Frontiers of Knowledge Award Goes to Scientists Who Revolutionized the Treatment of Several Types of Blood Cancer
American oncologist Carl June and French geneticist Michel Sadelain have been awarded the Frontiers of Knowledge Award for revolutionizing cancer treatment through immunotherapy based on so-called CAR-T cells. Their work has paved the way for therapies therapies for patients with various types of blood cancer, including leukemia, which have already benefited “tens of thousands of individuals, including many children,” according to the award committee’s citation.
The innovative therapeutic strategy developed by the laureates of the BBVA Foundation Frontiers of Knowledge Award in Biology and Biomedicine involves extracting T cells from the patient's own immune system, growing them in the laboratory, and genetically engineering them to recognize and selectively destroy cancer cells.
Once reinfused into the body, these modified cells can induce remission of the disease.Already used to treat various types of blood cancer, this method is now being developed for use against solid tumors, including breast, prostate, colon, and pancreatic cancers, as well as in the treatment of “infectious and autoimmune diseases.” The committee’s citation concludes that this research is driving a vast and highly promising field of biomedical inquiry that “is revolutionizing cell therapy through genetic engineering."
More Than 50,000 People Have Benefited From This Treatment
After its successful clinical application in 2010 on two terminally ill leukemia patients, the first CAR-T cell therapy was approved by the U.S. Food and Drug Administration (FDA) in 2017 for use in children and young adults with refractory acute leukemias and certain refractory lymphomas. One year later, the European Medicines Agency (EMA) also authorized its use across the European Union. To date, more than 50,000 patients with blood cancers have been successfully treated. “Today it is widely accepted that genetically engineered immunity, exemplified by CAR-T cells, can succeed against these types of cancer where no other treatment, chemotherapy, or bone marrow transplantation had previously done so,” Sadelain emphasizes.
So far, CAR-T cells have not achieved the same level of success in solid tumors, such as breast, colon, pancreatic, or lung cancers, as they have in patients with blood cancers. This is the next major challenge in genetically engineered immunotherapy—and one both award-winning scientists are actively pursuing.
“If the same approach is applied to solid tumors, CAR-T cells do not work as effectively as they do in leukemia and lymphoma,” Sadelain acknowledges. Both researchers, however, are confident it's only a matter of time: “Thousands of laboratories around the world, including my own, are working to make CAR-T cells effective in solid tumors, and I am convinced that we will succeed,” June affirms.
Potential in Autoimmune and Infectious Diseases
As the committee's citation highlights, the potential of CAR-T cells extends well beyond cancer, reaching into autoimmune disorders and infectious diseases. “Remarkably,” Sadelain notes, “very strong responses were achieved immediately in patients with lupus.” This has sparked “a wave of new clinical studies in the fields of rheumatology and neurology,” aimed at exploring whether CAR-T cells can help control autoimmune diseases such as arthritis and multiple sclerosis.
Other recent studies have also shown that the same strategy may prove beneficial in combating certain refractory infectious diseases such as AIDS. Research is currently underway to explore the possibility of using CAR-T cells to treat HIV infection “in such a way that all viral reservoirs can be eliminated and a complete cure truly achieved, without the need to continue taking medication,” June explains.
The same strategy has also been investigated in other infections such as COVID-19, and several recent studies suggest it could even prove useful in certain cardiac and pulmonary disorders.