If cell therapy isn’t about the cells or the therapy, what is it about?

Author: David Brindley, 11/19/14


With contributions from James Smith, a recent Oxford University graduate and current CASMI Translational Stem Cell Consortium Research Associate.


Both the IBC Cell Therapy Bioprocessing (ICTB) conference and the Stem Cells and Regenerative Medicine Congress (SCRMC) took place recently. The conferences shared several common themes: one being the presence of a significant focus on gene therapies. There were also talks covering immunotherapy and touching on tissue engineering. Given the conference titles, this might seem surprising.

‘Cell therapy’ and ‘regenerative medicine’ are closely intertwined and often used synonymously. In fact, cell therapy is just one of several disciplines under the umbrella term of regenerative medicine. As ‘stem cell technology’ has acquired status as a metonym for stem cell-based therapeutics, so the use of ‘cell therapy’ as a way to denote regenerative medicine has become common parlance.

The ultimate goal of regenerative medicine is to develop new therapies and improve patient outcomes, not necessarily via any particular route. The field is generally considered to be quite young. However, as was pointed out in a recent Nature editorial, some of the most successful products to emerge from the biotech industry are regenerative medicines. Neupogen® is used to treat neutropenia and works by stimulating white blood cell proliferation. Epogen® is used to treat anaemia and stimulates red blood cell production. Both have been immensely successful commercially and medically. Though cell therapy perhaps reserves the greatest potential in regenerative medicine, examples like these show that so far the most significant regenerative medicines have not been cell-based.

Gene therapy, like cell therapy, has great potential in regenerative medicine. At SCRMC, Philip Astley-Sparke, from uniQure, stated that the “dawn of gene therapy” was upon us after 20 years of anticipation. Glybera®, a treatment to restore (or ‘regenerate’) lipoprotein lipase (LPL) enzyme activity, is now commercially available and according to him, six gene therapy companies are now public.

Much like cells, safety was a large concern for gene therapy; now that many trials have been done in the U.S., largely demonstrating safety, if efficacy can be proven we may see many more gene therapies reaching the market.

Cyrus Mozayeni, from bluebird bio, also stressed the potential impact of gene therapies as disruptive platforms. Treatments for sickle-cell anaemia and β-thalassemia are currently under development and look promising, based on the data presented.

Gene editing technologies will play an important role in the success of gene therapy. We discussed CRISPR in a previous post, where George Church suggested that it could be used to ‘regenerate’ the epigenome. Similarly, at ICTB, Martin Giedlin, Sangamo Biosciences, described zinc-finger nuclease-guided genomic editing of stem cells and other cells involved in immunity, such as T cells. These developments will add to the already burgeoning area of immunotherapy: another area of focus in regenerative medicine.

The growing importance of immunotherapy is exemplified by the Novartis, University of Pennsylvania and Oxford BioMedica ‘immunotherapy trinity,’ and technologies such as Adaptimmune’s engineered T cell therapy, which has attracted significant early-stage investment from GSK.

Creative Commons license, deviantart.com

Bass and Treble Heart by pistolmaster5555. Creative Commons license, deviantart.com

Paradoxically, it seems that ‘cell therapy’ is not all about the therapy – or all about the cells. The reason is that regenerative medicine is not just cell therapy. It encompasses gene therapy, small molecules, proteins, tissue engineering, biomaterials and probably other areas too.

Why then have we started to use the term cell therapy so liberally? The answer, we think, lies in the fact that all of these fields are intrinsically linked and interdependent. Rather than diverging as they develop, our speculation is that they will become increasingly linked and indistinguishable: even now, stem cells are used as screens for small molecule treatments and tissue engineering often takes advantage of multipotent cells, amongst many other things.

Regardless, both cell therapy as a way to understand regenerative medicine, and cell therapy more specifically, are likely to significantly impact medicine in the near future. And for those who say science writers fail to include sufficient popular culture references, perhaps “it’s not all about the bass;” but “[all about the right therapy for the job]…and the treble!”

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David Brindley
David is an international thought-leader in the translation of life-science innovations into commercially viable products and services. His expertise spans the ‘Valley of Death,’ encompassing regulation, basic science, process engineering and finance. This distinctive skill set positions David at the forefront of socially responsible investments – in particular initiatives that make impactful contributions to global health. David currently holds a joint appointment between the University of Oxford and the Harvard Stem Cell Institute and is an active Fellow of the Royal Institution of Great Britain and the Royal Society for the Advancement of Arts and Manufacturing. In addition to being an Editorial Board member of a range of international academic and industrial journals, David is also a founder of Translation Ventures, a boutique consultancy that is actively engaged in maximizing the financial and societal value realized from cutting edge scientific innovations. Disclosure: David A Brindley has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in any postings apart from those disclosed. D.A.B. is subject to the CFA Institute’s Codes, Standards, and Guidelines, and as such, the author must stress that his contributions to this site are provided for academic interest only and must not be construed in any way as an investment recommendation.
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