A successful clinical trial in Stanford Medicine showed that an immune-cell therapy shrank children’s brain tumors, restored neurologic function, and, for one participant, even cured brain cancers normally considered incurable. Also erased traceable traces.

The trial – one of the first successes against solid tumors for engineered immune cells known as CAR-T cells – offers hope for children with a group of deadly brain and spinal cord tumors, including diffuse intrinsic pontine. Also includes a cancer called glioma or DIPG.

The findings of the study, published in the journal Nature, have received Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration, which gives researchers access to a fast-track version of the approval process.

One of the first successes of CAR-T cells against tumors

This trial has been one of the first successes of CAR-T cells against solid tumors. DIPG, a rare and aggressive brain cancer, has a five-year survival rate of less than 1 percent, with most patients living only about a year after diagnosis.

The trial included 13 participants with DIPG or similar brain and spinal cord tumors. Nine of the 11 who received the treatment showed significant benefits, including improvements in physical abilities and a reduction in tumor size. However, one patient experienced a complete response – as his tumor disappeared. The person remains healthy even four years after diagnosis.

“This disease is usually fatal, but this therapy has shown meaningful tumor reduction and clinical improvement,” said Dr. Michel Monje, the trial’s lead researcher. “Although there is still work to be done, this one case gives us hope.”

The treatment was designed to target GD2 – a marker found in DIPG cells.

Even though the therapy caused some initial side effects such as fever and swelling, patients showed remarkable progress. The only person who had a complete response was diagnosed at age 16 and has now regained many of the abilities lost to the disease, including walking without assistance. He is now studying forestry in college.

What are CAR-T cells?,

According to experts, CAR-T cells are engineered from the patient’s immune cells to target cancer. Previously successful in blood cancers, this trial tested their effectiveness on solid tumors.

CAR-T cell therapy has revolutionized cancer treatment, achieving significant success in hematological cancers through targeted immunotherapy. However, translating this success into solid tumors has proven challenging due to unique obstacles, such as the hostile tumor microenvironment, which induces CAR-T cell dysfunction, and tumor heterogeneity, which complicates effective targeting. Is.

Additionally, physical barriers within solid tumors hinder CAR-T cell infiltration, thereby reducing therapeutic efficacy. While recent clinical trials have shown promise in treating specific solid tumors, more research is needed to develop novel approaches to overcome these challenges and increase the efficacy of CAR-T cell therapy.,

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