Regenerative Medicine and Stem Cells: Unfulfilled Expectations or True Prospects?
It's a straightforward concept: take a donor's cells, biomaterials, or molecules and pop them into a patient to cure a disease or injury. That's the dream of stem cell therapy. But reality bites hard. Regenerative medicine, the application of cells, biomaterials, and molecules to restore malfunctioning body structures, has the potential to revolutionize healthcare. Unlike conventional drugs, which mainly treat symptoms, regenerative medicine aims to address the root cause of a patient's condition.
Regenerative medicine's tantalizing promise of a new medical revolution casts stem cells and biocompatible materials as the new kings and queens of the medical world. The media and scientific journals have been filled with breathtaking breakthroughs over the years. However, the number of regenerative medicine treatments in everyday practice is underwhelming, and a recent panel of commissioners has criticized this slow progress in a report published in The Lancet.
In fact, apart from a handful of breakthroughs, most treatments are unproven, and private clinics are cashing in on patients' desperate search for cures by offering questionable therapies, according to Prof. Giulio Cossu from the Division of Cell and Matrix Biology & Regenerative Medicine at the University of Manchester in the United Kingdom. So, what's keeping all these promising new therapies from reaching patients? What will it take for society to finally benefit from the immense potential of regenerative medicine?
What is regenerative medicine?
The commissioners explain in their report that regenerative medicine "aims to replace or repair human cells, or regenerate tissue or organs to restore normal function." The emphasis on "normal function" sets this approach to medical treatments apart from many commonly used drugs, which tend to treat symptoms but do not address the underlying causes.
"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 add.
For instance, an individual with type 1 diabetes cannot produce insulin. Instead, they require daily insulin injections to keep their blood sugar levels in check. Regenerative medicine hopes to solve this by regenerating the islets of Langerhans, allowing the individual to naturally produce insulin. While this treatment is still in the experimental stages, there are some areas of regenerative medicine that have made it to medical practice.
Early Successes
Blood transfusions are the earliest form of cell therapy, a commonplace treatment in most clinical settings today. Next on the list is the transplantation of bone marrow, giving patients with radiation damage or blood cancers a chance to make new, healthy blood cells using the donor's bone marrow stem cells. Cell therapy using a patient's own cells is also used in cases of severe burn and scald injuries, where skin cells are isolated from a small biopsy, expanded in a specialized lab, 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)." And, "As well as increasing life expectancy, regenerative medicine therapies could greatly improve the health-related quality of life of many patients with chronic diseases."
So, what's holding back these developments?
From research to medical practice
Researchers around the world are working tirelessly on new regenerative medicine solutions to common diseases and injuries. In the past year alone, we've reported on various breakthroughs, including a chip technology that can change one cell type into another and heal entire organs, a new method of spray painting biomaterials onto damaged hearts using minimally invasive surgery, 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, who grant approval for a new therapy, must be satisfied that the new treatment is safe and works. Regenerative medicine treatments tend to be very expensive due to the need for special production facilities and highly skilled staff. With health budgets continually shrinking in many countries, high costs are a significant barrier to making such therapies a reality.
"Huge benefits might be reaped from regenerative medicine but at huge cost," the commissioners explain, "and affordability might limit implementation, even if there is a good chance of cost savings down the line."
What is clear is that there is a massive demand for regenerative medicine strategies to address common health problems. Both small and large 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 players are profiting from patients' often desperate medical situations.
Are patients being exploited?
In August, FDA commissioner Dr. Scott Gottlieb issued a statement saying, "[...] dishonest actors exploit the sincere hopes 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." As part of the crackdown "to prevent unscrupulous actors from being able to deceive patients and potentially harm their health," the FDA issued a warning to a stem cell clinic in Florida for "marketing stem cell products without FDA approval."
In this particular case, stem cells from fat were isolated and given to patients intravenously or injected directly into the spinal cord for a variety of conditions, despite a lack of scientific or medical evidence supporting this type of treatment. The clinic also failed to adhere to guidelines intended 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 huge sums of money for unproven treatments," the commissioners write. Strict regulation and crackdowns by health authorities on institutions offering unlicensed products are crucial to protecting patients.
What does the future hold?
Regenerative medicine offers exciting prospects, but a study breakthrough does not mean a new therapy, which often leads to a disconnect between public expectations and the speed at which new treatments can be developed. However, regenerative medicine does have a track record of success in a small number of diseases. Prof. Giulio Cossu is optimistic about the future, saying, "From the first blood transfusion to bone marrow transplantation, cloning, development of viral vectors, ES [embryonic stem cells] and, more recently, iPS [induced pluripotent stem] cells, genome editing and organoids hold great promise for the future."
To move regenerative medicine into mainstream medicine, better science and better regulation must be partnered with innovative manufacturing methods that make treatments affordable and a way to show how they ultimately benefit the patient and society as a whole. 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 aims to restore normal function by replacing or repairing human cells, or regenerating tissue or organs, setting it apart from conventional drugs that mainly treat symptoms.
- In the field of regenerative medicine, an individual with type 1 diabetes could potentially have the islets of Langerhans regenerated, allowing them to naturally produce insulin.
- Despite some early successes such as blood transfusions and bone marrow transplantation, regenerative medicine treatments are yet to enter mainstream medical practice in most areas of medicine.
- The development of regenerative medicine treatments faces hurdles, primarily due to the high costs involved, stringent safety regulations, and the need for affordable and innovative manufacturing methods to make these treatments accessible to all.
