Novel drug development for chordoma treatment brings new hope to people with this rare cancer type
Chordoma is a rare kind of bone cancer that can occur in people of all ages but is most common among individuals between 40 and 75. However, chordoma can be very rare and occur in about one out of every million people. That means that about 300 patients are diagnosed with chordoma each year in the United States, and about 700 in all of Europe. At any given time, fewer than one in 100,000 people are living with chordoma. Though chordomas grow slowly, they can be aggressive and life-threatening tumors that can occur in the spine, the sacrum and base of the skull. The cancerous tumor is considered to develop from rod of the embryonic tissue or the cells left over from notochord. Chordoma affected people can undergo surgery and radiation, but the tumors often come back after treatment. The occurance of the cancer in critical structures such as the spinal cord, brainstem, and vital nerves and arteries, make chordomas extra difficult to treat. The chordoma market is expected to witness a CAGR of 6.8% over 2017-2023.
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Chordoma is not known to be caused by environmental, dietary, or lifestyle risk factors. The vast majority of chordomas occur at random without any direct result of an inherited genetic trait. However, research shows that there are several genetic factors associated with chordoma. For instance, more than 95% of individuals with chordoma have a variation, called a SNP, in the DNA sequence of a gene called brachyury. This SNP, though does not by itself, can cause an increase in the risk of developing chordoma. In fact, a large fraction of the general population has this SNP, but the chances of developing chordoma are less than two in a million.
The exact cause for chordomas is yet unknown. Research has shown that high expression of a transcription factor called brachyury can cause the cancer to occur. Though chordomas can occur in individuals of any age which also includes children, most often they occur in older individuals. Expanding global geriatric population is therefore expected to add impetus to the chordoma market. This is not to say that the incidence of chordoma will increase but since the demographic that is prone to develop chordoma will expand, diagnosis and treatment for the cancer form will also increase thus leading to the growth of the chordoma industry.
Since survival rate in chordoma affected people is rare, scientific research to find a cure for the disorder has been afoot for quite some time. Researchers at Broad Institute, Chordoma Foundation, Dana-Farber Cancer Institute, Baylor College of Medicine and Massachusetts General Hospital had come together to conduct a wide study to find the exact cause for chordoma and what can prevent the occurrence of the same. The study—a combination of genetic and small-molecule experiments in cell-line and animal models—reveals a potential molecular workaround for treating chordomas. Cell-line and animal model tests pointed that a chemical compound called THZ1 can significantly shrink tumors in mice with chordoma. The findings, if validated in additional studies, suggest that THZ1 or similar drugs could be valuable options for treating chordoma patients. A possible cure for the cancer can definitely pave better growth scopes for chordoma market in the future.
Chordoma market is poised to witness greater growth as recently the U.S. Food and Drug Administration granted BN-Brachyury Orphan Drug Designation for the treatment of chordoma.
Since brachyury protein may be a driver of chordoma BN-Brachyury vaccine targets this protein and can work as an alternative therapy for chordoma instead of radiotherapy and surgery. A phase 1 trial for BN-Brachyury vaccine is being conducted to ascertain its safety and tolerability. Researchers plan to initiate a phase 2 trial of the vaccine, which will enroll up to 25 patients with metastatic chordoma. BN-Brachyury will be evaluated in combination with radiotherapy. The vaccine, which was developed by National Cancer Institute in collaboration with Bavarian Nordic has received the Orphan Drug Designation, which is granted by the FDA to drugs that are developed for the treatment of diseases that affect less than 200,000 patients per year in the U.S.
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