$4.4 Million grant: California stem cell agency funds UC Davis - UCL tissue-engineering research

2 January 2014

Prof Birchall
Professor Birchall at the UC Davis press conference with laryngeal transplant recipient Brenda Jensen in the background
Prof Birchall and Dr Belafsky were members of the surgical team that provided her with the worlds second successful laryngeal transplant, and both are co-investigators on the new $4.4 million dollar grant to study tissue engineered larynx and trachea

(SACRAMENTO, Calif.) — Led by two renowned throat surgeons and two highly regarded stem cell researchers, a joint UC Davis-UCL team has just received a $4.4 million grant from the California Institute for Regenerative Medicine (CIRM) to answer key scientific questions relating to the development of stem cell-driven airway transplants. The funding was part of the agency’s Disease Team grants, which are designed to bring stem cell solutions to patients with unmet medical needs through late preclinical science and clinical trials.

With the grant, Peter Belafsky, professor of otolaryngology and principal investigator, Alice Tarantal, professor of paediatrics and cell biology and human anatomy, together with UCL investigators Martin Birchall, Professor of Laryngology, and Mark Lowdell, Director of Cellular Therapy at Royal Free London NHS FT and UCL, will work to develop the underpinning science for a safe and effective tissue-engineered treatment for patients suffering from the critical narrowing of the Airway (stenosis). This US research will provide fundamental under-pinning to the two clinical trials in the UK funded by MRC and the Technology Strategy Board in which human stem cell-derived larynges and tracheas will be manufactured by Dr Lowdell’s for transplant by Professor Birchall’s team. It is expected that the results from the CIRM-funded program and the two UCL phase I clinical trials which start in 2014 and 2015 will allow rapid technology transfer of the processes from UCL to UC Davis for formal US clinical trials in 3 to 4 years.

Dr Lowdell said, “this can’t have come at a better time as we are just expanding our capacity to manufacture these complex cell-based medicines thanks to a £2.4m investment by the Royal Free Hospital and it capitalizes on the £5m investment by the MRC and TSB in our programme. This is precisely the sort of UK-US collaboration which will allow the UK to remain at the forefront of regenerative medicine as proposed by the UK Government. The future supply of these therapies to the wider population will require commercialization of manufacture and we are already working closely with industrial colleagues to make this happen once the safety, efficacy and underpinning science have been established.”

Severe airway stenosis is a life-threatening problem, which can result from head, neck or throat cancers, as well as from trauma or rare physical conditions. According to Birchall and Belafsky, it occurs in approximately 200 people annually in each of California and the UK, and profoundly affects a person’s quality of life because it obstructs breathing and communication. Surgery, the current standard of care, can include use of tracheotomy tubes and stents, which are highly invasive, frequently providing less-than-satisfactory results, and can cause infection, pain, and voice loss.

“There are a number of scientific questions that we plan to explore in the next few years,” said Tarantal,. “We will develop and refine optimal transplantation techniques as well as determine the fate of implanted stem and progenitor cells for tissue-engineered airway implants and study biomechanical changes and angiogenesis. Firmly establishing the safety and efficacy, as well as the scientific mechanisms, of this process is critical to the goal of a transformative treatment for human patients.”

Belafsky noted that the team is uniquely positioned to address many of the clinical and scientific questions involved in regenerative medicine and tissue engineering. It already has experience in successfully using stem cell-based tissue engineered tracheal implants. And in 2010, a team co-led by Belafsky and Birchall successfully performed the world’s second documented larynx transplant. The team’s new research effort could yield results that have benefits well beyond addressing patients with a life-threatening narrowing of airway.

“The knowledge gained from our preclinical studies could easily provide us with a window into a new technology that can be applied not only to airway stenosis, but to disorders affecting other patient groups and other hollow organs as well,” said Birchall. “Stem cell-derived tissue engineered techniques could be extended to treat children who suffer from severe congenital conditions that surgery cannot cure.”

“Stem cell-derived airway transplants or bioengineered stents also might be used to treat adults with severe chronic obstructive pulmonary disease,” added Belafsky. “The methods and technology developed with our project could also be used to treat health disorders that require esophageal, bladder or bowel replacement, where the current standards of care remain very limited and impair quality of life.”

Page last modified on 02 jan 14 15:14