This section includes answers to questions patients often ask. It is not intended to be all inclusive, replace sound research and decision making or to serve as medical advice in any way. It is intended to serve as a tool for patients and health care consumers who may be interested in learning more about cellular therapy procedures.
Health care consumers must be informed of all aspects of potential therapies. A product's safety and effectiveness are two different questions. Even if a product is proven to be reasonably safe, it may not necessarily be effective for all medical conditions or treatments. Medical treatments are associated with both risks and benefits. Individuals considering medical treatments should discuss the risks and potential benefits of those treatments with a health care provider. There are a variety of cellular therapies that use cells from different sources including blood, bone marrow, umbilical cord blood and fat. These products each have varying characteristics, and the intended applications also may vary greatly. Many cell therapies — for example, hematopoietic stem cell transplants — have been used for decades and may be used to treat diseases such as leukemia. Some of these are even considered "standard of care" today. Other cell therapies may still be in development. Some of these may be referred to as an investigational new drug or biologic in the United States. As with any medical or investigational treatment, consumers considering a cell therapy procedure should speak with their doctors to learn more about the effectiveness of the treatment, as well as any potential risks and benefits associated with the therapy.
Stem cells have potential to develop into blood, bones, brain, heart and other organs of the body. It is because of these properties that they hold such potential to treat diseases or conditions for which there may be few other treatments. However, as with any procedure, there are safety risks which may be known, unknown or unforeseen. Risks may include, but are not limited to, infection, tissue rejection and changes in the characteristics of the cells in the product that may alter how they respond. It is essential that individuals considering cell-based treatments investigate and discuss possible risks with their physicians. Before proceeding with any treatment, a patient should have gone through the informed consent process in which a knowledgeable health care professional describes all of the material risks, benefits and alternatives, provides the patient an opportunity to have his or her questions answered and provides an informed consent form for the patient's signature.
Factors a patient may want to consider before undergoing a cell therapy procedure include the following:
Physician Selection: Consumers should ensure that the physician who performs the treatment holds a medical license and is board certified in the specific field of medicine or treating specialty for the condition being addressed. The physician should hold a valid and current medical license in the treating state, province or country of practice.
Medical Claims: Claims such as "miracle cure" or claims to cure serious conditions often are not credible and may pose a significant risk to public health and safety. All claims made by clinics and/or physicians should be based on accepted and acknowledged scientific evidence and medical experience. Supporting data should be available for any treatment or outcome claim. Claims based on patient experiences, including testimonials, are not sufficient in the absence of scientific data. Data also should include the long-term follow-up of treated patients. Claims of safety should be based upon the collection of data through an established and accepted grading scale, such as the Adverse Event Reporting system used by the U.S. Department of Health and Human Services Office of Human Research Protections. Data should be validated by an independent, third-party organization prior to publication of any claim of safety or outcome. Outcomes data from treatments often are published in peer reviewed medical journals. Peer review means that qualified individuals or experts in the field have reviewed the methods, data and conclusions in a journal article to determine whether the article should be published. This helps ensure it meets scientific standards. The reviewers of peer reviewed medical journals have a process to evaluate the quality of the outcome data reported.
Travel for Therapies: Consumers considering stem cell transplantation in the U.S. should ask their physicians if the necessary Food and Drug Administration, or FDA, approval has been obtained or if the procedure will be part of an FDA-regulated clinical study. The FDA is the federal agency of the U.S. responsible for monitoring trading and safety standards in the food and drug industries. Appropriate FDA oversight applies whether the treatment will use another donor's cells or the patient's own cells. Even with the patient's own cells, some safety risks may be introduced when cells are manipulated after their removal. Consumers in other countries should explore the same considerations through their own medical practitioners and regulatory agencies.
Consumers who are considering treatments outside of their own country should carefully evaluate the regulatory status of that treatment. For example, consumers in the U.S. should be aware that, in most cases, FDA has not reviewed clinical data associated with products offered in other countries and is not able to comment on their safety and effectiveness. Moreover, treatments offered in other countries may be subjected to different levels of oversight than products marketed in the U.S. Consumers are encouraged to become familiar with the regulations governing any product they are considering and to ask questions about safety and efficacy studies of the regulating authority in the country where the treatment will be received. Click here to learn more about how stem cells are regulated.
Consumers should be sure that the facility in which they will receive the treatment is accredited or certified by a recognized accrediting or certifying body such as AABB. Consumers also should ask if the facility meets all regulatory requirements for the country in which it is located. Serious adverse events have been reported in some cases, with some additional treatments and emergency medical care being required. Therefore, it is best to ensure that the facility is either part of a hospital or has an agreement in place to transport and admit patients in the event of an unexpected medical problem. The treating facility should be close to a hospital that can deliver urgent medical care.
A clinical trial is a research study involving human volunteers that is focused on answering specific health or scientific medical questions. The clinicaltrials.gov database provided by the U.S. National Institutes of Health, or NIH, lists federally and privately supported clinical trials conducted in the U.S. and around the world. The NIH is the principal biomedical research agency of the U.S. Department of Health and Human Services, whose mission is to employ science in the pursuit of knowledge to improve human health. Please note, as stated on the site, that this information should be used in conjunction with advice from health care professionals. Once clinical trials are complete, data often are submitted to a scientific or medical peer reviewed journal for publication and to regulatory agencies for product approval or advancement to the next phase of product approval.
Consumers are encouraged to become empowered with as much reliable and independently verified information as possible. Individuals should ask whether the treatment is routine, if there are any scientific data to predict expected response, and the benefits or adverse effects of the treatment. Consumers also should learn about how the treatment will be performed, the collection of outcome data and any follow-up treatment necessary. Individuals should seek out articles and literature that have been reviewed or examined by experts in the field and check that the treatment claims are substantiated or have been reproduced by credentialed groups and credible studies.
The consumer also should assess the facility where the treatment will be given and determine whether the personnel are knowledgeable, trained in the particular area and know what to do in an emergency situation. Informed decision making also includes understanding regulatory oversight, certification and accreditation.
July 21, 2021
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