Resolving quality, standards and productivity related challenges of a ground breaking regenerative therapy for heart failure

Heart failure is when the heart cannot efficiently pump blood around the body due to damage to the heart muscle. As a result, important organs (such as the brain and lungs) do not get enough blood and patients suffer from tiredness and breathlessness and are unable to exercise. As the heart failure progresses, patients experience an ever-decreasing quality of life and an increased dependence on hospital services. The number of heart failure patients continues to increase and, despite new treatments, many remain symptomatic as current treatments do not repair the heart muscle (thereby enabling it to pump blood efficiently again). For these patients, the only way to alleviate these symptoms is a very rare, and highly invasive, heart transplant.

The applicants originate from the world-renowned Barts Heart Centre, where this team has developed and tested a cell therapy which uses stem cells taken from the patient's own bone marrow. Once processed, these cells are infused into the heart to help repair the heart muscle (thereby enabling it to pump blood more effectively) and improve the patient's symptoms. A charity, the Heart Cells Foundation, was formed to promote the adoption of cell therapy for heart failure. This charity raises hundreds of thousands of pounds annually, and has created a Compassionate Treatment Unit. The team has treated over 500 heart failure patients, who were told they had no more treatment options, with their own stem cells.

The Heart Cells Foundation wishes to commercialise this treatment so it is more widely available nationally and internationally. To lead this commercial step, they formed the Heart Cells Company (HCC). HCC needs approval from regulatory agencies in order to secure the investment needed for a large trial (which could lead to market authorisation - required before the NHS will adopt the treatment). However, regulatory agencies are unwilling to grant HCC approval until it has a more definitive measurement to assess the quality of the cell product.

HCC is seeking support from the Analysis for Innovators (A4I) competition to develop this measurement technique (commonly called an assay) to assess the cell product's efficiency and quality. This assay will also enable HCC to improve and research the treatment method to make it more effective in the future.

This A4I project will develop this efficiency and quality assay by analysing samples of bone marrow taken from heart failure patients participating in ongoing clinical trials at Barts Heart Centre.