How to make regenerative medicine happen

Research published by the UK’s Cell Therapy Catapult earlier this year shows there are now 41 regenerative medicine products in clinical trials in the UK, up from 34 in 2013.

These products embody both significant health benefits and the opportunity to build global markets. Keith Thompson, chief executive of the Cell Therapy Catapult, set up to help realise the health and wealth potential of regenerative medicine, is convinced a shift is on the way. “Traditional medicines based on small molecules have dominated up to now, but stem cells are really the next big thing,” he told Science|Business.

Cell therapies that can prompt the regeneration and repair of damaged and ageing tissues are being developed to treat a range of diseases, many of which are largely untreatable at present, including spinal cord injuries, heart disease, the after-effects of stroke, certain forms of blindness.

Further off, but now within the bounds of reality, is the prospect of growing new organs for transplant.

However, the majority of cell therapies are still in the early stages of development , and it is likely to be some time before there is a range of marketed products. “It’s the Cell Therapy Catapult's job to make the industry happen,” said Thompson. “We are here to lower the barriers and generate projects that make a good investment proposition.”

The 70 staff at the Cell Therapy Catapult offer expertise across the board, helping companies embark on clinical development, and providing technical, manufacturing and regulatory expertise.  The Catapult also provides access to clinical trial infrastructure and patients within the National Health Service. There is a focus on collaboration and lowering barriers to investment and funding. “All drug development is a risk-adjusted bet,” Thompson said. “Our objective is to get the bet [to the point where it is] more likely to succeed.”

The Cell Therapy Catapult, funded by the UK Technology Strategy Board, was founded in 2012, and is based at Guy’s and St Thomas’ Hospital  in London. “We have £70 million for the first five years and we also hope for contract research from industry, which could be £10 million extra,” Thompson said. In addition the Catapult will bid for grant funding as part of research consortia.

In May, the Catapult was awarded a further £55 million to build a facility for manufacturing cell therapies for late stage clinical trials and commercial supply.

Commercial prospects

There is potential for a multi-billion industry, with new companies entering the space and pharmaceutical companies beginning to take an interest.

In the UK the pipeline now consists of 86 projects in late preclinical development and in clinical trials. While the US has a greater number of trials, “The UK is certainly in a leading position, and its breadth is sufficient to think some will come through.”

Although working predominantly with UK companies, the Catapult is also becoming a handy touch point for foreign firms. “Companies in the US and Japan in particular see doing trials in Europe as complex; they like to work with us,” he said.  As an example, the Catapult is collaborating with the US company Athersys to progress its MultiStem cell therapy product in Europe. Among other projects, the Cell Therapy Catapult will carry out a European market access assessment, considering factors such as data requirements for regulators, payers and health technology assessment bodies, value perceptions and pricing benchmarks.

MultiStem is an off-the-shelf stem cell product in development for the treatment of cardiovascular, neurological, inflammatory and immune system diseases.

Selling cells

The late-stage development and the manufacture of cell therapies is highly technical and complex. As yet, there is “little evidence yet that new cell therapies can be developed, licensed and adopted successfully,” according to the Catapult’s website. Investment from the corporate sector is limited; larger companies are holding back and waiting.

In its first collaboration, the Catapult worked with one of Europe’s leading stem cell companies, ReNeuron on the development and optimisation of the processes required to scale-up manufacture of the company’s cell line, including rapid cell culture techniques, cryopreservation and automated manufacturing processes.

Cryopreservation to extend the shelf life of its products has removed an important barrier to commercialization for ReNeuron. In the first clinical trial the company ran, in treating the after-effects of stroke, fresh cells had to be produced for each patient, and these were only viable for eight hours.  The cryopreserved product has a shelf life of at least one month.

Cryopreservation has also cut the cost of goods, paving the way for Reneuron to set up its own manufacturing facility in South Wales, with funding from the Welsh government.

The Cell Therapy Catapult is also working with University College London and Imperial College London to develop a cell therapy for treating leukaemia. As an academic project, “It wasn't progressing as fast as it could, so we formed a spin-out company with the academic partners. We’re putting up £10 million for trials and looking for long-term partners,” Thompson said.

In addition, the Catapult has signed collaborations with GlaxoSmithKline, details of which have not been disclosed, and with Smith and Nephew, where it is helping with requirements for regulatory and market approval of a spray-on wound healing product consisting of skin cells.

For a sector so young, any success is relative and Thompson is realistic about what can be done within the scope of the initial five year funding. “The mission, and our mandate, is to help grow a significant industry for cell therapies over five years and beyond.” he said. “Five years is a short time in drug development but we hope we’ll get some products over the finish line; we’ll get others close.”