Medical research scientists, academics and clinicians mingled with entrepreneurs and patient advocates at the World Stem Cell Summit last December in West Palm Beach, Florida. In its fourth year as co-sponsor, Mayo Clinic Center for Regenerative Medicine sent a delegation to discuss the promise and the peril of the advancing science with about 1,200 attendees. Topics ranged from cardiovascular regeneration to growing stem cells in a microgravity environment in space.
An Informed Consumer
About 300 health care consumers were asking questions and engaging in discussions with presenters on Public Day. Karen Krucker, R.N., stem cell therapy program manager at Mayo Clinic, explained what patients and their families need to know before receiving stem cell or regenerative medicine treatments. Krucker worked at the Regenerative Medicine Consult Service at Mayo Clinic, the first consult service established in the United States to provide guidance for patients and families regarding stem cell-based protocols. In her role, instead of giving medical advice, she educated patients on how to make an informed decision.
“You need to be very careful. You need to have skepticism,” she told the audience. “Even injecting stem cells from your own fat cells can have risks.”
She stressed the importance of learning all you can about the formal training and experience the clinicians have before the treatment. “You don’t want someone trained as a dentist injecting stem cells into your heart,” she said. Are these treatments within their scope of practice?”
Krucker asked a series of questions to encourage the audience to be informed consumers about the treatments. “Who is going to follow up with you, what kind of care is available to you after the treatment, what happens is something goes wrong?” Krucker asked. “What are the risks, the benefits, and how do they measure results?
Training and Educating Medical Professionals
Dr. Richard Hayden, head and neck surgeon at Mayo Clinic, stressed the importance of education for the medical community, the public, and the workforce of the future. He said that on average, physicians have a poor understanding of regenerative medicine because of a gap between physician training and the emerging field of regenerative sciences.
In recognition of this challenge, Mayo Clinic is expanding its regenerative medicine programs at five Mayo Clinic Schools. One example is a new graduate program in the regenerative sciences at the Mayo Clinic Graduate School of Biomedical Science starting in July 2017.
Sarayna Wyles, M.D., Ph.D. candidate, described the regenerative medicine and surgery course for first year medical students at Mayo Clinic School of Medicine. She shared a video that shows how the course offers an introduction to the principles of regenerative medicine as applied to medical practice.
Panelists also gave information on educational programs at Wake Forest Institute for Regenerative Medicine and on the Student Society for Stem Cell Research, an international network for students.
Dr. Hayden, who is also the education director for the Center for Regenerative Medicine, discussed the impact of stem cell clinics on consumers, clinicians, and society.
“We are believers or we wouldn’t be here at the Summit,” he said. “We are concerned that the proliferation of unregulated clinics could jeopardize stem cell therapy and regenerative medicine in the United States. The issue is about patient safety. ”
Dr. Hayden says for consumers who are seeking treatment to look for clinical trials that are FDA regulated. ClinicalTrials.gov is an online resource and a registry of public and private sponsored human clinical trials. However, not all of the trials listed on clinicaltrials.gov are FDA regulated.
Mayo Clinic is conducting more than 40 active regenerative medicine clinical trials and is monitoring or participating in an additional 100 trials. The challenge for consumers is to find the right clinical trial and then to go through the qualification process. The number of people participating in clinical trials is small compared to the number of consumers who are seeking new treatments says Dr. Hayden.
Safety First: New Therapies, New Risks
Timothy Nelson, M.D., Ph.D., head of the Regenerative Medicine Consult Service at Mayo Clinic, moderated a discussion on the scientific challenges for the safety of cell-based therapeutic products, for which tumor formation is an identified risk.
“I believe we are on the cusp of some very significant breakthroughs,” Dr. Nelson says. “We have cells that stimulate the regenerative response we need to grow into new tissues. We also have cells that grow into tissues that we do not want.”
On the panel were Yoji Sato, Ph.D., head of the Division of Cell-Based Therapeutic Products, National Institute of Health Sciences of Japan in Tokyo; as well as Keiji Yamamoto, D.V.M, Ph.D., from Takeda Pharmaceutical Company, who leads an industry consortium called FIRM-the Committee for Non-Clinical Safety Evaluation of Pluripotent Stem Cell-derived Product.
The panelists called for guidelines to assess the risk of CTPs and for quality control of intermediate and finished products during the manufacturing process.
From Translation to Application: Meeting the Unmet Needs of the Patient
Dr. Shane Shapiro, an orthopedic surgeon at Mayo Clinic, says one of his challenges is having few treatment options for degenerative joint diseases such as arthritis. Non-surgical treatment options are limited to a combination of exercise or physical therapy, general pain relievers, and an occasional steroid injection.
Joint disorders, such as arthritis, are one of the most common medical conditions treated by stem cell clinics in the United States and around the world. To repair or prevent further degeneration, professional athletes turn to sports medicine doctors for help. One of the most commonly used products containing stem cells is concentrated bone marrow in which the blood and other elements are removed, leaving behind the stem cells.
Mayo Clinic researchers set out to study the safety of this treatment in a single-blind placebo-controlled trial of bone marrow aspirate concentrate for the knee. Early results show that bone marrow aspirate concentrate (BMAC) is safe to use and there were no serious adverse events from the procedure. Further study is required to determine efficacy and regenerative potential. Study participants reported pain relief in both knees, in one treated with BMAC and one treated with a saline injection. Pain scores in both knees decreased from baseline and continued for six months.
“We are encouraged because our patients did well and our product is safe, but we also recognize the need for further studies to clarify how stem cells work,” says Dr. Shapiro.
More research is needed to understand how naturally occurring regeneration works in humans, how it can be safely harnessed, reproduced, and consistently manufactured into new treatments.
Learn more about the Mayo Clinic Center for Regenerative Medicine. Watch our video: Healing from Within: The Promise of Regenerative Medicine.
– Angela Bingham, Dec. 21, 2016