A Q&A with Adan Rios, MD; Professor in the Division of Oncology-Department of Internal Medicine of The University of Texas McGovern Medical School at Houston, Texas Medical Center; firstname.lastname@example.org
Q: Glioblastoma multiforme (GBM) remains a scourge with a typically rapid fatal course resistant to most therapy. All solid tumors must receive sufficient blood supply to grow. Plerixafor is an FDA-approved drug that may inhibit tumor angiogenesis. How might plerixafor be sensibly used off-label as an adjunctive therapy for GBM?
A: Glioblastoma multiforme (GBM) is a CNS (central nervous system) tumor with post-therapy median time to progression of 7 months and median overall survival of 15 months. I decided to use plerixafor for the prevention of recurrence of GBM in one patient treated with standard chemo-radiotherapy five years ago and since then have studied this patient and this subject in depth. Continue reading…
Q: Many people are coming to believe that active patient participation will be a key to more rapid movement forward in cancer research. Data sharing can help. But who owns the data? And what rights and responsibilities are thus conferred? Your recent NEJM article provides helpful background. Can you help us better understand?
A: Exchange of data between patients and doctors is essential for the practice of medicine – and patient data are essential for medical research and progress.
Traditionally, doctors collected patients’ health information (typically the medical history, laboratory tests, drugs prescribed, outcome of treatment, etc.) and sometimes shared that information, in confidence, with colleagues to seek advice and advance science. The medical record was the physician’s property, and still is in many countries and legislations. But do physicians own the patient data? Continue reading…
A Q&A with Thomas N. Seyfried, PhD, Professor of Biology, Boston College
Q: As a geneticist, you know that the genomic makeup of cancers recently has captivated much of the scientific community with new knowledge and new treatments. And yet, cancer outcomes remain dismal for many patients. You have written about cancer from a very different perspective. Why do you consider cancer to be a metabolic disease, and how might we look at different therapeutic options under that rubric?
A: Over 1,600 people die each day from cancer in the U.S., according to recent data from the American Cancer Society (Siegel et al., 2018). The failure to manage cancer has been due in large part to the dogmatic belief that cancer is a constellation of genetic diseases. Accumulating evidence, however, indicates that cancer is primarily a mitochondrial metabolic disease involving disturbances in energy production through respiration and fermentation (Seyfried et al., 2014).
The disturbances in tumor cell energy metabolism are linked to abnormalities in the structure and function of mitochondria that disrupt ATP synthesis through oxidative phosphorylation (OXPhos) (Seyfried, 2015; Seyfried & Shelton, 2010). Consequently, all cancer can be considered a single disease with a common pathophysiological mechanism involving dysfunction of mitochondrial OxPhos. As reactive oxygen species (ROS) arise from defects in mitochondrial OxPhos, the gene mutations observed in various cancers and all other recognized cancer hallmarks are considered downstream effects, and not causes, of the initial disturbance of cellular energy metabolism (Seyfried, 2012; Seyfried et al., 2014). Continue reading…
A Q&A with Eddy Yang, MD, PhD, Professor and Vice Chair of Translational Sciences Department of Radiation Oncology; Deputy Director, Associate Director of Precision Oncology at the Hugh Kaul Precision Medicine Institute; Birmingham, AL; email@example.com
Originally published December 5, 2017
Q: You are a radiation oncologist with a particular interest in cancer of the prostate. How does the molecular study of prostate, as well as other cancers, including Next Generation Sequencing (NGS), help inform Precision Radiation Oncology?
A: Radiation oncology is a specialty where the accuracy and precision of treatment delivery is vital to the safety and outcomes of our patients. Many specialized techniques are utilized to enhance this precision, including intensity modulated radiation therapy, image-guided radiation therapy, and volumetric arc therapy. Emerging modalities such as proton and carbon therapy take advantage of the physics of heavy ions to potentially minimize normal tissue toxicity. With these methods, we are in essence, performing precision oncology, tailoring radiotherapy to each individual patient. However, precision oncology is much more than that, as novel technologies have expanded our understanding of the drivers of cancer that may be targetable or dictate response to treatment. Currently, emerging evidence has shown the benefits of biomarker-directed systemic treatments, but what about genomic markers to guide radiation therapy? Although the preclinical and retrospective data supports the notion of this possibility, results from prospective studies are not yet available. Continue reading…
A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY; email: firstname.lastname@example.org, phone: 888-295-4740
Q: You direct an established foundation that supports research and information about brain tumors. What would you do if you yourself were diagnosed with a glioblastoma multiforme (GBM)?
A: Now that GBMs are in the news again, I would like to discuss what I would do if it happened to me—a newly diagnosed GBM in an adult in otherwise good shape. There are several choices.
Standard of care: Surgery, radiation, Temozolomide. Chance of 5 year survival is about 5%.
Standard of care PLUS Optune. Bumps my chance of 5 year survival up to 24.9% (if used over 90% of the time) with no added toxicity.
Phase 3 Clinical trials: There are now about nine phase 3 trials for newly diagnosed GBM. Some have impressive phase 1 and phase 2 data. By the time a treatment gets to phase 3, it has shown enough promise in earlier trials that the sponsor is willing to risk a lot of money to test in a phase 3 trial. Most have two big downsides: 1) Most have a control group of patients who receive the old standard of care so that some of the participants do not get the experimental treatment. 2) Most do not allow you to use Optune, so you are trading a known benefit for a chance at an unknown benefit.
A Q&A with Diane E. Meier, MD, FACP, Director, Center to Advance Palliative Care; Professor of Geriatrics and Palliative Medicine, Icahn School of Medicine at Mount Sinai; New York, NY; email@example.com
Originally published November 8, 2017
Q: You wrote in MedGenMed in 2007 that palliative care was the job of all hospitals. In October 2017 you were honored at the National Academy of Medicine for your achievements in this field. How fully has your charge to hospitals in 2007 been realized?
A: Palliative care is a fairly new medical specialty devoted to reducing suffering and improving quality of life for people living with serious illness-whether the disease is curable, chronic, or life threatening and progressive. Palliative care teams work alongside disease treatment specialists to provide an added layer of support in service of pain and symptom management, family support, attention to the social determinants of health, and skilled communication about what to expect and what matters most to the patient in the context of the reality of the illness. Multiple studies demonstrate palliative care’s contribution to achievement of the triple aim: better experience of care, better care outcomes (including survival in several studies), and as an epiphenomenon of better care, much lower unnecessary utilization of 911 calls, ED visits, and hospitalization. Continue reading…
A Q&A with Jared Adams MD, PhD, Chief Science Officer at Self Care Catalysts; firstname.lastname@example.org
Q: Patient-reported outcomes (PROs) are health care outcomes, such as symptoms or quality of life, reported directly by a patient. In recent years, PROs have emerged as a potentially powerful new way to understand cancer outcomes. Could PROs lead to the next breakthrough in our understanding of cancer?
A: When biochemist and Nobel Prize winner Kary Mullis spoke to my undergraduate class some 20 years ago about his invention of the PCR method for genetic amplification, he put it in historical context by mentioning that every major clinical advance has been preceded by a breakthrough in scientific investigative methods that allowed us to “see” in new ways. Dutch scientist Antonie Van Leeuwenhoek’s microscope allowed scientists to see cells for the first time, advancing us beyond the notion of cancer being caused by an abundance of black bile. The PCR method allowed us to see and manipulate cancer at the genetic level, leading us down the road to targeted therapies aimed at specific genetic mutations. Advances in computer hardware and modeling techniques have allowed us to map the genomes of cancers, moving us beyond a simplistic organ-based model of disease and setting up the possibility of new drug discoveries in silico. Observing and understanding how cancer cells interact with circulatory and immune systems led to VEGF inhibitors, PD-1/PD-L1 inhibitors, and the list goes on… Continue reading…
A Q&A with Charles L. Bennett, MD, PhD, MPP; Smart State and Frank P and Jose M Fletcher Chair, Medication Safety and Efficacy, Smart State Center of Economic Excellence, University of South Carolina and the Hollings Cancer Center at the Medical University of South Carolina; Charleston, South Carolina; Email: email@example.com
Originally published December 27, 2017
Q: The opioid epidemic is now a public health emergency in the United States. Diabetes is now the leading public health emergency worldwide. We recently (June 21, 2017) discussed how Pharmaceutical Benefit Managers (PBMs) have developed from 1968 to controlling not only pricing, discounts, and drug selection for > 250 million Americans, but are also threatening to become the prescriber. Does this raise heightened concerns as the country faces two public health epidemics of opioids and diabetes, in particular?
A: You and I continue to believe that medical care responsibility logically devolves to the doctor who cares for the patient and is accountable for management, treatment, and outcomes. We are increasingly wrong. Since medicines have grown in cost and pharmaceutical costs continue to increase, profit-oriented businesses muscle the patient and the doctor (and stay tuned, even the hospital) out of the way. Pharmacy Benefit Manager (PBM)’s plans now often replace physician choice, and if the physician’s choice is expensive, they replace it with more profitable alternatives, a practice known as non-medical switching. These concerns are magnified as one of the largest PBMs (CVS) seeks to merge with one of the largest health insurers (AETNA), ultimately increasing PBM involvement where patients receive care. Just as worrisome is the “invisible” hand of PBMs in restricting treatment options for chronic pain often experienced by cancer patients and others.
A Q&A with Mary Woolley, President and CEO of Research!America
Q: You attended the December 2016 signing by President Obama of the 21st Century Cures Act and are recognized to be a strong supporter. Yet harsh criticism of it has quickly appeared in JAMA, BMJ, a variety of other venues, as well as on these pages. Please tell our readers why this is good legislation and how the public health will be protected from exploitation in this very different regulatory world.
A: The bi-partisan 21st Century Cures Act is grounded in a commitment to assuring that our nation’s research ecosystem has the capacity to accelerate the pace at which safe and effective medical advances reach patients. The Act will expand the efficiency, reach and impact of medical discovery in a manner that sustains crucial safeguards against unsafe or ineffective products. The law finances more research, helps to reduce the administrative cost surrounding basic research, and takes additional steps to overcome challenges the Food and Drug Administration (FDA) faces. Patient groups, health care professionals, academic leaders, industry leaders and the FDA and the National Institutes of Health (NIH) were frequently consulted regarding provisions of this bipartisan bill, and their insights were incorporated. We at Research!America were closely involved throughout development of the bill, and are pleased that it crossed the finish line last December. Continue reading…