Regenerative medicine and stem cells: Delivering on hopes or just overhyped prospects?
The simplistic principle behind stem cell therapy sounds promising: harness the powers of cells, materials, or molecules from a donor and use them to fix or replace damaged parts in a patient. However, reality falls far short of this dream.
Embracing the domain of regenerative medicine, we find cells, biomaterials, and molecules being employed to mend the body's structures harmed by disease or injury. This approach diverges significantly from traditional drugs, which primarily address symptoms, not root causes.
Capping on the fascinating charm of regenerative medicine, its potential to redefine medical treatments demands our attention, with stem cells and biocompatible materials taking center stage in this revolution. Over the years, numerous breakthroughs have been reported, capturing the spotlight in scientific journals and the media alike.
Unfortunately, the number of regenerative medicine treatments currently in use is dishearteningly low, a fact lambasted by a panel of commissioners in a report published recently in The Lancet.
According to Professor Giulio Cossu, from the Division of Cell and Matrix Biology & Regenerative Medicine at the University of Manchester in the United Kingdom, and his fellow commissioners, only a handful of breakthroughs have been sanctioned for clinical use, with private clinics cashing in on patients' desperation for treatments by offering unproven therapies.
The question lingers: why has the excitement surrounding new therapies failed to materialize? What does it take for society to reap the immense potential that regenerative medicine promises?
Understanding regenerative medicine
The commissioners define regenerative medicine as an approach to restoring "normal function" by replacing or repairing human cells, tissues, or organs. The focus on "normal function" marks this method of treatment from many commonly used drugs, which primarily treat symptoms without addressing underlying causes.
"Cell therapies and regenerative medicine, with their potential to enhance the health of patients, represent a paradigm shift in healthcare by focusing on the root causes of diseases through repair, replacement, or regeneration of damaged cells in the body," the authors explain.
Consider, for example, an individual with type 1 diabetes, who cannot produce insulin. With regenerative medicine, the islets of Langerhans in the pancreas could be regenerated, allowing the individual to produce insulin naturally. This would mean no more insulin injections and a return to normal sugar metabolism.
While this treatment for type 1 diabetes is not yet feasible, there are some successes in other areas of regenerative medicine.
Triumphs in regenerative medicine
Cell transfusion, a precursor to modern cell therapy, has become commonplace in clinical settings. Following this, bone marrow transplants were employed to help patients with radiation damage or blood cancers make new, healthy blood cells using the donor's bone marrow stem cells.
In cases of severe burn and scald injuries, where a patient lacks sufficient undamaged skin for skin graft treatment, skin cells are extracted from a small biopsy, expanded in a specialized lab, and transplanted onto burn wounds to speed up healing.
Despite these accomplishments, and an international community of scientists tirelessly developing new therapies, regenerative medicine treatments have yet to enter mainstream medical practice in most areas.
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)."
"As well as increasing life expectancy, regenerative medicine therapies could greatly improve the health-related quality of life of many patients with chronic diseases."
But what stands in the way of these developments?
From lab to bedside
The road from successful research to medical practice is arduous, due in part to rigorous health authority requirements such as those set by the FDA in the United States, which must be satisfied that a new treatment is safe and effective before granting approval.
Regenerative medicine treatments tend to be expensive due to the need for specialized production facilities and expert personnel. With tightened health budgets in many countries, steep costs pose a significant barrier to making these therapies accessible.
"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."
Yet, both small and large players in the pharmaceutical and healthcare industries continue investing in the development of new therapies.
Battling exploitation
In August, FDA commissioner Dr. Scott Gottlieb issued a warning stating, "[...] 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."
As part of a 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 instance, fat-derived stem cells were extracted and administered to patients intravenously or injected directly into the spinal cord for a variety of conditions, despite a lack of scientific or medical evidence to support such treatments.
The clinic was also found guilty of disregarding guidelines that aim to prevent microbial contamination during stem cell processing, placing patients at risk of receiving contaminated cells.
"So the question remains about what to do about desperate patients paying exorbitant sums of money for unproven treatments," the commissioners write.
Strict regulation and FDA crackdowns on unlicensed institutions offering such treatments are crucial to keeping patients safe.
The future beckons
Exciting advances in stem cell and regenerative medicine research are heralded as breakthroughs. However, a breakthrough in the lab does not always equate to a new therapy, leading to a discord between public expectation and the speed at which new treatments can be developed.
Regenerative medicine does have a successful track record in rare diseases.
"More complex diseases such as diabetes or heart infarct will require more advanced approaches to see a significant clinical impact," Professor Cossu states.
Despite these challenges, Professor Cossu emphasizes the enormous potential that lies within regenerative medicine. From the first blood transfusion to bone marrow transplantation, cloning, development of viral vectors, ES and iPS cells, genome editing, and organoids - the future is brimming with possibilities.
To propel regenerative medicine into mainstream medicine, we must embrace better science, better regulation, innovative manufacturing methods that make treatments affordable, and a way to demonstrate the benefits of these treatments for both patients and society as a whole.
- Regenerative medicine, an approach that focuses on replacing or repairing damaged human cells, tissues, or organs, represents a new paradigm in healthcare, addressing the root causes of diseases instead of merely treating symptoms.
- The successes in regenerative medicine include cell transfusion, bone marrow transplants, and the use of expanded skin cells to treat severe burn injuries, speeding up the healing process.
- However, despite these accomplishments, regenerative medicine treatments have yet to enter mainstream medical practice in most areas, due to rigorous health authority requirements, high costs, and the need for affordable manufacturing methods.
- The FDA has issued warnings against unscrupulous actors exploiting the potential of regenerative medicine, advertising unproven treatments and disregarding guidelines aimed at preventing microbial contamination during stem cell processing.
- To accelerate the adoption of regenerative medicine, we need a concerted effort towards better science, innovative manufacturing methods that make treatments affordable, stricter regulation to ensure patient safety, and clear demonstration of the benefits of these treatments for both patients and society as a whole.
- The enormous potential of regenerative medicine, from blood transfusion to advanced genome editing, offers a bright future for treatment of medical conditions, from rare diseases to complex ones like diabetes and heart infarct.
