A pioneering new treatment for cancer, whereby a patient’s blood cells are currently sent to the United States to be genetically modified before being returned to the patient a few weeks later, can now be carried out entirely in the University Medical Center Groningen. It will no longer be necessary to send the blood to the USA, which will not only save time but will also improve the quality of the treatment and be considerably faster. Zorginstituut Nederland (The Dutch National Health Care Institute) and ZonMw (the Netherlands Organisation for Health Research and Development) have awarded a €30 million grant the research at the UMCG, which must now show whether this version of the treatment is as successful as the ‘USA route’ and does, indeed, generate the expected savings.
Since the end of last year, patients with lymphoma, who have exhausted all other treatment options, have been successfully treated by having their own T cells genetically modified outside their body. At around €330,000 per patient, the treatment, available at a few UMCs in the Netherlands, is extremely expensive. This is because the process is lengthy and complicated.
T cells (a type of white blood cells) are extracted from the patient’s blood. These cells, which play an important role in the body’s natural resistance to cancer cells, are genetically engineered in a laboratory by adding a so-called CAR to the T cells. This CAR is an extra piece of DNA that allows the T cells (which are called CAR-T cells after the modification) to recognize and attack cancer cells. In other words, the patient’s own immune system is primed to eliminate the cancer cells.
The UMCG can now produce CAR (CAR stands for chimeric antigen receptor) in its own hospital pharmacy. This saves valuable time: at present, the entire procedure, including transport to and from America, takes an average of 4 to 6 weeks, time that many of these patients do not have. This can now be reduced to just under two weeks. As well as saving time, the researchers expect that the quality of the treatment will improve too. As the white blood cells can now be harvested and modified in the same place, work on fresh cells can begin immediately, dispensing with the need to freeze them for transport. The financial saving is also important: treatment with CAR-T cells produced in the UMC’s own laboratory is expected to cost around €80,000 per patient. This compares with €330,000 per patient for treatment with commercially produced CAR-T cells. The research is a joint project with Radboud University Medical Center, Erasmus Medical Center and Amsterdam UMC. Patients from Rotterdam and Amsterdam will also be treated with CAR-T cells from Groningen.
The head of the research programme, internist-haematologist Tom van Meerten from the Department of Haematology at the UMCG, describes the research as ‘A real game-changer. These are patients with no further treatment options, who will probably deteriorate during the waiting time. Around 40% of the patients who have received CAR-T cell therapy are still in remission two years later. This is a very long time, compared with other types of cancer and cancer treatments. If the teaching hospitals can produce their own CAR-T cells, the whole process will be much better and cheaper.’
Until recently, this was an experimental treatment. But it has proved so successful that, since early this year, it is given to all eligible patients with lymphoma. Nearly half of them are in remission. The treatment also seems to be promising for patients with other types of cancer but more studies are needed to prove this.
The CAR-T cell therapy method is in its infancy. Only a few medical research centres worldwide are using and studying the method. More patients are expected to be eligible for this treatment in the future. In fact, it will possibly be the first option, and not only used if other treatment options (such as chemotherapy) fail. Research like this is very difficult to fund without a grant. The grant will cover the high costs of treatment while the long-term research is being carried out. This is usually the bottleneck in funding research into innovative care. The insurance companies will continue to pay for the standard treatment being used as a comparison in the research.
The six-year research programme is monitoring 299 patients. Once the programme has finished, the Zorginstituut will decide within six months whether the treatment has proved to be effective and whether it should be paid for via basic medical health insurance.
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