Progress in treating rare cancers

Cancers is one of the top noxious diseases which probably as much old as the human existence. However, there have been immense development and advances in cancer therapeutics. If not much, the life expectancy of cancer patients is enhanced to much extent. Scientific discipline including genetics, molecular biology, recombinant DNA technology and many more have contributed significantly not only in better diagnosis but also in better therapeutics for cancers.       

The progress in terms of better and efficient cancer therapeutics is continuing rare cancers such as anaplastic thyroids cancer, neuro-endocrine tumors, uterine cancers and few more are less prevalent forms of cancers. These types of cancer make up approximate 20% incidences of cancer in United States. 

If we just talk about the rare forms of cancers, significant advances are here as well. However, at the same time, the understanding and research on these rare cancers is more challenging as well compared to others disease. One major reason is that having fewer incidences of these rare cancers makes it challenging for researchers to better understand its basis. Understudying the facts what possible genetic mutation, proteins overproduction or deficiency or obstruction in catalytic pathways of specific enzyme is root cause of cancer is no doubt a challenge. The recent advances and directions in research areas about finding better therapeutics for rare cancers are discussed here: 

Treatment Of Dismoid Tumors 

Desmoids tumor (also known as deep fibromatosis) is rare type of cancer. These are soft-tissues sarcoma and develop in connective tissues of body. The tumors may re-appear unexpectedly even after systemic treatment. 

A recent advancement in treatment of desmoids tumors is targeted therapy. A drug sorafenib (Nexavar) is used as target drug therapy. Sorafebin is also used in treatment of thyroids, liver and kidney cancers. This targeted therapy is an enzyme inhibitor of tyrosine kinase enzyme in tumor cell thereby, inhibit tumor cells proliferation.  Tyrosine kinases are class of enzyme involved in signal transduction pathways of cells. Inhibiting the signal transduction specifically in cancerous cells is a key step to inhibit the rapid proliferation of cancerous cells.      

Mid Gut Neuro-Endocrine Tumors (Nets) 

These are also known as carcinoid tumors which develop in gastrointestinal tract. These tumors are of malignant nature. Synthetic somatostatin hormones such as lanreotide (somatulin Depot) are applied for NETs treatment.

A recent advancement in NETs theraeputics is a radio-labelled drug lutathera (¹⁷⁷LDotatate). The radio-labeled isotope is ¹⁷⁷Lu. The drug targets the cancerous cells and bind to their somatostatin receptors. Later, the drug enters the cells and kills the cancerous cells due to radioactive properties of isotope. A few people developed side effects of this targeted drug. However, as supported by various studies, the FDA has approved this targeted drug therapy for treatment of NETs. 

Uterine Serous Carcinoma 

Uterine cancer is rare yet aggressive type of endometrial cancer. One major underlying genetic factor is HER2 gene mutation. Based on the molecular studies, researchers have developed a drug trastuzumab (Hercptin) in combination with chemotherapy, as targeted therapy for uterine cancer. Studies suggest that using trastuzumab for a period of 12.6 months significantly inhibit the tumor progression.

Tenosynovial Giant Cell Tumor 

This is a rare type of sarcoma which affects the tendons and joints in young patients. As the tendons and joints are affected, the patient’s physical capabilities are badly affected. One of the treatment options is to perform surgery to remove a membrane in joints. Sometimes a complete joint replacement surgery is required.

A targeted treatment, pexidartinib is gaining popularity in terms of tenosynovial tumor treatment. In a study, 39.3% of total 61 patients developed side effects to pexidartinib. Therefore, further research is required before approval of this treatment option for tenosynovial sarcoma.

Anaplastic Thyroid Cancer 

Anaplastic thyroid cancer is fast growing cancer of thyroids. A combination of two targeted therapies has been approved by the food and drug administration FDA in year 2018. The therapy combines dabrafenib (a BRAF gene inhibitor) and tramatinib (MFK inhibitor). BRAF gene mutation is major genetic factors of thyroid cancer. The drug trial on anaplastic thyroid patients revealed that combinational therapy is useful for approximately 80% of patients taking this therapy. Therefore, FDA has approved this combinational targeted therapy is now a standard treatment option for anaplastic thyroid patients.

Bottom line…

Treating rare types of cancers is a challenge. The recent development and advances in fields of science and research are achieving new heights for better and accurate therapeutics for rare cancers.