By Lauren Taylor from In The Cloud Copy
Onconova Therapeutics, Inc. is a biopharmaceutical company that works to develop new drugs to treat cancer, primarily focusing on myelodysplastic syndromes. Myelodysplastic syndromes, or MDS, are a group of cancers that occur when something goes amiss in the bone marrow’s process of producing healthy, mature blood cells. In MDS, the blood cells end up dying, either in the bone marrow or shortly after entering the bloodstream. Eventually, there are more immature and defective cells than there are healthy, functioning cells, which is what ultimately leads to symptoms.
Myelodysplastic syndromes rarely cause signs and symptoms in the early stages of the disease. This can make diagnosis and treatment considerably more difficult. Myelodysplastic syndromes eventually might cause symptoms such as fatigue, shortness of breath, paleness, easy or unusual bruising or bleeding, petechiae (pinpoint-sized red spots beneath the skin), or frequent infections.
Some forms of MDS have no known cause while others are caused by exposures to other cancer treatments like chemotherapy or radiation, or exposure to other toxic chemicals or heavy metals.
Current treatments aim to manage the symptoms associated with MDS. Some of these treatments might include medications that increase blood cells produced in the body, blood transfusions that replace the unhealthy cells produced in the patient’s body, or a bone marrow transplant. Current treatments are supportive and limit disease while attempting to extend the patient’s life. A bone marrow transplant is the only potential cure for MDS, but it comes with its own set of complications.
Rigosertib Therapy and the Fight Against Myelodysplastic Syndromes
Rigosertib plays a potentially important and life-changing role in anti-cancer treatment. Rigosertib works to balance the RAS pathway. RAS pathway mutations are the most common mutations seen in cancers. RAS proteins work by controlling the signaling pathways that regulate different aspects of either normal cell growth or malignant cell growth. Activation of the RAS pathway can occur either through direct mutation of RAS or one of its proteins. The mutations seen along this pathway significantly contribute to later stage, high risk myelodysplastic syndromes.
Rigosertib is a first of its kind treatment that works by blocking the activation of the RAS effector proteins. While this treatment is being developed by Onconova specifically for the treatment of myelodysplastic syndromes, it would likely have other applications in solid and liquid tumors that have RAS pathway activations.
Rigosertib is currently in a clinical development stage with both oral and IV compounds being tested. The single agent IV rigosertib is being studied in patients with MDS that is considered higher risk, while the oral rigosertib in addition to azacitidine (a chemotherapy drug), is being studied in hypomethylating agent (HMA)-naïve and refractory higher-risk MDS patients. These studies are in phase 3 and phase 2 respectively. Further studies are being conducted in investigator-initiated study (IIS) programs. In the IIS programs, oral rigosertib is being used for treatment of a variety of cancers with RAS mutations with solid tumors.
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