The Commercialisation of Unproven Mesenchymal Stem Cell Therapies
Evely Lass, Hollie Kennerley, Ryan Beadle, Charlotte Brookes, Ignacio Ruiz Fernández.
Department of Biology, University of York, York, UK, YO10 5DD
Introduction
Stems cells are primitive cells that can give rise, or “differentiate” into different cell and tissues types. As part of our tutorial assignment in Professor Paul Genever’s laboratory at the University of York, we were asked to look into a particular type of stem cell called the “mesenchymal stem cell” or “MSC”. The focus of the work was an examination of the commercial organisations that were offering MSC-based therapies for the treatment of different disorders, and the availability of the supporting scientific evidence. We were shocked by what we found.
A Background to MSCs
MSCs are adult stem cells that were first identified in bone marrow. They can give rise to cells that make bone, cartilage and fat. Similar cells to MSCs can also be found in adipose tissue and umbilical cord. Some researchers prefer not to call MSCs “stem cells” and instead refer to them as “stromal cells” as they tend to be used as a mixed cell population that may only contain a few genuine stem cells. Having said that, MSCs do have the potential to treat some diseases through their ability to differentiate and therefore repair damaged tissues, such as those affected by bone and joint diseases like osteoarthritis. MSCs also appear to have some immuno-suppressive functions and so may be used to help disorders that affect the immune system. We carried out a comprehensive search of the internet to discover what kinds of MSC therapies were being offered to patients on a commercial basis
Diseases and Procedures Offered for MSC Treatment
We found that commercial organisations are currently offering MSC therapies for a wide variety of diseases (see Table 1). The table collates all the different treatments we found across various organisations and the route through which the MSCs were administered to the patient. Some of these aim to treat age-related and degenerative disorders such as arthritis, neurodegeneration and back pain, but the range is huge, covering autism, cancer and hypogonadism among others. From an understanding of the biology of MSCs, it is almost impossible to explain rationally how such a cell could be used to exert reparative effects on such a long and diverse list of complex disease types. It was abundantly clear that many companies were offering unproven interventions that lacked scientific backing and were actively promoting “Stem Cell Tourism” by encouraging patients to travel to pay for their treatment.
Many of the procedures involve administering expanded MSCs intravenously via a saline drip. The number of MSCs introduced into a patient varies, often from 100 million to 300 million cells depending on the company and treatment offered. The MSCs are then presumed to home to the site of damage. However, there is good evidence from animal studies that the vast majority of intravenously infused MSCs are short-lived, they become entrapped in the lungs and will never reach the target tissue. In other treatment offerings, MSCs are injected directly into the tissue site, which may improve delivery and targeting. However, some of the company websites provide very little information about the administration methods they use. This is a concern. An important procedure such as this should be fully explained as this can have a huge influence on the thought processes of the patients who are considering opting for these procedures. In addition, most websites are unclear about any follow-up appointments to determine the clinical outcome.
Table 1:
Quality Control
The source of MSCs for these purposes is frequently adipose tissue. They acquire “adipose-derived stem cells”, which are broadly similar to MSCs, by taking a small amount of fat (around 200cc) from the patient’s fat tissue, using a method similar to liposuction. Most company websites refer to the isolation step and further amplification of the isolated MSCs, but most do not provide the specific procedures. Only a few claim to test the presence of cell “markers”, features that can identify certain cells, but do not provide the details. It is important to know what the cells are and how pure the sample is. Furthermore, the lack of a successful purification step not only implies that not enough MSCs are present to potentially induce a beneficial effect, but might also lead to the inclusion of unknown and unwanted elements that could have adverse consequences. In some cases, allogeneic therapies are offered, where the tissue comes from another donor, which poses the additional risk of rejection. This is why some companies also administer anti-inflammatory drugs in an attempt to combat this risk. Among the possible declared side effects of the MSC treatments are pain, discomfort, sleepiness and flu, but we could also find reference more severe conditions including cancer.
Treatment Cost and Clinical Outcome
Some companies that offer MSC therapies advertise treatment costs, others require the patient to contact the company after providing some personal information. Websites that do provide cost details have different payment programmes depending on the procedure they offer and extra expenses that might be required (e.g. overnight stay at the hospital, follow-up treatments). One company requires the patient to enrol firstly for the treatment costing around $6000, which includes qualifying blood test, fat extraction, processing and storage of the cells for one year. However, if the test results are unsatisfactory and the patient is not eligible for the treatment, the company will provide a full refund. Another company states that the cost of the treatment purely depends on the patient and the treatment. They do however specify that a “Platelet-Rich Plasma Injection Procedure” starts from $850, while the “Stem Cell Procedure” starts at $8500. For another firm, the price depends on diagnosis and the treatment but the fees start at $15,825 for children and $23,150 for adults.
Most of the companies do not show any scientific evidence of clinical outcome. Some of them do have links or references to review papers that discuss how and why MSC therapy has therapeutic potential. However, none of them mentions that the therapy they are providing is fully reliable and scientifically proven. In addition, the positive outcome and the success of the treatments mostly relies on client testimonials. The websites often have multiple success stories on how MSC therapy cured their or their family members’ diseases. We can conclude that the treatment with MSCs is expensive and the price can vary quite dramatically depending where and who is carrying out the procedure. Also, just by looking at the website the patient cannot find any information on safety profile and likelihood of a positive outcome based on published research specific to that treatment.
Final Thoughts
MSCs offer significant therapeutic potential, but must be used only for appropriate conditions with the correct safety controls, quality assurance and following robust scientific investigation. False advertising of unproven claims could persuade vulnerable individuals to take up overpriced ineffective treatments. High profile cases of adverse effects will drive investors away and damage the reputation of the field. It cannot continue. We feel that better educational resources are required for patients to understand their options so that they are able to make informed decisions. We have assembled some informative websites from authoritative sources that address the use of stem cells in medicine from a patient’s perspective. We encourage those interested in this subject to take a look.
Resources
A Closer Look at Stem Cells
www.closerlookatstemcells.org/
Produced by the International Society for Stem Cell Research (ISSCR).
Medicine and Stem Cells.
https://www.eurostemcell.org/landing/medicine-stem-cells
Education
https://www.eurostemcell.org/landing/education-resources
Both produced by The EuroStemCell Network
Stem Cell Basics: Educational Resources
https://www.stemcellfoundation.net.au/stem_cell_basics#educational_resources
Produced by the National Stem Cell Foundation of Australia