Author Interviews, JAMA, Stem Cells / 25.07.2017
Unregulated Direct-to-Consumer Treatment Centers Provide Stem Cells for Patients With Heart Failure
MedicalResearch.com Interview with:
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Dr. Hauptman[/caption]
Dr. Paul J. Hauptman, MD
Professor of Internal Medicine
Division of Cardiology
Assistant Dean, Clinical and Translational Research
Saint Louis University School of Medicine
St. Louis MO 63110-0250
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: A publication in 2016 by Leigh Turner from the University of Minnesota in 2016 shed light on the proliferation of stem cell centers or "businesses" that offer non FDA approved treatments, described as "stem cell therapy" for a variety of conditions. We opted to evaluate sites that claimed to treat heart failure. We collected data on type of infusion, need for a medical evaluation, board certification status of the center physician, cost and other factors. Self reported patient volumes were very variable. Most centers/businesses claimed to use autologous stem cells; a number offered ancillary treatment (i.e. vitamin infusions and hyperbaric oxygen); only one appeared to have a board-certified cardiologist involved. The costs were high for single infusions (mean price of $7694, SD 2737 for autologous cells; slightly less for allogeneic cells). Efficacy claims made during telephone calls with the centers were highly positive.
Dr. Hauptman[/caption]
Dr. Paul J. Hauptman, MD
Professor of Internal Medicine
Division of Cardiology
Assistant Dean, Clinical and Translational Research
Saint Louis University School of Medicine
St. Louis MO 63110-0250
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: A publication in 2016 by Leigh Turner from the University of Minnesota in 2016 shed light on the proliferation of stem cell centers or "businesses" that offer non FDA approved treatments, described as "stem cell therapy" for a variety of conditions. We opted to evaluate sites that claimed to treat heart failure. We collected data on type of infusion, need for a medical evaluation, board certification status of the center physician, cost and other factors. Self reported patient volumes were very variable. Most centers/businesses claimed to use autologous stem cells; a number offered ancillary treatment (i.e. vitamin infusions and hyperbaric oxygen); only one appeared to have a board-certified cardiologist involved. The costs were high for single infusions (mean price of $7694, SD 2737 for autologous cells; slightly less for allogeneic cells). Efficacy claims made during telephone calls with the centers were highly positive.


















Dr. Fu Guosheng[/caption]
Fu Guosheng MD
Professor and Chairman, Department of Cardiology
Sir Run Run Shaw Hospital, College of Medicine
Zhejiang University
Hangzhou, China
MedicalResearch.com: What is the background for this study?
Response: Acute myocardial infarction (AMI) remains a major cause of long term morbidity and mortality worldwide. Although we can re-vascularize the occluded vessels by cardiac intervention or coronary artery bypass graft (CABG), it is not helpful for the damaged myocardium, which urges us to find a new therapeutic method. An increasing body of evidence from a wide range of experimental animal studies and clinical trials suggests that endothelial progenitor cell (EPC) transplantation can repair “broken” heart by involving direct angiogenesis and secreting protective paracrine factors, which has a bright prospect for clinical application. However, transplantation of autologous EPC has numerous limitations, including the limited supply of expanded EPC, the impaired function and activity of the transplanted cells, and so on. Therefore, it is desirable to develop novel proangiogenic strategies that improve the efficacy of EPC transplantation.
Dr. Joseph Alvarnas[/caption]
Joseph Alvarnas, MD
Associate clinical professor
Department of hematology and Director of value-based analytics
City of Hope National Medical Center
Duarte, CA
MedicalResearch.com: What is the background for this study?
Dr. Alvarnas: Patients with HIV infection have a significantly increased risk of non-Hodgkin lymphoma and Hodgkin lymphoma. Prior to the availability of effective anti-retroviral therapy, HIV-infected patients with lymphoma had very poor treatment outcomes. Following the availability of effective anti-HIV therapy, patient outcomes for HIV-infected patients now parallel those of non-infected patients. Historically, however, HIV infection has been used as a criterion for not offering patients autologous blood stem cell transplantation outside of centers with unique expertise. The purpose of this trial was to evaluate outcomes, complication rates, and immunological reconstitution of HIV-infected patients following autologous blood stem cell transplantation.
Dr. Philip McCarthy[/caption]
Philip McCarthy, BA, MD
Professor of Oncology
Director, Blood and Marrow Transplant Program
Roswell Park Cancer Institute
Associate Professor of Medicine
Jacobs School of Medicine and Biomedical Sciences
State University of New York at Buffalo
Buffalo, NY 14263
MedicalResearch.com: What is the background for this study? What are the main findings?
Dr. McCarthy: There have been three Phase III studies that examined the role of maintenance lenalidomide after autologous stem cell transplant (ASCT) for newly diagnosed multiple myeloma patients. IFM 2005-02 (France), CALGB 100104 (Alliance, USA), GIMEMA-RVMM-PI-209 (Italy). All three studies had progression free survival (PFS) as their primary endpoint and all demonstrated a superior PFS when compared to placebo or no therapy after ASCT. However only the CALGB 100104 study demonstrated a statistically superior overall survival (OS). Thus, a meta-analysis was necessary to assess the effect of post-ASCT lenalidomide maintenance on overall survival. This study utilized a pooled analysis of updated primary-source patient data from all three studies after the primary efficacy analyses had been conducted. The meta-analysis demonstrated that there is a statistically superior OS (P value=0.001, HR=0.74 (0.62-0.89)), Median OS for no maintenance or placebo was 86 months and the median OS for lenalidomide had not been reached. The median OS for lenalidomide treatment arm was extrapolated to be 116 months based on median of the control arm and HR (median, 86 months; HR = 0.74). Thus, there is a 26% reduction in the risk of death which is an estimated 2.5 year increase in median OS. There is an increased incidence of second primary malignancies with lenalidomide maintenance when compared to placebo but this risk is less than the risk of dying when not receiving lenalidomide.
Dr. Timothy Henry[/caption]
Timothy D. Henry, MD, MSCAI
Director, Division of Cardiology
Cedars-Sinai Heart Institute
MedicalResearch.com: What is the background for this study? What are the main findings?
Dr. Henry: Heart failure it the #1 cause of morbidity, mortality and cost in the United States today. Patients with Class 3 heart failure, despite optimal medical therapy and device therapy have limited options beyond heart transplantation and left ventricular cyst device.
Transplantation and LVAD are expensive and are challenged by both availability and complications. Therefore, treatment for patients with ongoing symptoms despite medical therapy is an admiral goal. Stem cell therapy appears to be an attractive choice for these patients, in particular patients with ischemic cardiomyopathy.
The ATHENA trial was designed to treat patients with ischemic cardiomyopathy and ongoing ischemia with autologous adipose-derived regenerative cells. Patients would undergo liposuction with onsite processing of their stem cells in 1 ½ - 2 hours, followed by intramyocardial injection of adipose-derived regenerative cells (ADCRs) vs. placebo.
Dr. Keir Menzies[/caption]
Keir Menzies PhD
Assistant Professor
University of Ottawa Brain and Mind Research Institute
University of Ottawa
MedicalResearch.com: What is the background for this study? What are the main findings?
Dr. Menzies: Currently there is significant amount of research identifying the power of stem cells to regenerate damaged or aging tissue. Our research discovered that reduced stem cell health was linked to unusually low levels of a small molecule called NAD, one of the most important cellular molecules to maintain the performance of mitochondria, the engine of the cell. Then by boosting NAD levels, using a special form of vitamin B3 called nicotinamide riboside, stem cells could be rejuvenated during aging by improving mitochondrial function. We then go on to show that by improving stem cell function we could prolong the lifespan of mice, even when the treatment began at a relatively old age.
