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 help unlock treatments that slow or reverse the effects of such devastating diseases as amyotrophic lateral sclerosis (ALS) or repair brain function in people with traumatic brain injuries, among many other uses.
But the promise of stem cells has been co-opted by unscrupulous clinics in the United States and around the globe that are offering unproven, even fraudulent, therapies.
Only a handful of approved treatments
“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.”
So far the U.S. Food & Drug Administration (FDA) has vetted and approved only 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.
State requires warnings about unapproved treatments
The problem is so pervasive that 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.
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.
Research moving at a rapid pace
While it may seem that stem cell research is moving slowly to patients and the general public, Cummings says the pace of change actually 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.”
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 also are 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.
New research funding needed
But California’s future as an international leader in stem cell research isn’t set in stone. 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 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.”
Consumers beware of claims
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. Know who is conducting the trial, though.
“No reputable trial would ask patients to pay for experimental treatments, except for maybe travel costs in some cases,” Cummings says. “If you’re being asked to pay out-of-pocket for a stem cell treatment, it’s not a legitimate clinical trial.”
Evaluating stem cell trials and treatments
Patients considering investigational stem cell therapies should first educate themselves to make sure a clinical trial is legitimate. Bota advises taking the following measures:
- Check ClinicalTrials.gov: Legitimate clinical trials are listed on this website, which is maintained at the National Institutes of Health, but not every trial listed is legitimate. “Just because a trial is registered on the site does not mean the government has actually vetted it,” she says.
- Ask about informed consent: Investigators of legitimate trials provide an informed-consent document that fully explains the treatments and the risks involved. “It’s very important you’re given enough time to ask questions and to be fully informed about the nature and risks of a treatment,” she adds.
- Get independent confirmation: Is the study approved by a review board? The FDA requires institutions engaging in human trials have a review board that assures the protection of the subjects’ rights and welfare.
- Check the data. Check to see if early research data is available in peer-reviewed publications.
- Is it FDA-approved? Determine whether the trial, itself, is FDA-authorized.
“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.