Capturing Patients’ Real-World Experiences to Improve Cancer Research and Care

A Q&A with Grace Castillo-Soyao, founder and CEO of Self Care Catalysts; grace@selfcarecatalysts.com

Q: You are well known as a visionary in the field of Real World Experience-Evidence (RWEE). As the founder and CEO of Self Care Catalysts, headquartered in Toronto, how do you see RWEE evolving to favorably impact the field of oncology?

A: I started Self Care Catalysts with some very basic questions. Why is the patient at the farthest end of the care line, treated as simply the recipient of care? It’s a very industrial mindset, a bit like an assembly line; the patient as something to be acted upon. But patients are often experts at their own conditions, including the many kinds of cancer. Why are they not invited to become participants in their own care, in contributing their own experiences? Why are patient experiences not considered to be scientifically valid?

As noted physician Sir William Osler famously said, “It is much more important to know what sort of a patient has a disease than what sort of a disease a patient has.” Continue reading…


The Crucial 90% Missed by Doctors on Computers

A Q&A with Kevin B. Knopf MD, MPH, chairman of hematology and oncology at Highland Hospital in Oakland, California; kevinbknopf@gmail.com

Q: A successful patient-physician relationship depends upon effective bidirectional attention and mutual understanding. Many patients and physicians believe that common current versions of mandated electronic health records (EHRs) severely impede that interaction, especially eye contact. How can a competent and caring clinical oncologist overcome that problem?

A: For all my faults as a doctor, and I’m sure there are many, there is one thing I think I do correctly, and that is I am never on a computer in front of a patient.

I hear from many colleagues that they can be efficient and personable while going back and forth from the patient to their electronic health record (EHR)—and it is true there are various levels of skill here. However, none do as well, in my opinion, as a computer-free patient environment. I say this from my side as a patient having seen dozens of doctors myself—nothing compares to a doctor who spends all of their time looking you in the eye and interacting face to face. This human contact costs nothing, and yet is so vital. Continue reading…


Challenging Oncology Therapies With Moonshot Price Tags

A Q&A with Pramod John, PhD, CEO of VIVIO Health, a specialty drug management company in San Leandro, CA, that aims to provide better outcomes at lower costs; pramod@viviohealth.com

Originally published December 13, 2017

Q: Some American pharmaceutical companies are well-known for pricing drugs at “whatever the market will bear.” In oncology, some specialty drugs seem to have price tags completely unrelated to the proven effectiveness of the drug. Your company has been taking a lead in confronting this problem. What do you envision as possible solutions?

A: New oncology therapies carry astronomical price tags—most people know this. Receiving far less attention is the question of actual therapeutic value. Drug manufacturers spend billions on advertisements and PR, but unfortunately, real-world patient results are frequently unimpressive. Two recent articles in BMJ make this point, 1) No evidence of benefits for popular oncology therapies and 2) Do cancer drugs improve survival or quality of life? Continue reading…


Best Uses of Physical Medicine and Rehabilitation in Patients with Cancer

A Q&A with Val Jones, MD, Medical Director of Admissions, Saint Luke’s Rehabilitation Institute, Spokane, WA

Originally published February 1, 2017

Q: Your principal practice in Spokane, Washington is physical medicine and rehabilitation (PM&R). What do you find to be the best uses of PM&R in patients with cancer at your facility?

A: Rehabilitation medicine is one of the best-kept secrets in healthcare. Although the specialty is as old as America’s Civil War, few people are familiar with its history and purpose. Born out of compassion for wounded soldiers in desperate need of societal re-entry and meaningful employment, “physical reconstruction” programs were developed to provide everything from adaptive equipment to family training, labor alternatives and psychological support for veterans.

Physical medicine and rehabilitation (PM&R) then expanded to meet the needs of those injured in World Wars I & II, followed closely by children disabled by the polio epidemic. In time, people recognized that a broad swath of diseases and traumatic injuries required focused medical and physical therapy to achieve optimal long term function. Today, cancer patients frequently benefit from comprehensive rehabilitation as they recover from the effects of chemo (neuropathy, weakness, and cognitive impairments), radiation (scarring and range of motion limitations), surgery (flaps, plastics procedures, tumor resection, amputations), and brain injuries (edema, debulking, gamma knife and neurosurgery). Continue reading…


The Promise of Plerixafor in Glioblastoma Treatment

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; adan.rios@uth.tmc.edu

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…


Who Owns Patient Data in Clinical Research?

A Q&A with Charlotte J. Haug, MD, PhD, MSc, International Correspondent, New England Journal of Medicine; Senior Scientist, SINTEF Techology and Society; Adjunct Affiliate, Stanford Health Policy; Oslo, Norway; charlottejohanne@gmail.com

Originally published October 25, 2017

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…


Might Cancer Be a Metabolic Disease?

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…


Accuracy and Precision Define Radiation Oncology

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; shyang@uabmc.edu

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…


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

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. Phase 1 or 2 trials: There are about 75 of these trials in the USA. There are many interesting choices here, but we do not have enough data to make an informed decision on which one to try. We do have early results from some phase 1 trials, which are much better than those seen with standard therapies, but it is not likely that any one of these alone will make a big difference in survival for most patients. We do not (under the current system) have the ongoing results of these trials—we only get the results a few months after the trial is over. And while inside the trial, we cannot combine them with other treatments.
    5. Off label use of drugs approved for other diseases. There are many choices here and a rational approach might be to select a “cocktail of drugs” based on a genomic analysis of my tumor.
    6. Cocktail approach involving experimental and approved treatments. Right now, this is impossible or very difficult to obtain. However, if it were possible, this would be my approach. Especially if we had a registry of all of the patients, the treatments tried, and the outcomes so we can learn from every patient.

Continue reading…