Study Design

Pharmaceutical Outsourcing – Using Science to Drive Successful Study Strategy in Hematological Malignancies

Advances in our understanding of the genetic drivers of cancer and the immune system’s complex response to cancer have led to significant breakthroughs in the treatment of hematological malignancies. While gene therapy technologies are addressing unmet needs in hemato-oncology, the design and execution of clinical trials of these therapies can be challenging.

Obstacles in researching cancer immunotherapy range from variability in patient response and inability to predict treatment efficacy to appropriate endpoint selection, safety considerations, and recruitment. Planning and operationalizing clinical studies of immuno-gene therapeutics to achieve conclusive data outcomes requires sponsors to select an appropriate regulatory pathway and understand the nuances of endpoint selection, adverse event management, site selection, and patient engagement.

Current Trends in Gene Therapy Technologies for Hemato-oncology

Among the wide array of gene therapy technologies, therapeutic cancer vaccines and adoptive cell transfer therapies are the most studied to date. Therapeutic cancer vaccines aim to help the body’s own T-cells produce more anti-cancer T-cells and may be cellular-based, protein-based, or vector-based. Adoptive cell transfer therapies involve taking T-cells from a patient, modifying and expanding them ex vivo, and then infusing them back into the patients. These therapies may be tumor infiltrating lymphocytes (TILs), T-cell receptors (TCRs), or chimeric antigen receptor (CAR)-T cells.

In August 2017, the U.S. Food and Drug Administration approved the first-ever chimeric antigen receptor (CAR)-T therapy, tisagenlecleucel (marketed as Kymriah™).1 This landmark approval was followed by the approval of axicabtagene ciloleucel (marketed as Yescarta™) in October 2017.2 The hefty price tag of these therapies reflects not only the complex drug development pathway for immuno-gene therapeutics, but also the complexity and coordination involved in manufacturing and administering them. Thus, for many immuno-oncology (IO) agents, the product technology may influence or even define the clinical trial strategy.

Read more at Pharmaceutical Outsourcing.