CAR-T therapies are described as the most promising direction in the development of medicine in the fight against blood cancer in recent years. According to the draft announcement of the Ministry of Health, from September 1, 2021, treatment with tisagenlecleucel of patients r / r B-ALL in certified centers will be fully financed by the National Health Fund. CAR-T therapy (tisagenlecleucel) in the treatment of refractory / relapsed B-cell acute lymphoblastic leukemia in children and adults up to the age of 25 years. inclusive was registered in Europe in August 2018.
Press material
Annually in Poland, over 1000 children are diagnosed with cancer. In most cases, the youngest respond very well to standard treatment regimens. However, there are groups of patients in whom the disease relapses or is refractory to treatment. An example of such a group of patients are patients with refractory or recurrent B-cell acute lymphoblastic leukemia, it is estimated that there are about 15 such people annually. treatment of r / r B-ALL patients.
Cape Hope’s first patient received CAR-T cells on March 3, 2020. Thanks to the therapy, it was finally possible – 7 years after starting the treatment of very resistant leukemia – to obtain a negative residual disease. Several months after the administration of CAR-T cells, they are still present in the body of a small patient and work. Since then, we have applied the treatment to several small patients who have exhausted other treatment options. These children are doing well, most of them were able to return home in a short time. This is very good information because we could not help these children in any way before. Therefore, the effectiveness of CAR-T observed in our clinical practice raises hope and gives a chance to overcome an extremely difficult opponent, which is resistant or recurrent B-cell acute lymphoblastic leukemia, where there was no such chance until recently, says Prof. Krzysztof Kałwak from the Clinic of Bone Marrow Transplantation, Oncology and Pediatric Hematology of the University Hospital in Wrocław. I am so happy that science has given us this medical miracle that makes the impossible possible. And I am even more pleased that CAR-T therapy will be available to each of our patients who qualify for this breakthrough treatment – adds Prof. Kalwak.
The positive reimbursement decision for CAR-T in the r / y treatment of B-cell acute lymphoblastic leukemia is a gift of life for young patients and their families, and at the same time an important moment for Poland, which joins the group of countries offering this unique therapeutic option of ‘living medicine’. This is a breakthrough that is taking place before our eyes – says Marta Wielondek, General Director of Novartis Oncology in Poland. Therapies such as CAR-T appear in medicine extremely rarely, every few dozen years. For Novartis, commitment to the unique research process of CAR-T cells began in 2012. The first patient to receive treatment celebrated their 9th anniversary of recovery in May this year. That is why I am so glad that in Poland, thanks to the positive decision of the Minister of Health, small patients and their families also have a chance to celebrate such joyful anniversaries. Polish doctors are among the world’s leading specialists, so Polish patients are in responsible hands – adds Marta Wielondek.
Already 4 centers in Poland have obtained Novartis certification for the use of CAR-T therapy (tisagenlecleucel): in adult patients, these are: Department of Hematology, Transplantology and Internal Diseases UCK, Medical University of Warsaw, Department of Hematology and Bone Marrow Transplantation of the Clinical Transfiguration Hospital of the Medical University in Poznań, Department of Transplantation Of Marrow and Oncohematology, the National Institute of Oncology Maria Skłodowskiej-Curie of the National Research Institute, with a branch in Gliwice, in pediatric patients with B-cell acute lymphoblastic leukemia, it is the Clinic of Marrow Transplantation, Oncology and Pediatric Hematology “Cape of Hope” of the University Hospital in Wrocław.
CAR-T wr / y B-ALL: what do clinical data say?
Results from a long-term analysis of CAR-T studies show that a sustained response is maintained in patients with advanced blood cancers. The results of the 24-month follow-up in the ELIANA study in children and young adults with B-cell ALL showed a profound and sustained response to CAR-T treatment in a significant proportion of patients without the need for further therapy. 82% of 79 patients achieved complete remission (CR) or complete remission with incomplete haematological recovery (CRi) within three months of treatment, and 98% of patients who responded had no minimal residual disease (MRD-) . Relapse-free survival at 18 months was 66%. The study did not achieve the median duration of remission (mDOR) and the median overall survival (mOS). The probability of a 12-month overall survival was 76% and an 18-month probability was 70%. The observed safety profile was consistent with previously reported results.
How does CAR-T therapy work?
How does the CAR-T therapy mechanism work in practice? In a specialized process of separating blood components (leukaferase), leukocytes are isolated from it, including T lymphocytes. Then they are sent to the laboratory for modification. With the help of a viral vector, T cells are genetically programmed to recognize cancer cells. The newly formed CAR-T cells then multiply and return to the patient’s blood. CAR-T cells programmed in this way are able to recognize cancer cells, attach to them and actively destroy them.