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Nadia received CAR-T therapy, thanks to which the girl came home and the cancer cells disappeared. In 2020, Nadia was diagnosed with treatment-resistant acute lymphoblastic leukemia. The drug that saved the girl is to be included in the list of reimbursed therapies from July onwards.
- If all goes well, CAR-T-Cells will be added to the reimbursement list on July 1
- Doctors, little patients and their parents are waiting for this moment. Until today, for a child to have a chance for this treatment, a lot of money is needed
- Once policymakers live up to their promise, despairing parents will no longer have to ask for help through online fundraising. Children suffering from leukemia or lymphoma will receive assistance under the National Health Fund
- More information can be found on the Onet homepage.
CAR-T cell therapy, a “living drug” – is a chance for life for children who cannot be effectively treated by medicine. It is also the still unfulfilled dream of doctors, little patients and their parents about reimbursement of the terribly expensive, but almost magically effective treatment. July this year may bring a positive change – in the form of entering CAR-T-Cells therapy on the list of reimbursed therapies.
At least, these are the promises made by policymakers. Everyone will be happy if CAR-T is reimbursed. Children will have access to technologically advanced therapy, and parents will be left out of fear of whether social fundraisers will manage to collect a substantial sum of over PLN 1,3 million for treatment before the disease takes the child away. Doctors will also be able to finally focus on qualifying for treatment and the treatment itself, instead of observing the growing indicator of the amount donated by people of goodwill, which determines the price of a child’s life.
All our children are ours
In Poland, only the center in Wrocław has undergone the arduous accreditation process to conduct CAR-T-Cells therapy in children.
– The truth is that since November 2019, when our center was certified to perform CAR-T therapy, we conduct this treatment, but with the enormous support of the Polish society. Even though the therapy is fantastic and it genuinely works, there are no refunds yet. Maybe from July 1 this year it will change. And let it happen – says prof. dr hab. Krzysztof Kałwak from the Clinic of Bone Marrow Transplantation, Oncology and Pediatric Hematology of the Medical University in Wrocław.
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Therapy using genetically modified CAR-T cells is intended for use in children who have no other treatment options and have exhausted all available therapies. Including transplantation or transplantation of hematopoietic (hematopoietic) cells – because it happens that in order to save a child’s life, 2-3 transplants are performed. And including the use of modern drugs such as blinatumomab or inotuzumab, which are partially reimbursed.
CAR-T therapy – a chance for life and return home
In Poland, 10–15 children can benefit from CAR-T cell therapy every year – this is not a large group of patients. These are children who have relapses after transplantation, who are initially refractory to chemotherapy, or who have a second or subsequent relapse. The indications are therefore limited; but the effects – spectacular.
– Our first patient received CAR-T cells on March 2020, 7. Thanks to the therapy, he was finally able to obtain negative residual disease 15 years after starting treatment for his very resistant leukemia. XNUMX months after the administration of CAR-T cells, they are still present in the body of this little patient and are working – says prof. K. Kałwak.
These are several million mature cells that activate, live and function on their own, and thanks to this the boy also lives.
It is said that if 6-9 months have passed without a relapse since the administration of CAR-T cells, then it should be fine.
– In the remaining group of children from Wrocław, the observation periods from the infusion are 2-7 months, but all five children who received therapy with CAR-T cells between November 2020 and March 2021 are doing well and we have molecular remission of their underlying disease, i.e. leukemia. Most of the children – except for the last patient – are at home. The side effects that were experienced by two patients were quickly resolved. So these are fantastic results, and the therapy works – literally – miracles – prof. K. Kałwak does not hide his enthusiasm for this treatment and emphasizes:
– It is possible that in the near future, thanks to CAR-T cells, our perception of leukemia treatment will change, and the therapy itself will no longer be a third-line treatment (as it is today), but it will be available in the second line, and maybe it will become the first-line treatment with time after minimal chemical preparation of the child.
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More children are waiting for a therapy with almost magical effectiveness. In two, the administration of genetically modified CAR-T cells is scheduled for June. If all goes to plan, a total of 9 patients will be treated with CAR-T cells by mid-July.
Nadia fell ill in May 2020. The diagnosis was terrifying: refractory acute lymphoblastic leukemia. Thanks to CAR-T-Cells therapy, called last-chance therapy or living medicine, the girl got a new life – free from leukemia.
A sudden deterioration in the well-being of a previously completely healthy, active child is always a cause for concern. About three weeks before the diagnosis, Nadia became lethargic, lacked energy, her feet started to ache during walks, and she drank plenty of water.
– I saw and felt that something was wrong, because my child began to look anemic. I bought vitamins, but they didn’t help. The climax that made us go to the pediatrician immediately was the moment when Nadia’s feet changed rapidly. They became very emaciated and pale, and they looked as if they were slightly distorted – recalls Katarzyna Makarczuk, Nadia’s mother.
CAR-T-Cells – a therapy tailored to Nadia’s needs
The girl’s life changed dramatically after receiving the blood count results. They indicated that the amount of hemoglobin was very, very low. The pediatrician did not wait – he wrote a referral to the hospital “na cito”.
Nadia, after a short stay in a hospital in Międzyrzecze, went to Poznań, and then to the “Cape of Hope” of the Supra-Regional Center for Children’s Oncology in Wrocław and under the care of Professor Krzysztof Kałwak.
– My daughter went to the first line of treatment – in accordance with the therapeutic protocol. But the chemistry didn’t work. After a genetic test, it turned out that Nadia was a carrier of the gene for resistance to the treatment used, which meant problems with obtaining remission of the disease and – as a consequence – the inability to transplant bone marrow from a healthy donor – explains Katarzyna.
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Then prof. K. Kałwak and her team decided to use CAR-T-Cells therapy for Nadia, which consists in giving the patient his own, but genetically modified immune cells. This treatment is not reimbursed. You have to pay for them out of your own pocket, or rather from the pockets of all people of good will, thanks to whose donations patients can receive this modern, but horribly expensive therapy.
– We did not allow ourselves to think that CAR-T-Cells is a last resort therapy and that we may not be able to collect the astronomical amount of PLN 1,305 million for treatment. It was supposed to be a therapy that would open the door to Nadia’s health and life, says the girl’s mother. And adds:
– My daughter approached the therapy very well prepared and informed. In the “Cape of Hope” she received an educational book, and there were also meetings with a psychologist. She was eager to get the cells to defeat cancer.
The moment “zero” took place on January 7, 2021. Nadia was then the fourth child in Poland and the second girl treated with CAR-T cells.
Leukemia in retreat
Although CAR-T therapy is an excellent tool in the fight against leukemia, it carries the risk of many side effects. Nadia had no complications.
She received CAR-T treatment like a regular saline infusion. Three weeks after the administration of the modified cells, the blood tests were satisfactory and we were discharged from the hospital – you can hear genuine joy in Katarzyna’s voice.
For the next quarter, once a month, Nadia showed up for bone marrow tests at the hospital. Now she shows up for a checkup every 3 months, and she has a complete blood count every week in a laboratory close to her home. The results are sent electronically on an ongoing basis to prof. K. Kałwak, who thus knows what goes on in Nadia’s body when she cannot see the girl. And it is doing really well, because no cancer cells have been detected in the marrow, but the blood count is good enough for Nadia to stay at home.
The article comes from the educational campaign “Hematology – learn about blood diseases” prepared by Warsaw Press, and whose media partner is medTvoiLokony. All materials can be found on http://www.warsawpress.com/
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