Options to Treat a Glioblastoma

A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY; email: musella@virtualtrials.com, phone: 888-295-4740

Originally published August 9, 2017

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.

  1. Standard of care: Surgery, radiation, Temozolomide. Chance of 5 year survival is about 5%.
  2. 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.
  3. 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.
  4. Continue reading…


Huge Progress in Palliative Care

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; diane.meier@mssm.edu

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…


Patient-Reported Outcomes Could Transform Cancer Care

A Q&A with Jared Adams MD, PhD, Chief Science Officer at Self Care Catalysts; jared@selfcarecatalysts.com

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…


Do You Have Pain, Cancer, or Diabetes? Your PBM May Now Be Your Doctor for These Illnesses.

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: charlesleebennett@gmail.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.

Continue reading…


Why the 21st Century Cures Act is a Good Thing

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…


Q&A: Air Traffic Control for Cancer

David K. Cundiff, MD
Jeff Shrager, PhD

A Q&A with David K. Cundiff, MD, Retired internist and palliative care physician from LA County + USC Medical Center; Email: dkcundiff@whistleblowerdoctor.org. Jeff Shrager, PhD, Director of Research, Cancer Commons; Adjunct Professor, Symbolic Systems Program, Stanford University; Email: jshrager@gmail.com.

Originally published August 16, 2017

Q: After May 17, 2017, you and Jeff Shrager engaged in a robust discussion about the place, if any, for an “Air Traffic Control system” called “Global Cumulative Treatment Analysis” (GCTA) for Cancer. Will you share excerpts with our readers?

DC: I see some major ethical and financial problems with GCTA:

  1. Who is going to authorize and pay for all treatment regimens (TRs) to be available to all patients at all times? USA health care costs are already out of control. An estimated one-third of all medical interventions do not benefit patients according to many evidence-based medical experts.
  2. How will vulnerable, terminally ill cancer patients and their loved ones be protected from ineffective, toxic, and expensive cancer treatments in the guise of advancing medical science?
  3. How will society be protected from the costs of ineffective cancer treatments with GCTA given that patient self-selection for experimental treatments generally means inconclusive results?

JS: There is no patient self-selection for experimental treatments in GCTA — or at least no more than there is now, and probably significantly less so. Under GCTA the patient, via their care team, is offered a set of options, along with rationales and data needed to make a rational choice about these options. This is just as it is today. The only difference is that under GCTA the set of options (rather than what your doctor happens to like) is guided by the GCTA process, which is running a global PROSPECTIVE adaptive trial over the whole equipoise set. Note that this is NOT “every damned thing” – just the things that we don’t know are better or worse.

Continue reading…


Forget Moonshots: Biomedicine Needs an Air Traffic Control System

A Q&A with Jeff Shrager, PhD, Director of Research, Cancer Commons; Adjunct Professor, Symbolic Systems Program, Stanford University

Originally published May 17, 2017

Q: There never seem to be enough patients matched to cancer clinical trials to quickly test new cancer treatments. Might there be a better way, using new communication technology?

A: Among the few things that everyone can agree upon, one is surely that biomedicine is not an efficient engineering system — that is, a system that efficiently (in time, cost, lives, or whatever else you’d like to measure) reaches its goals to alleviate suffering, etc. When we think of efficient engineering systems, companies such as Apple, Boeing, Google, Tesla, NASA, GE, and Toyota come to mind. Perhaps Big Pharma and parts of the VA and military are efficient engineering systems but surely biomedicine, as a whole, is not.

Granted, biomedicine faces the highly complex problem of matching patients to treatments while at the same time creating and ethically testing novel treatments with budgets that are under constant pressure. But this problem is faced by a huge number of extremely smart people. What is the difficulty? I contend that the difficulty is, in large part, lack of coordination. Continue reading…


Cancer Pain and the Opioid Epidemic

A Q&A with Kevin Sevarino, MD, PhD, President-elect of the American Academy of Addiction Psychiatry and Consulting Psychiatrist at Gaylord Hospital in Wallingford, CT

Q: Opioid abuse, addiction, and overdose are huge American problems right now. Many cancer patents experience chronic pain. What is the best way to use opioids to manage chronic pain?

[Note: The views expressed below represent the opinion of the author, and do not necessarily reflect the views of the American Academy of Addiction Psychiatry nor those of Gaylord Hospital.]

A. We live in amazing times. Targeted immunotherapies, stem cell transplants of transfected cells, identification of unique molecular targets in cancer cells through differential gene expression profiling—all promise to expand survival rates (or cures!) with diminished adverse effects compared to the “blunt hammer” approach of chemotherapy, radiation treatments, and more. Continue reading…


Proposed FDA “Conditional Approval”- More Details

A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY. Marty Tenenbaum, PhD, Founder and Chair, Cancer Commons, Los Altos, CA

Originally published May 10, 2017

Q: Your April 5, 2017 blog post that proposed a new “Conditional” category for FDA drug approval elicited a number of positive and negative responses. Please explain the proposal in more detail to enable better reader understanding.

A: In Response to “Conditional Approval: Right Solution for the Wrong Problem” by
Shannon Brownlee:

We appreciate Ms. Brownlee’s comments on our recent blog post, but think she missed our key points—perhaps we weren’t clear enough: Continue reading…