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World’s first clinical trial of stem cell voice box

A BRC-supported researcher and his team have been awarded £2.8m to carry out the world’s first clinical trial of a stem cell based voice box transplant.

Professor Martin Birchall, Chair of Larynogology, and his team at the UCL Ear Institute will seek to produce a safe and effective therapy suitable for routine NHS use, resulting in improved quality of life for patients and carers.

The award was one of seven made to universities, totalling 13.7m, announced by the Minister for Universities and Science David Willetts this week. It is part of the £180 million Biomedical Catalyst programme which addresses the need for new and effective healthcare solutions for a growing and ageing UK population.

The larynx (voice box) protects the airway during swallowing, regulates breathing, and enables voice: all fundamental human functions. Over 2,000 UK patients a year lose laryngeal function due to trauma or cancer and 1,300 NHS patients a year have their larynx removed entirely.

Conventional treatments for these patients leave many with substantial problems talking, swallowing and breathing. For example, the use of combined chemotherapy and radiotherapy for laryngeal cancer results in hoarseness and painful swallowing and can even render the larynx completely disabled.

Being able to accurately replace the normal contours and structure of the larynx, for example by using a living tissue-engineered replacement, would transform the quality of life (and in some cases survival) of these patients.

In 2008, Professor Birchall co-led the pioneering research team which carried out the first transplant of a human windpipe (trachea) reconstructed using stem cells. Now, MRC Biomedical Catalyst funding is helping him to build on this success by developing the first clinical trials of a stem-cell-derived larynx transplant; a project known as RegenVOX.

The RegenVOX procedure involves preparing a reconstructed larynx, made from the patient’s own stem cells and a donor larynx. In the lab, the team uses chemicals to remove the cells from the donor larynx leaving behind a scaffold, onto which stem cells from the recipient can be grafted. This means that the finished implant will not get rejected, like normal transplants, so patients do not need to take immunosuppressant medication.

The team is also able to turn the patient's stem cells into cartilage-producing cells to give natural strength to the transplant, and into replacement mucous membrane cells to line the inside, just like a normal larynx.

The MRC previously funded the preclinical development of RegenVOX, and with this latest grant Professor Birchall hopes to carry out the first transplant procedures in around a year. The research team will then follow ten patients for two years to demonstrate that the procedure is safe and effective.

They will also evaluate the economics of moving treatments like this into routine healthcare, and determine the most cost-effective ways this can be managed, as well as ensuring the UK economy benefits from the potential value of this opportunity. The project will run for just over four years.

Professor Birchall said:

“Losing the larynx can be devastating as it impacts on so many of the qualities that make us who we are. Patients not only lost their voice, but their breathing, swallowing, sense of smell, the ability to cough and even kiss are all affected.

“The possibility that we may soon be able to give these patients back their voice and vastly improve their quality of life is incredibly exciting.

“I’m very hopeful that our success in this field will lead the way for other, related, stem cell-based organ replacements in the future, such as for the oesophagus and even lung.”

Announcing the awards today at the innovation event, InnovateUK, David Willetts said:

“The Biomedical Catalyst is making a real impact by making sure that our innovative businesses in the UK are able to develop new products for the healthcare industry.

Many great innovations often fall into the ‘valley of death’ between the creation of an idea and the market place. The Catalyst is helping the UK to bridge that gap, so that the best new ideas in healthcare can be transformed into innovative products and services.”

The scheme is a part of the Government’s Strategy for UK Life Sciences, which supports the translation of an idea from concept to commercialisation and a number of the projects announced today involve late-stage human trials of new healthcare solutions.

Among the projects winning funding are: a novel drug for treating multiple sclerosis; the world’s first clinical trial of a stem cell based voice box transplant; an innovative low-cost implantable blood pump for advanced heart failure; gene therapy for a genetic visual disorder; and a new therapeutic approach to controlling the immune system in infectious disease.