A3 - Cell based gene therapy – promise and challenges

Conference Hall B1

Organised by FIP’s Hospital Pharmacy Section, in collaboration with the Industrial Pharmacy Section


Ryan Forrey (FIP Hospital Pharmacy Section, USA) and Igor Linhares de Castro (BiocadBrazil Farmaceutica Ltd, Brazil)


For several years, the standards of cancer treatment were based on surgery, chemotherapy, and radiation. Over the past two decades, targeted therapies mostly based on monoclonal became the gold standard for many cancer treatments. More recently, immunotherapies aimed to strengthen the power of a patient’s immune system to attack tumours have emerged as a powerful tool in cancer treatment regimens. An emerging immunotherapy approach is called cell-based therapy. It can involve the transplantation of stem cells or adoptive cell transfer (ACT), which consists of collecting and using patients’ own immune cells to treat their cancer.

There are several types of ACT, but CAR T-cell therapy is the one that has advanced furthest in clinical development with two CAR-T therapies approved by the US Food and Drug Administration and the European Medicines Agency, one for for adults with advanced lymphomas and the other also for acute lymphoblastic leukemia in children.

As understanding of the molecular basis on human disease has developed, the potential to develop cellular therapies by manipulating human genes holds great promise. Despite all scientific efforts, several questions remain unanswered, such as whether cell-based therapies will ever be effective against solid tumours like breast and colorectal cancer.

As these therapies are finally being commercialised, there are many important issues to consider regarding the rational use of these treatments. These include considerations of supply chain logistics, safe handling, formulary management, assuring an effective medicine use process, and pharmacoeconomics.

Given that these treatments are novel, it is important for pharmacists and pharmaceutical scientists to consider the full spectrum of the challenges and promise of these treatments in order to assure their rational and safe use. This session will cover the challenges in the development and the application in practice of cell-based gene therapies.


09:00 – 09:10 Introduction by the chairs

  1. 09:10 – 09:45 Introduction to cell-based therapies ― How they work and what they do
    Didier Mouliom (One Quality of Life, Cameroon)
  2. 09:45 – 10:20 Formulary and logistical considerations with the introduction of cell-based gene therapies
    Sue Kirsa (Monash Health, Australia)

10:20 – 10:40 Coffee/tea break

  1. 10:40 – 11:05 Cell-based gene therapies in oncology: Case study of the challenges of CAR-T therapy
    Jill Kolesar (University of Kentucky, USA)
  2. 11:05 – 11:30 Introducing cell-based gene therapies into the healthcare system — Pharmacoeconomic challenges
    Josep Guiu (Catalan Health and Social Care Consortium, Barcelona)
  3. 11:30 – 11:50 Panel discussion

11:50 – 11:55 Conclusion by the chair

11:55 – 12:00 Room refresh

Learning Objectives

At the end of this session, participants will be able to:

  1. Define gene therapy and describe the challenges with the development of cellular therapies and their commercialisation
  2. Describe the challenges and considerations in introducing gene therapy into the healthcare system, including formulary, safe handling, and logistical issues
  3. Describe the challenges presented by the introduction of CAR-T therapies in the management of cancer and the role of pharmacists in assuring safe and rational use
  4. Describe the pharmacoeconomic impact of gene therapies and potential approaches to deal with the significant costs.

Type of session: Knowledge-based