Clinicians’ Expectations for Gene-Driven Cancer Therapy

Abstract

A new era of medicine is rapidly approaching, which will change not only pathological diagnosis but also medical decision-making. This paper raises the question of how well prepared doctors are to address the new issues that will soon confront them. The human genome has been completely sequenced and general understanding about cancer biology has increased enormously with understanding that unregulated gene function and complicated changes in signal pathways are related to uncontrolled cell growth. Thus, gene-driven therapy involving alterations to genes are recognized to present new therapy options. This advance will necessitate major changes to the decision-making aspect of physicians. This article focuses on defining the pertinent changes and addressing what they mean for practicing physicians.

Physician perspectives on compassionate use in pediatric oncology

“…Most clinical trials for cancer therapies are designed to enroll patients 18 years of age or older, even when there is a biologically plausible potential for efficacy in the pediatric population . Children with cancer who fail to respond to standard treatment, for whom there are no approved therapies available, and who are not eligible for a clinical trial may benefit from compassionate use programs.…”

“…Most clinical trials for cancer therapies are designed to enroll patients 18 years of age or older, even when there is a biologically plausible potential for efficacy in the pediatric population. 1,2 Children with cancer who fail to respond to standard treatment, for whom there are no approved therapies available, and who are not eligible for a clinical trial may benefit from compassionate use programs. Compassionate use programs-also known as expanded access programs, named patient programs, single patient investigational new drug (IND) applications, early access programs, or preapproval access programs-provide an opportunity for select patients to access investigational drugs, biologics, or medical devices when enrollment into a clinical trial is not Abbreviations: COG, Children's Oncology Group; FDA, Food and Drug Administration; IND, investigational new drug; IRB, Institutional Review Board; NCI, National Cancer Institute; rFVIIa, recombinant activated coagulation factor VIIa possible.…”

SMART precision cancer medicine: a FHIR-based app to provide genomic information at the point of care

“…The ultimate goals of the SMART PCM app are to (1) provide practitioners with contextdependent population-level cancer mutation information, (2) act as a within-workflow intermediary to select external knowledge bases, and (3) enable a patient-centered and gene-driven shared decision-making model. [38][39][40] Patients, caregivers, and clinicians do not wish "precision medicine" to be a mere buzzword, but rather want to know the context of the disease (eg, untreated BRAF-mutated metastatic colon cancer), the prognosis of the disease (eg, the median survival for a patient with untreated BRAF-mutated metastatic colon cancer), and what can be done about the disease (eg, published efficacious treatment regimens for untreated BRAF-mutated metastatic colon cancer). At the same time, personalization does not mean consignment to isolation.…”

Mechanisms and clinical implications of tumor heterogeneity and convergence on recurrent phenotypes

“…Tumor -omics vary greatly across cancer cells through time and space. Clinicians use profiling of tumor mutations and gene expression to diagnose, treat, and make prognostic decisions regarding a patient’s cancer [17–19]. Response to targeted therapies requires presence of specific -omic events throughout a patient’s tumor as well as robust methods of detecting these events.…”