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Saúde / 13/04/2021


Brain cancer vaccine has historic success in human testing

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Brain cancer vaccine has historic success in human testing

A vaccine against brain cancer with the first test on humans has been successfully published by the scientific journal Nature by the leaders of the experiment.

After promising results, the researchers will go on to phase 2 immunotherapy tests to help the immune system kill brain tumors.

According to the Nature article, the vaccine is safe for all patients and shows the expected immune response to cancerous tissue.

Diffuse gliomas are usually incurable brain tumors that spread in the brain and are difficult to remove completely by surgery. Chemotherapy and radiation therapy also tend to have a limited effect.

“Our idea was to support patients' immune systems and use a vaccine as a targeted way to alert them to the specific tumor neoepitope,” explained study director Michael Platten, Medical Director of the Department of Neurology at University Medicine Mannheim and Chief Division of the German Cancer Research Center (DKFZ).

The IDH1 mutation is a particularly suitable candidate here, as it is highly specific for gliomas and does not occur in healthy tissue. In addition, the IDH1 mutation is responsible for the development of these gliomas: "This means that a vaccine against the mutated protein allows us to attack the problem at the root," added Platten.


A total of 33 patients at several different centers in Germany were included in the study. In addition to the standard treatment, they received the peptide vaccine produced by the University Hospital in Heidelberg and the University of Tübingen. The immune response could be assessed in 30 patients, according to a study published in Nature.

Doctors did not see any serious side effects in any of the vaccinated patients. In 93 percent of patients, the immune system showed a specific response to the vaccine peptide and did so regardless of the patient's genetic basis, which determines the important presentation molecules of the immune system, the HLA proteins.

In most of the vaccinated patients, doctors observed "pseudoprogression", swelling of the tumor caused by a series of cells invading the immune system.

These patients had a particularly large number of helper T cells in their blood with immune receptors that responded specifically to the vaccine peptide, as revealed by single cell sequencing.

"We were also able to demonstrate that the specific mutation-activated immune cells invaded brain tumor tissue," reported Theresa Bunse, of DKFZ, who coordinated immunological analyzes for these studies.

High survival

The three-year survival rate after treatment was 84 percent in fully vaccinated patients, and in 63 percent of patients, tumor growth had not progressed within this period.

Among patients whose immune system showed a specific response to vaccines, a total of 82 percent had no tumor progression within three years.

The researchers are also preparing a phase II study to examine for the first time whether the IDH1 vaccine leads to better treatment results than standard treatment alone.

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