Transforming treatment paradigms with cell therapy

Written by:

Johan Hyllner

Head of Cell Therapy, Biopharmaceuticals R&D

Ryan Hicks

Head of Bioscience Cell Therapy, Biopharmaceuticals R&D

We are constantly pushing the boundaries of science to deliver life-changing medicines. One of our approaches to do this is with cell therapy. Through our investment in cell therapy and commitment to studying the mechanisms of repair and regeneration, we are creating the next generation of cell-based therapies across BioPharmaceuticals R&D.

There are over 200 different specialised cell types in the human body, each responsible for specific functions. The loss of many of these cells as a result of injury, disease or ageing is often irreversible. One of the aims of cell therapy is to introduce new, healthy cells into the body to restore function.

The beginning of cell therapy

Some cell therapies have been successfully used for many years now. The oldest example is from a little more than 200 years ago when James Blundell did the first successful blood transfusion at Guys Hospital in London and then later, the bone marrow transplant, which is now routinely used in medicine to effectively treat certain diseases of the blood and immune system. These are two examples of cell therapies that use minimally manipulated cells. Nowadays, with the development of new technologies such as pluripotent stem cells, next generation sequencing and gene editing, cell therapy efforts can include further modifications of the cells to greatly aid research and new medicines for some of the most difficult to treat diseases.

Cell therapy technology platforms

Cell therapy as an area has matured over the last 15 years and there are now medicines in the clinic, including a treatment used in the eye to replace damaged cells on the surface of the cornea and a gene therapy for Adenosine deaminase severe combined immunodeficiency (ADA-SCID), often referred to as the "bubble boy" disease. With many further potential therapies in pre-clinical and clinical phases, utilising a host of different approaches, a number of seemingly dissimilar diseases can be potentially treated, from Crohn’s disease, stroke, to Parkinson’s disease and deafness to name a few.

We have also seen significant breakthrough in Oncology with cancer immunotherapy. The launch in 2017 of the first CAR T-cell therapies to treat blood cancers was a critical milestone in the cell therapy field.

In BioPharmaceuticals R&D at AstraZeneca, we are researching pioneering approaches in the field of disease regression and organ regeneration for conditions such as chronic kidney disease, coronary artery disease, chronic heart failure, diabetes and nonalcoholic steatohepatitis (NASH). Our most advanced cell therapy project in the pipeline today is in heart failure, where the damage to heart muscle tissue caused by a heart attack is, at present, irreversible. In our preclinical research, we were able to replace the damaged heart tissue with healthy tissue. We are now closer to bringing a candidate drug to clinical trial, with the aim of ultimately delivering much needed regenerative therapies to patients.

Cell biology is a team effort

At AstraZeneca, we work across BioPharmaceuticals and Oncology to capitalise the existing and emerging platforms in stem cell technologies, new modalities, universal cell lines, next generation CAR-T and CRISPR-mediated genome editing. Our Discovery Science group is driving innovation across several platforms including gene engineering technologies. Our colleagues in Biopharmaceutical Development work with us from early on in the project, exploring efficient solutions for the manufacturing of cells and identifying the analytical development that is needed. The Antibody Discovery and Protein Engineering group has been instrumental in creating universal cells and CAR-T engineering. Our Clinical Pharmacology and Safety Sciences department use the latest methods to ensure our cell therapies are safe as well as efficacious.  Bringing in our experts from across our organisation, including our disease areas, regulatory and late stage departments, is extremely important as we are forging new paths with the aim of bring novel medicines to patients.