A woman with a neuroblastoma-a type of nerve cell cancer-, which as a child was treated with a car-T cell therapy, has managed to send the tumor for more than 18 years and without the need to receive additional treatments.
This is the a longer duration remission after such a therapy described to date.
This patient participated in a phase 1 clinical trial to test a modified car-T therapy to treat neuroblastoma, a type of cancer that can be of poor prognosis.
The authors of that essay, conducted with 19 children between 2004 and 2009 to test this CAR-T therapy, have monitored long-term and this Monday publish the results in an article in the journal Nature Medicine.
CAR-T cell therapy is a treatment that modifies a patient’s T cells-a white blood cell type that is part of the immune system-to specifically recognize and eliminate cancer cells.
This therapy has been approved for the treatment of patients with some types of blood cancer, such as leukemia and lymphoma, but has been less effective in patients with solid tumors.
Neuroblastoma is a rare solid type of solid tumor that usually affects children and is a difficult disease to treat, with high relapse rates despite treatment.
An essay with 19 children
The Baylor College team, led by Helen Heslop, carried out a phase 1 clinical trial with 19 children with neuroblastoma, between 2004 and 2009, in which they tested modified T cells to recognize GD2, a protein with high levels of expression in expression in The neuroblastoma.
Eleven of these patients had active disease.
Although phase 1 trial determined that the treatment was safe, twelve patients died between 2 months and 7 years after treatment, due to a relapse of the neuroblastoma.
Of the remaining seven patients, five continued in follow -up for at least 13 years after treatment.
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Heslop and his colleagues observed that cancer of one of the patients has been in remission for more than 18 years without receiving other oncological treatments. In addition, this woman has given birth to two healthy babies.
They also found evidence that the CAR-T cells persisted for at least 5 years in five of the treaties, including the patient with 18 years of remission.
The equipment points out that the CAR-T cells used in that trial lack the design elements of modern car-t cells, which now include coestimulatory molecules, and it is possible that patients with active disease at the time of treatment have not been benefited as much as those without evidence of illness or with a load of less disease at the time of treatment.
These data suggest that CAR-T cells have potential to provide long-term benefits to patients with solid tumors, and also provides biological knowledge about the behavior of CAR-T cells that could be informative for other studies.
The opinion of the experts
In statements collected by the SMC Spain, Marta María Alonso Roldán, a researcher at the Solid tumors program at the top and the University of Navarra clinic (northern Spain), stressed the importance of the study, although she lamented the reduced number of patients participating in The essay.
And although this type of first generation car is no longer used, it provides data on long survivors, “of which we still have much to learn.”
For Luis Álvarez-Vallina, of the Mixed H12O/CNIO Cancer Unit, these data “demonstrate the security of the strategy and suggest that CAR-T cells could provide long-term benefits in patients with some types of solid tumors.”
However, he warned that to validate the therapeutic impact of CAR-T therapies “it is essential to have broader series.”
For Ignacio Melero, Professor of Immunology at the University of Navarra and CIMA researcher, “it is good news that patients treated with car-t cells have a sustained clinical benefit over time”, especially if it is thought that “Car -T that were used a decade ago in Baylor College of Medicine for the treatment of neuroblastoma have been very improved today. ”
“Neuroblastoma is a fearsome pediatric solid tumor, and that the duration of the referrals obtained with the treatment with anti-GD2 car -ts can be so long is an excellent news for the immunotherapy community,” concluded the researcher.
With EFE information.
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