Researchers are one step closer to large scale trials of a cell therapy for acute myeloid leukaemia, after a positive review of a BRC-supported Phase I/II study.
In the trial, patients’ T cells are modified by gene therapy so the T cells recognise and destroy the WT1-expressing cells associated with acute myeloid leukaemia and myelodysplastic syndrome.
The review by the Data Safety Monitoring Board (DSMB) found the data showed no serious adverse events related to the cell therapy in the first cohort of three patients treated. The therapy also met requirements for cell persistence, showing that the targeted T cells can survive and expand in patients.
Passing this first interim review by the DSMB independent panel has enabled the clinical trial to enter its second phase. Dosing has now started in the second cohort of patients in the trial which allows patients to receive a higher dose of the gene modified WT1 T-cell receptor (TCR) cells.
BRC Programme Director Professor Emma Morris, Chief Investigator and co-inventor, said: “We are delighted with the progress of this clinical trial in acute myeloid leukaemia. This is an important area of unmet medical need and we are progressing with the next stages of recruitment now ongoing in the second cohort of the acute myeloid leukaemia trial.”
The trial is being operated by Catapult Therapy TCR, a company formed by the Cell and Gene Therapy Catapult with UCL Business and Imperial Innovations. The gene modified immune cells are currently being manufactured by a joint UCL-Great Ormond Street Hospital team of scientists.
Keith Thompson, Cell and Gene Therapy Catapult Chief Executive Officer, said: “Developing TCR gene-modified T cells to target WT1-overexpressing cells continues to show promise as an immunotherapy for a range of cancers including the trials that we have underway in acute myeloid leukaemia and myelodysplastic syndrome. The progress of the WT1 TCR clinical trials, supported by the positive DSMB, is encouraging and we look forward to expanding these trials across the UK and Europe. Further results are expected during 2017.”
Tony Hickson, Managing Director of Technology Transfer at Imperial Innovations, said: “We are pleased to note the progress of this innovative therapy. Catapult Therapy TCR is a great example of collaboration between UK universities and technology transfer organisations and we look forward to the results of forthcoming trials.”
Pre-clinical development of the TCR gene therapy programme was supported by Bloodwise, previously Leukaemia and Lymphoma Research. In August 2015 the Cell and Gene Therapy Catapult appointed Cellular Therapeutics Limited as a manufacturer for the clinical trial, who will take over cell manufacture next month.
