FDA approves cell therapy to reduce GVHD risk after transplant

The US Food and Drug Administration has approved a new cell therapy, Tregzi, for patients with haematological malignancies undergoing allogeneic bone marrow or haematopoietic stem cell transplantation. The FDA’s decision has been one of the most significant developments in the fields of transplantation and cell therapy in recent years. For patients undergoing stem cell treatment, the main concern following transplantation remains not only the risk of disease relapse, but also the development of severe immune complications, primarily graft-versus-host disease.

What is ‘graft-versus-host’ disease?

‘Graft-versus-host’ disease occurs when the donor’s immune cells begin to attack the recipient’s tissues. The skin, liver, intestines, mucous membranes and lungs are most commonly affected. In severe cases, this complication can lead to prolonged hospitalisation, disability or death. Despite advances in transplantation techniques, the risk of developing GVHD remains one of the main limitations in the use of donor stem cells. Existing prophylaxis regimens are based primarily on the use of immunosuppressive drugs, which simultaneously reduce the activity of the immune system and increase the likelihood of infectious complications.

What is Tregzi cell therapy?

The new Tregzi therapy has been developed by Orca Bio and is a cell-based product comprising carefully selected donor immune cells and stem cells. Unlike standard transplantation, the technology involves separating the various cellular components and precisely dosing them before administration to the patient. Regulatory T-lymphocytes play a key role in the composition of the product. These cells help regulate the immune response and are capable of suppressing excessive activity of the immune system. It is these cells that reduce the likelihood of the donor’s immune system attacking the recipient’s body following transplantation.

Which patients is the new treatment intended for?

The FDA has approved Tregzi for adult patients with acute myeloid leukaemia, acute lymphoblastic leukaemia, myelodysplastic syndrome and a number of other blood disorders for which allogeneic haematopoietic stem cell transplantation is indicated. This primarily concerns patients at high risk of disease relapse or for whom standard treatment regimens have been ineffective, where transplantation remains the only option for achieving long-term remission. The therapy is used in transplants from HLA-compatible related or unrelated donors. The use of a specially prepared cell product makes it possible to preserve the anti-tumour activity of the donor’s immune system whilst reducing the likelihood of damage to the patient’s healthy tissues.

The registration was based on the results of the PRECISION-T trial. The trial involved patients who had undergone transplantation from compatible donors. The data demonstrated a reduction in the incidence of severe graft-versus-host disease compared with standard transplantation approaches. An additional benefit was the faster recovery of the immune system following transplantation and a reduction in the number of severe infectious complications, which often accompany long-term immunosuppressive therapy. Furthermore, some patients were able to discontinue maintenance immunosuppressant therapy sooner after transplantation.

The significance of the FDA’s decision for the development of cell therapy

The approval of Tregzi confirms a shift in approaches within modern transplant medicine. Whereas previously the main focus was on donor selection and drug regimens to prevent complications, today the emphasis is gradually shifting towards the precise tuning of the cellular composition of the transplant. In effect, this represents a shift from the standard transfer of donor cells to the management of the transplant’s immune composition even before it is administered to the patient. This approach makes it possible to influence, in advance, the balance between the anti-tumour effect and the risk of immunological complications.

Experts regard technologies based on regulatory immune cells as one of the most promising areas of development in personalised medicine. Similar approaches are already being studied in the context of autoimmune diseases, organ transplantation and a number of rare immunological disorders. The FDA’s decision may also accelerate the development of new cell-based products aimed not only at treating the underlying disease but also at preventing treatment-related complications. For the biotechnology sector, this means expanding the application of cell-based technologies beyond oncohaematology.

Patient support and treatment coordination

Eternity Life Tourism specialises in organising programmes in the fields of regenerative medicine, cell therapy and treatments utilising cutting-edge biomedical technologies at leading international clinics. The company’s specialists support patients throughout all stages of preparation for treatment, assisting with the selection of a medical facility and the organisation of their trip. Eternity Life Tourism collaborates with specialist medical centres and clinics working in the fields of cell therapy and transplant medicine. If you are interested in more detailed information about modern treatment methods, including programmes utilising cell technologies and stem cells, the company’s specialists are ready to provide advice and assist with organising treatment abroad.

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