The Promise and Perils of Stem Cell Therapies
Stem cell therapies are among the most exciting medical advancements of the 21st century. Harnessing the power of stem cells — the basic building blocks of the human body — may unlock treatments that slow or reverse the effects of devastating diseases like amyotrophic lateral sclerosis (ALS) or repair brain function in people with traumatic brain injuries, among many other uses. However, the promise of stem cells has also been co-opted by unscrupulous clinics in the United States and around the globe that are offering unproven, even fraudulent, therapies.
“People read about stem cells in the news and presume that therapies must already be available,” says Brian Cummings, PhD, professor and vice-chair for research in physical medicine and rehabilitation at the UCI School of Medicine and the UCI Sue & Bill Gross Stem Cell Research Center (SCRC). “They find a clinic and go without knowing they’re being taken advantage of.”
Separating Fact from Fiction
So far, the U.S. Food & Drug Administration (FDA) has only vetted and approved eight stem cell therapies as effective and safe — and only for the treatment of blood malignancies, says Cummings, who received a $4.8-million CIRM grant to address traumatic brain injury in July. Clinics advertise treatment for Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, diabetes and cancer, among other conditions, for hefty prices that aren’t covered by insurance — without offering scientific evidence that they work, says Daniela A. Bota, MD, PhD, the UCI School of Medicine’s senior associate dean for clinical research and chief scientific officer for its Center for Clinical Research.
To address this issue, California passed a law requiring such stem cell clinics to post warnings that their treatments are unapproved and to provide the same statement to each customer before treatment begins. However, legitimate research and promising results also abound.
A Hub of Stem Cell Innovation
The California Institute for Regenerative Medicine (CIRM) was established in 2004 through the voter-approved Proposition 71 to help fund the establishment of stem cell research centers, including the SCRC. That effort has made the state a global hub for stem cell research. To date, CIRM has funded more than 55 clinical trials to study stem cell treatments for conditions ranging from cancers and HIV/AIDS to diabetes and blindness.
While it may seem that stem cell research is moving slowly to patients and the general public, Cummings says the pace of change has been rapid considering the regulatory challenges of working with human tissue and the complexities inherent in launching a new field of research.
“We had to build research facilities, establish research training programs, then we had to do the basic science of learning how to harvest the cells and how to direct them,” Cummings explains.
With more than 1,200 patients now enrolled in CIRM-supported clinical trials around the state, many investigators are on the cusp of major breakthroughs in treatments for blindness, blood diseases and cancer. Promising stem cell studies are also underway at UCI for amyotrophic lateral sclerosis (ALS) and diabetes, as well as CIRM-supported research in retinitis pigmentosa, Huntington’s disease and brain injuries.
The Future of Stem Cell Research in California
California’s future as an international leader in stem cell research isn’t set in stone, however. Because Proposition 71 funding is nearly spent, supporters of CIRM are planning a 2020 ballot initiative to ask voters to authorize $5 billion to continue supporting leading-edge stem cell science.
“If CIRM funding gets renewed, there will be a new burst of research efforts,” Cummings says. “I can’t overemphasize how important it is that scientists have the freedom to explore and work together with others to make connections that will take us down new, unexpected paths.”
Navigating the Stem Cell Landscape
In the meantime, consumers should be wary of claims touting stem cell cures. The risks of untested or fraudulent stem cell therapies can range from scam treatments that do nothing to the injection of stem cells that haven’t been properly programmed — potentially leading to harmful, perhaps even deadly, side effects.
“Stem cells are designed to divide and propagate, and they can form a tumor if you haven’t figured out their growth characteristics,” Cummings says. “Unlike a drug, you can’t remove the stem cells if you have an adverse reaction.”
Cummings recommends looking closely at a clinic’s language describing a therapy. It should say “FDA-approved,” which means a treatment has gone through years of required preclinical and clinical testing to prove that a treatment or drug is safe, does what is claimed and is available as a prescription.
Patients can find a much wider range of stem cell therapies being tested in the world of clinical trials — the research studies that are a core part of the FDA approval process. Eligible patients can join these trials and gain access to experimental stem cells therapies. However, Cummings cautions that “no reputable trial would ask patients to pay for experimental treatments, except for maybe travel costs in some cases. If you’re being asked to pay out-of-pocket for a stem cell treatment, it’s not a legitimate clinical trial.”
Empowering Patients through Education
Patients considering investigational stem cell therapies should first educate themselves to make sure a clinical trial is legitimate. Bota advises taking the following measures:
- Research the credentials and background of the researchers and clinicians involved.
- Verify that the trial is registered with clinicaltrials.gov, the U.S. National Library of Medicine’s database of privately and publicly funded clinical studies.
- Consult with your primary care physician or a specialist in the relevant medical field.
- Seek information from trusted, independent sources beyond the trial organizers.
“It’s extremely important to seek information from sources outside of the clinical trial,” Bota says. “Patients need to be well-informed to protect themselves from scams and fake studies.”
To learn more about UCI stem cell research, visit stemcell.uci.edu. UCI Health offers dozens of classes, seminars and support groups to help you live well. Take a class, join a group or attend a lecture, and allow us to be your partner in well-being.
The Hype and Hope of Regenerative Medicine
While the promise of stem cell therapies has been marred by unscrupulous clinics, the field of regenerative medicine as a whole holds tremendous potential for transforming healthcare. Regenerative medicine focuses on the body’s natural ability to repair, replace, and regenerate damaged or diseased cells, tissues, and organs.
At the heart of regenerative medicine are stem cells, which can differentiate into a wide variety of specialized cell types. Stem cell therapies aim to harness this remarkable ability to treat a range of conditions, from spinal cord injuries and neurodegenerative diseases to diabetes and heart disease.
Unlocking the Potential of Cord Blood Stem Cells
One promising area of regenerative medicine is the use of stem cells derived from umbilical cord blood. Cord blood is a rich source of hematopoietic (blood-forming) stem cells, which can be used to treat conditions like leukemia, lymphoma, and inherited blood disorders.
Cord blood stem cells have several advantages over other stem cell sources. They are more primitive and less likely to trigger immune rejection, and they can be easily collected and cryogenically stored for future use. Furthermore, the FDA has approved the use of cord blood stem cells for the treatment of certain blood and immune system disorders.
However, the field of cord blood stem cell research is not without its challenges. There are ongoing debates about the ethics and regulation of cord blood banking, both public and private. Some experts have raised concerns about the commercialization of cord blood banking and the potential for unequal access to these therapies.
Organoids and the Future of Disease Modeling
Another exciting area of regenerative medicine is the use of organoids, which are miniature, three-dimensional organ-like structures grown from stem cells. Organoids can be used to model a wide range of diseases, from cancer and neurodegenerative disorders to infectious diseases and genetic conditions.
Organoids offer several advantages over traditional cell culture and animal models. They more accurately reflect the complexity and functionality of human organs, and they can be used to study disease mechanisms, test new drugs, and even explore personalized treatments.
The development of organoids has been hailed as a major breakthrough in regenerative medicine, with the potential to accelerate the pace of drug discovery and improve the clinical relevance of pre-clinical studies. However, the field is not without its limitations and challenges, including the difficulty of scaling up production and the need for more robust validation and standardization of organoid systems.
Navigating the Ethical Landscape
As with any emerging field of biomedical research, regenerative medicine raises a host of ethical and regulatory considerations. Issues around informed consent, equitable access, and the potential for commercialization and commodification of human tissues and cells must be carefully navigated.
Policymakers and regulatory bodies, such as the European Union and the International Society for Stem Cell Research, have developed guidelines and frameworks to address these ethical concerns. These include principles of respect for human dignity, autonomy, justice, and the need for transparent and accountable processes for tissue and cell donation and use.
Ultimately, the success of regenerative medicine will depend not only on the pace of scientific and technological advancement but also on the ability of stakeholders to address the ethical and regulatory challenges that come with this rapidly evolving field.
Conclusion: Hype or Hope?
The field of regenerative medicine, with its promise of stem cell therapies and other innovative approaches, has generated tremendous excitement and hope. However, it has also been marred by hype, misinformation, and the proliferation of unproven and potentially harmful treatments.
As the field continues to evolve, it will be crucial for policymakers, researchers, clinicians, and the public to work together to separate fact from fiction, ensure rigorous scientific standards, and protect vulnerable patients from exploitation. Only then can the true potential of regenerative medicine be realized and translated into safe, effective, and equitable treatments that improve human health and well-being.
By staying informed, critically evaluating claims, and supporting legitimate research and clinical trials, we can help shape the future of regenerative medicine and harness its transformative power for the benefit of all.
