Regenerative medicine and stem cells: Unfulfilled hopes or genuine possibilities?
The dream of stem cell therapy, where cells from a donor are used to treat diseases or injuries, seems simple, but the reality is not so rosy. Regenerative medicine, which uses cells, biomaterials, and molecules to repair damaged structures in the body, has proven potential, but its use in mainstream medicine lags far behind.
Despite numerous breakthroughs, the number of regenerative medicine treatments in clinical practice is abysmally low. Experts, including Prof. Giulio Cossu from the Division of Cell and Matrix Biology & Regenerative Medicine at the University of Manchester, have criticized this state of affairs in a recent report published in The Lancet. They claim that only a few treatments have made it to the public, with many private clinics exploiting patients' desperation by offering unproven therapies.
So, why hasn't regenerative medicine lived up to its promise? And what needs to be done for society to reap its immense potential?
Regenerative medicine offers a structural shift in healthcare by focusing on the root cause of diseases, unlike common drugs that merely manage symptoms. For instance, a person with type 1 diabetes cannot produce insulin, requiring daily injections. Regenerative medicine aims to regenerate the islets of Langerhans, which would eliminate the need for insulin injections and restore normal sugar metabolism.
While these treatments aren't yet a reality, well-established regenerative medicine applications do exist. Blood transfusions and bone marrow transplants for radiation damage or blood cancers are common clinical practices today. There's also cell therapy using a patient's own cells in cases of severe burn injuries, where skin cells are isolated and grown in a lab before being transplanted onto the burn wound to accelerate healing.
However, most regenerative medicine treatments are yet to enter mainstream medical practice, despite scientists racing to develop new therapies worldwide. The Food and Drug Administration (FDA) website features a surprisingly short list of approved cellular and gene therapy products—only 15 entries.
Several factors contribute to this issue. Health authorities impose stringent safety and efficacy standards, which are challenging for these novel therapies. The slow-paced approval process adds to costs, making regenerative medicine treatments out of reach for many patients.
"Huge benefits might be reaped from regenerative medicine, but at huge cost," the commissioners explain. Disappointedly, affordability might limit implementation, even if costs eventually drop.
Governments must consider ways to make these therapies affordable for all, especially as demand for regenerative medicine strategies to address common health problems continues to grow. Yet, the report's authors criticize the way some players profit from patients' desperate medical situations. The FDA regularly cracks down on institutions offering unlicensed products, as evidenced by the recent case of a Florida stem cell clinic.
While scientific advancements in stem cell and regenerative medicine offer hope, they also highlight the conflict between public expectations and the speed at which new treatments can be developed. Regenerative medicine comes with immense potential, from blood transfusions to genome editing and organoids. Prof. Cossu believes that "the biggest challenge of all" will be for researchers, doctors, patients, regulators, and society as a whole to navigate this brave new world.
Sources:1. Cell & Gene Therapy Strategic Review 20182. Cell and gene therapy: Closing the translational gap3. Global Markets for Gene Therapies4. Scaling up gene therapy manufacturing5. Regenerative Medicine: An Overview of Current Barriers and Future Prospects
- The slow adoption of regenerative medicine in mainstream medical practice can be attributed to stringent safety and efficacy standards imposed by health authorities, which are challenging for novel therapies like stem cell transplants.
- Despite the potential of regenerative medicine to address medical-conditions such as type 1 diabetes through cell regeneration and eliminate the need for daily insulin injections, most regenerative medicine treatments have yet to enter patient care, with only a few approved products listed on the FDA website.
- The high costs associated with well-established regenerative medicine applications like blood transfusions and bone marrow transplants, as well as the slow-paced approval process, make these therapies out of reach for many patients and raise concerns about affordability and accessibility in health-and-wellness.
- Navigation through the complexities of stem cell and regenerative medicine requires collaboration between researchers, doctors, patients, regulators, and society as a whole, as the potential of these therapies in treatments and therapies-and-treatments is immense, from blood transfusions to genome editing and organoids.
