreds of thousands of lives [...] However, many cell therapies have had limited, variable, or transient efficacy."
The road from successful research to medical practice is long because health authorities like the FDA must be confident that a new treatment is safe and efficacious.
Regenerative medicine treatments tend to be expensive because they often need special production facilities and highly skilled staff. With health budgets stretched thin in many countries, high costs are a barrier to making such therapies a reality.
"While the market grows over the next few decades," the report's authors explain, "thinking of ways that regenerative medicine products can be made more affordable and cost-effective will be useful so that patients can benefit."
What's clear is that there's an enormous demand for regenerative medicine strategies to address common health problems, and that both small and big players in the pharmaceutical and healthcare industries are investing in the development of new therapies.
Yet, the report's authors heavily criticize the way that some bad apples are profiting from patients' often desperate medical situations. In August, FDA commissioner Dr. Scott Gottlieb issued a statement saying, "[...] dishonest actors exploit the sincere reports of the significant clinical potential of properly developed products as a way of deceiving patients and preying on the optimism of patients facing bad illnesses."
In this particular case, stem cells from fat were isolated and given to patients intravenously or injected directly into the spinal cord for various conditions, despite a complete absence of scientific or medical evidence to support this type of treatment.
The clinic was also found to have failed to adhere to guidelines that intend to prevent microbial contamination when processing the stem cells, leaving patients at risk of being treated with contaminated cells.
"So the question remains about what to do about desperate patients paying big bucks for unproven treatments," the commissioners write.
Strict regulation and crackdowns by health authorities on institutions that offer unlicensed products will be essential in keeping patients safe.
What's Next?
Scientific advances in stem cell and regenerative medicine research are celebrated as breakthroughs. But a study breakthrough doesn't guarantee a new therapy, which often leads to tension between public expectation and the speed at which new treatments can be developed.
However, regenerative medicine does have a track record of success, albeit in a very small number of diseases. It's important to remember the huge potential that regenerative medicine has. From the first blood transfusion to bone marrow transplantation, cloning, development of viral vectors, ES and, more recently, iPS cells, genome editing, and organoids, regenerative medicine holds great promise for the future.
But, to move regenerative medicine into the realms of mainstream medicine, we need to step up our game. Better science, better regulation, innovative manufacturing methods that make treatments affordable, and showing the ultimate benefits regenerative medicine has for patients and society as a whole need to be our top priorities.
The commissioners conclude that "exploration is essential for companies and academics to move the field forward, balancing risks, costs, and potential benefits as much as possible."
"How we proceed in this new global terrain might be the biggest challenge of all for researchers, doctors, patients, relatives, regulators, and society as a whole."
- Regenerative medicine, with its focus on repairing or replacing damaged cells and regenerating tissue or organs, aims to restore normal function and address the underlying causes of diseases.
- Stem cells, particularly bone marrow stem cells, have been instrumental in treatments such as blood transfusion and bone marrow transplantation, allowing patients with radiation damage or blood cancers to generate new, healthy blood cells.
- Despite these successful applications, many regenerative medicine treatments are yet to enter mainstream medical practice, and only a handful of promising new therapies have been made accessible to patients so far.
- The potential of regenerative medicine to reduce the burden of common medical conditions like stroke, heart disease, and autoimmune diseases is promising, but the road from successful research to medical practice is long due to the need for health authorities to ensure the safety and efficacy of new treatments, and high costs associated with producing these therapies.
