The House of Lords inquiry into regenerative medicine: mapping the UK route for the commercialisation of cell therapies

The
UK House of Lords Science and Technology Select Committee (1) has launched an
inquiry into cell therapy and regenerative medicine to take place this autumn. The
focus of this inquiry will be whether the UK is sufficiently well placed for the
successful translation and commercialisation of research into patient-ready
treatments. The inquiry will also ask whether the UK can integrate its efforts
into facilitating this process.

The
Select Committee analyses Government policy on a wide range of topics relating
to science and technology, including a variety of public policy areas. The
Committee currently has 14 members from across the political spectrum and is
chaired by Lord Krebs, the acclaimed zoologist.

This
is not the first time that the House of Lords has examined issues in this
sector. In 2002, a Select Committee on Stem Cell Research published a notable
report on matters relating to stem cell research and human cloning. The latest
inquiry, however, is broader and more focused on translation and more
specifically commercialisation than research. It also reaches beyond stem cells
to the wider cell therapy and regenerative medicine sectors.

'Regenerative
medicine' is the replacement or regeneration of human cells, tissues or organs
to restore or establish normal function (2). It includes cell therapies, gene therapies, tissue engineering and biomedical engineering techniques.

A
large amount of world-class research and development in regenerative medicine
takes place in the UK, including work on cell therapies, on small molecules to
trigger in vivo responses to disease,
and early developmental-stage research. Internationally, there is a wealth of
regenerative medicine and cell therapy developments underway. This fierce pace
of development has advanced the field but also revealed new challenges
including how best to commercialise the technology. In this context, the focus
of the inquiry on regenerative medicine, and how best to facilitate UK
translation and commercialisation, is timely.

Significant
developments in the UK over the past few years have improved the funding
environment for those working in this sector. The recent establishment of the
Technology Strategy Board (TSB)-backed Cell Therapy Catapult at Guy's
Hospital in London, and the announcement of a £25 million cross-research
council UK Regenerative Medicine Platform fund are two prime examples. In
addition, the £180 million TSB/Medical Research Council Biomedical Catalyst
Fund launched earlier this year and the £650m interdisciplinary medical
research initiative (the Francis Crick Institute) are both likely to include
regenerative medicine-focused translation projects within their remit.

However, the later stages of the process from lab bench to patient
bedside remain particularly important. Gaining regulatory approval, negotiating
reimbursement with healthcare providers, improving access to patients, and
establishing long-term clinical uptake all pose challenges.

Regenerative
medicines face unique issues with regards to many of these steps. For example,
clinical trials  that require large numbers of identical batches of cells to be
used in treating patients present challenges in relation to proven reproducible
manufacturing. In addition, the regulatory approval for a clinical trial will often be
such that it allows treatment of patients only in the late phase of their
condition. This often results in the trial therapy being tested on the target
condition but with associated complex multisystem pathology and thus little
real opportunity to demonstrate significant patient benefit.

At
the same time, therapies need to remain cost-effective to garner recommendation
from the National Institute for Health and Clinical Excellence (NICE). Making
such highly-personalised medicines, such as autologous cell therapies,
available on the NHS, is essential for their reaching large numbers of patients.
Then, of course, their price has to be sufficient to ensure commercial
viability.

The
Select Committee inquiry presents an excellent opportunity for those involved
with and interested in the sector to express their views and help shape the
future of policy and regulation in this field. The Committee has published a call for evidence inviting written
submissions by 20 September 2012. The
call covers topics including the research base, the application of science,
barriers to translation and commercialisation, and international comparisons.

With
the UK being a world leader on cell therapy development, stem cell research and
personalised medicine, we now have the opportunity to identify and resolve the
bottlenecks in the translation and commercialisation process, and move
efficiently towards economic growth from the sector. It is to be hoped that there will be broad
and enthusiastic support for this Select Committee as it will be the UK's last
chance to become a global leader in the commercialisation of cell therapies.