Rare brain cancer cured thanks to new therapy

DIPG brain cancer (diffuse pontine glioma) mainly affects children between the ages of 5 and 7. This is a rare form of cancer, with about 150-300 new diagnoses each year in the US, The 5-year survival rate is less than 1%. However, a patient named Drew survived four years after diagnosis and showed no signs of the disease. This is an unprecedented result. To achieve this, the doctor used Drew’s own immune cells, injected into his body to destroy cancer cells.

“This result gives me confidence that is hard to describe in words. At least one person in this trial seems to have recovered,” said Michelle Monje, a professor of neurology at Stanford Medicine who led the study. shall.

About eight years ago, when using tissue donated by a DIPG patient, a student of Professor Monje discovered that cancer cells all had certain identifying marks on their surface. If they can target and destroy just those cells, cancer treatment will be possible.

It was not until June 2020 that the research team was able to test this method on patients. The average age of the participants was only 15.

The patient’s own modified immune cells are introduced into their bodies to target cancer cells. It causes serious side effects and must be given along with chemotherapy to destroy most of the existing immune cells, to make room for the cells to mutate.

The study, published in the journal Nature in mid-November, found that Drew was the only patient out of 11 with DIPG and related cancers to have a positive outcome. Success brings the first realistic ray of hope for this incurable disease.

Drew was diagnosed with DIPG in 2020, during his second year of high school. His symptoms were headaches, abnormal movements in the left eye and left facial paralysis. The growing tumor affects his hearing and balance, making it almost impossible for him to walk.

The male student was treated with an experimental method in June 2021. The first dose of cells is administered to the entire body. The second and subsequent doses are administered directly into the cerebrospinal fluid. Like other patients, he experienced typical side effects: vomiting, chills and loss of balance. Side effects decreased significantly from the second dose.

He continued to study remotely during treatment. By the spring of his senior year, he was healthy enough to return to school, moving in the hallways with a walker. Drew graduated in May 2022 on time, attended the senior ceremony with his classmates, and was tumor-free.

Pathologically, DIPG progresses rapidly. Professor Monje said, a young patient who was healthy enough to go surfing in the early stages of the trial died three weeks later.

“The boy doesn’t look like someone who only has a few weeks to live. That shows how dangerous this disease is,” she said.

The patient’s tumor is located in the spine or brain stem, the part that controls basic functions such as breathing and heart rate. They are extremely difficult to treat because they connect to the nervous system and become part of the vessels they invade. Chemotherapy is ineffective, radiotherapy is only effective for a short time.

As tumors grow, they can cause serious disability, affecting the ability to walk, laugh, swallow, hear and speak. Spinal cord damage leads to pain, burning, paralysis, loss of sensation and urinary incontinence.

Another patient was almost completely paralyzed when participating in the trial, and the disease also progressed rapidly, Professor Monje said. After treatment, her tumor decreased by more than 90%. Patients regain the ability to walk, simply using a cane or using a walker. She was able to climb mountains, go on vacations with friends and stop most pain medications. However, the tumor recurred, and she died about a year later.

“This trial was like an emotional roller coaster for everyone. The pain was deepened after some rays of hope,” Professor Monje shared.

Professor Monje went on to recall the first patient in the trial, a young man named Jace Ward. He volunteered to accept the risk, hoping the method could cure other patients.

Jace was the first person to receive more than one dose of cells. This is also the first time they have been injected directly into the brain stem, despite concerns that it could cause death. However, the method has not been successful. Scientists don’t know why treatments often have an initial benefit, but stop being effective at a later stage. The team believes it is related to other aspects of the immune system. In the trial, nine patients improved function. In four people, tumor volume was reduced by at least half.

Drew is in college majoring in forestry and minoring in chemistry.

“Watching him grow up, develop strongly, go to college, choose a major and contribute to the world, all of it strengthens my faith and gives me more energy,” Professor Monje said. .