An experimental immune-cell treatment has put a German teenager with advanced kidney cancer into complete remission, offering new hope that engineered T-cell therapies could eventually be used against some difficult solid tumors.
The boy was diagnosed with kidney cancer at the age of seven. After years of treatment, he was able to return to a relatively normal life and developed a passion for cycling. However, by the time he was 17, the cancer had returned and spread to several organs, including his brain.
Doctors at the Hopp Children’s Cancer Center in Heidelberg believed conventional treatment options had been exhausted. Dr. Christian Seitz and his colleagues then identified markers in the boy’s cancer that could potentially be recognised by specially engineered immune cells.
The team used a treatment known as T-cell receptor-engineered therapy, which modifies a patient’s T-cells so they can identify and attack cancer. Unlike some conventional CAR T-cell treatments, this approach targets proteins located inside cancer cells rather than proteins displayed on their surfaces.
The cancer carried a protein known as PRAME, which has attracted scientific interest as a possible target for immune-cell treatments in several cancers, including melanoma and sarcoma.
Researchers worked with German biotechnology company Immatics to develop the specially programmed T-cells for the teenager.
The treatment was difficult for the patient. He spent around two weeks in intensive care following the infusion. But eight weeks later, doctors were stunned by the results.
Scans showed that cancerous growths in his liver had disappeared, while tumors elsewhere had dramatically shrunk. Tests of tumor tissue found no evidence of living cancer cells.
More than a year after receiving the single infusion, the teenager still has no detectable signs of cancer, according to Seitz. T-cells capable of recognising PRAME also remain in his bloodstream, raising the possibility that they could help attack future cancer cells if the disease returns.
The findings were published on August 12 in the New England Journal of Medicine.
Engineered T-cell therapies have transformed treatment for some blood cancers, but solid tumors have generally proved much harder to treat. Researchers believe the environment surrounding solid tumors can prevent immune cells from effectively reaching and attacking cancer cells.
Seitz said the results challenge the long-held view that large solid tumors are generally unsuitable for engineered T-cell treatment.
The discovery does not mean the therapy will work for every patient. Researchers still need to determine how effective and safe it is across larger groups.
Seitz is now preparing a clinical trial involving 18 children whose tumors contain PRAME. The trial could help establish whether the treatment can produce similar results in other young cancer patients.
For the teenager who received the experimental therapy, the treatment has already allowed him to return to the activity he loves. Seitz recently learned that the boy had taken part in an extreme cycling event involving steep climbs and fast descents through rocky terrain.
After years of battling cancer, the teenager is once again living the active life he had fought to regain.
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