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There is always research going on in the area of brain and spinal cord tumors. Scientists and doctors are looking for causes and ways to prevent them, better tests to help characterize these tumors, and better ways to treat them.
In recent years, researchers have found some changes in genes, chromosomes, and proteins inside brain tumor cells that can be used to help predict a child’s outlook (prognosis) or help guide treatment. Some examples of changes that can now be tested for include:
For children with medulloblastomas, doctors can now also test for other gene changes that can help show if they are likely to have a better outlook (and therefore might require less intensive treatment).
For more on these tests, see "Lab tests of biopsy specimens" in Tests for Brain and Spinal Cord Tumors in Children.
Researchers are also looking for other changes in tumor cells that might help guide treatment.
Recent advances have made surgery for brain tumors much safer and more successful. Some of these newer techniques include:
Children's brains are very sensitive to radiation, which can lead to side effects if normal brain tissue receives a large dose, especially if the child is very young. Several newer types of radiation therapy now let doctors aim radiation more precisely at the tumor, which helps spare normal brain tissue from getting too much radiation. Newer techniques such as stereotactic radiosurgery, 3-dimensional conformal radiation therapy (3D-CRT), intensity modulated radiation therapy (IMRT), and proton beam therapy are described in Radiation Therapy for Brain and Spinal Cord Tumors in Children.
Clinical trials have shown that in some situations, using chemotherapy can let doctors use lower doses of radiation therapy without lowering the chance that treatment will be effective. Doctors are now trying to determine if even lower doses of radiation can be used and still give the same results.
New approaches may help make chemotherapy (chemo) more useful against brain and spinal cord tumors.
In some children and infants with brain tumors, chemo is given right after surgery to either delay radiation therapy (particularly in infants) or to decrease the radiation dose needed to treat the tumor. This is known as adjuvant chemotherapy. Some studies are looking at whether giving prolonged chemo can help avoid the need for radiation therapy at all in certain cases.
One of the main factors that limits the doses of chemo that can be given safely is its effects on the bone marrow, where new blood cells are normally made. A stem cell transplant allows higher doses of chemo to be given than would normally be possible. First, blood stem cells are removed from either the child’s blood or the bone marrow and are stored in a deep freeze. The child is then treated with very high doses of chemo. The blood stem cells are then thawed and infused back into the body, where they settle in the bone marrow and start making new blood cells.
Although some children with certain brain or spinal cord tumors (such as medulloblastomas) have responded well to this very intensive treatment, it can have serious side effects, and it is not yet known if it is effective enough to become a standard treatment. For now, most doctors consider this treatment experimental for brain and spinal tumors. Clinical trials are being done to determine how useful it is.
Many chemo drugs are limited in their effectiveness because the tightly controlled openings in the brain capillaries, sometimes referred to as the blood-brain barrier, prevents the drugs from getting from the bloodstream to some parts of the brain tumor. Researchers are now trying to modify some of these drugs by coating them with tiny layers of fat (liposomes) or attaching them to molecules that normally cross the blood-brain barrier, to help them work better. This is an area of active research.
Some newer approaches might help doctors get chemo directly to brain and spinal cord tumors.
For example, in one method called convection enhanced delivery, small tubes are placed into the tumor in the brain through a small hole in the skull during surgery. The tubing extends through the scalp and is connected to an infusion pump, through which chemo drugs can be given. This can be done for hours or days and might be repeated more than once, depending on the drug used. This technique can also be used to get other, newer types of drugs into the tumor. This is still an investigational method, and studies are continuing.
Researchers are also looking at the possibility of using lasers or other means to disrupt the blood-brain barrier and allow drugs to more readily reach brain tumors.
Researchers are also testing some newer approaches to treatment that may help doctors target tumors more precisely. The hope is to develop more effective treatments that cause fewer side effects. Although these treatment approaches are promising, most are still experimental at this time and are only available through clinical trials.
As researchers have learned more about the gene changes in tumor cells that help them grow, they have developed newer drugs that target these changes. These targeted drugs work differently from standard chemo drugs. Here are some examples of targeted drugs now being studied or in use:
Many other targeted drugs are already being used to treat other types of cancer, and some are being studied to see if they will work for brain tumors as well.
Tumors have to create new blood vessels (a process called angiogenesis) to keep their cells nourished. Targeted drugs that attack these blood vessels are used to help treat some cancers, including some brain tumors in adults. Several drugs that impair blood vessel growth are now being studied for use against brain tumors in children.
Some drugs increase the oxygen content in the tumor, which makes tumor cells more likely to be killed by radiation therapy if the drugs are given before treatment. Studies are now looking to see if this affects treatment outcomes.
The goal of immunotherapy is to help the body’s own immune system fight the tumor.
Several types of vaccines are being developed against brain tumor cells. Unlike vaccines against infectious diseases, these vaccines are meant to help treat the disease instead of prevent it. The goal of the vaccines is to stimulate the body’s immune system to attack the brain tumor cells.
Early study results of some of these vaccines have shown promise, but more research is needed to determine how effective they are. At this time, brain tumor vaccines are available only through clinical trials.
Other types of drugs that affect the immune system are also being studied.
Researchers have done a great deal of lab work with viruses that reproduce only within brain tumor cells and then cause those cells to die, while leaving normal cells alone. Research using these viruses in humans with brain tumors is still in very early stages.
Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.
Chang SM, Mehta MP, Vogelbaum MA, Taylor MD, Ahluwalia MS. Chapter 97: Neoplasms of the central nervous system. In: DeVita VT, Lawrence TS, Rosenberg SA, eds. DeVita, Hellman, and Rosenberg’s Cancer: Principles and Practice of Oncology. 10th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2015.
Dorsey JF, Hollander AB, Alonso-Basanta M, et al. Chapter 66: Cancer of the central nervous system. In: Abeloff MD, Armitage JO, Niederhuber JE. Kastan MB, McKenna WG, eds. Abeloff’s Clinical Oncology. 5th ed. Philadelphia, Pa: Elsevier; 2014.
National Cancer Institute. Childhood Brain and Spinal Cord Tumors Treatment Overview (PDQ®). 2017. Accessed at www.cancer.gov/types/brain/hp/child-brain-treatment-pdq#section/all on April 26, 2018.
Williams D, Parsons IF, Pollack DA. Chapter 26A: Gliomas, Ependymomas, and Other Nonembryonal Tumors of the Central Nervous System. In: Pizzo PA, Poplack DG, eds. Principles and Practice of Pediatric Oncology. 7th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2016.
Last Revised: March 17, 2023