Drug therapy, also called systemic therapy, is the use of treatments that spread throughout the body to kill cancer cells wherever they are located. These treatments are prescribed by doctors called medical oncologists or neuro-oncologists. Systemic therapy includes different types of drugs and treatments including targeted therapy, immunotherapy, and chemotherapy, which are described below.
If you are considering drug therapies, the Chordoma Foundation Medical Advisory Board recommends that you consult with a chordoma-experienced medical oncologist about drug therapies that may be right for you based on your individual tumor and medical situation, taking into consideration the full range of options and associated evidence, including those presented in the Chordoma Foundation’s list of drug therapy options.
Since surgery and radiation are the recommended treatments for newly diagnosed chordoma, drug therapy is usually only used to treat locally advanced tumors that can no longer be adequately treated with surgery or radiation, as well as tumors that have spread to other parts of the body. Drug therapies may also be considered if the tumor is growing rapidly, or if it has been determined that the tumor is either the poorly differentiated or dedifferentiated type of chordoma.
Currently, no drug therapies have been approved by government regulatory agencies such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) for the treatment of chordoma. However, in some countries, doctors are allowed to prescribe treatments to chordoma patients that have been approved for other cancers. This is called “off-label” use of the treatment.
When doctors prescribe drug therapies off-label, they are typically not paid for by insurance or by health systems. However, this varies widely, so it is important to check with your insurance provider or health system. Occasionally, drug companies have programs that make their treatments available to patients at minimal or no cost. These programs are called “compassionate use” or “expanded access”. It may also be possible for chordoma patients to receive certain therapies by participating in clinical trials.
All drug therapies have the risk of potential side effects, ranging from mild to severe. These include:
Any new side effects experienced while taking a drug therapy, whether in a clinical trial or off-label, should be reported to your doctor immediately.
Typically, you will continue with a particular therapy until your tumor grows significantly, which suggests the treatment has stopped working, or unless the side effects are too difficult. If your doctors discontinue treatment for one of these reasons, they will work with you to find another therapy to try.
Personalized medicine, also called precision medicine, uses information about a person's genetic makeup and other characteristics to prevent, diagnose, and treat diseases. In cancer specifically, personalized medicine involves testing a patient’s tumor cells to learn more about the tumor’s genetic alterations. Doctors then use this information to guide their decisions on what treatment is best for that particular patient. Personalized medicine is routinely used in more common cancers such as breast and lung cancer and has shown promise in many others.
Genomic profiling provides information about the genetic makeup of cancer cells. To get this information, a small piece of tumor tissue, often taken from a recent surgery or biopsy, is sent to a lab where the DNA – and sometimes RNA, depending on the test – of the cancer cells is analyzed in order to identify changes and alterations.
A report is generated that includes the results of the test. In some cases, an actionable target is found – for example, the test finds a mutation in the patient’s tumor cells that can be targeted by a certain drug therapy. In other cases, no actionable mutations are found or the mutations found do not have accessible treatments – for example, the treatment is only available through a clinical trial but the patient is not eligible for the trial.
Some clinical trials offer genomic profiling as part of the trial and profiling tests are also available through some major medical centers. Many private companies also offer tumor profiling services. For U.S. patients, some insurance plans pay for the tests and Medicare (and in some states, Medicaid) now covers some types of tests from certain companies (e.g., Foundation Medicine). Private companies may offer financial assistance programs to patients who are uninsured or cannot afford the cost of testing. In some countries, the government health system may pay for profiling tests.
Genomic sequencing
Targeted sequencing tests examine specific genes or regions of the genome known to be associated with particular diseases or conditions. Targeted sequencing for cancer usually focuses on a small subset of about 500 genes that are often involved in common cancers. This makes targeted sequencing like looking through a specific section of books in a library that might contain information you're interested in. By focusing on these key areas, targeted sequencing provides information that can help guide treatment decisions in some cases.
Whole exome sequencing (WES) is a more comprehensive genomic profiling test that examines all the genes in your tumor cells — around 20,000 — to provide a more complete overview of the genetic makeup of your tumor. This approach is far more extensive than targeted sequencing panels. If your genome is like an extensive library, WES is like reviewing nearly every book on the shelves.
Next-generation sequencing (NGS) can refer to targeted sequencing or whole exome sequencing. If your doctor is recommending NGS, it can be helpful to ask if the testing will be targeted sequencing or WES.
Transcriptomic sequencing
In addition to genomic sequencing tests, there's RNA sequencing (RNA-Seq), which assesses the genes that are active (or being “transcribed”) in your tumor cells and determines the level of their activity. This is like determining which books in the library are being read and how avidly.
Some tests offer both WES and RNA-Seq together, which is called multi-omics profiling. This dual approach provides the most comprehensive information about your tumor.
Many chordoma patients considering drug therapy options are offered or can request genomic profiling tests. A recent tumor sample is best for testing, but the tests can usually also be done on tissue stored after a previous surgery if a recent sample is not available.
For many cancers, including chordoma, there is no certainty that the results of tumor profiling tests will be able to help guide treatment decisions. Discussing the pros and cons of testing with your medical oncologist can help you make the best decision for your individual situation.
Some points to consider with your doctors might include:
Hear from chordoma experts about drug therapies for chordoma
The resources and information below can help you make the most informed decisions about your treatment.
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Our Doctor Directory can help you locate a doctor with chordoma expertise.
Our list of drug therapy options provides more information on off-label drug therapies and clinical trials.
The information provided herein is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your or your child’s physician about any questions you have regarding your or your loved one’s medical care. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.