Cancer Commons leadership, including founder Marty Tenenbaum, executive director Erika Vial Monteverdi, and editor in chief George Lundberg, are speaking at the 2019 Precision Medicine World Conference (PMWC) in Silicon Valley. Every year, the conference attracts doctors, innovators, and patients “to learn first-hand about the latest developments and advancements in precision medicine and cutting-edge new strategies and solutions that are changing how patients are treated.”
On January 22 at 8:45 am, Marty will lead a panel discussing the potential of real-world evidence to inform research and clinical decision making: “Traditionally, the majority of evidence regarding the benefits and risks of cancer treatments is derived from clinical trial populations. However, the vast majority of cancer patients receive treatment outside the context of clinical trials. There is tremendous motivation to evaluate the benefits and risks of cancer treatments delivered in the context of ‘real world’ care. Real-world evidence (RWE) is typically defined as treatment that is not delivered in accordance with an investigational protocol and therefore lacks clearly specified endpoints and assessment intervals for determining benefit. This session will cover opportunities and challenges in using real-world evidence to inform clinical decision making in cancer. The session will review opportunities and challenges in defining real world endpoints and the development of methods and analytical tools capable of generating insights from RWE.” Continue reading…
A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY; email: email@example.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.
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.
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.
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.
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.