Myelodysplastic syndrome therapeutics pipeline is expected to grow primarily due to the increasing prevalence of the disease as a result of increasing exposure of general population to chemicals and radiation. Some of the main factors driving the pipeline for myelodysplastic syndrome include rising older population, increasing exposure of heavy metals, smoking, tobacco consumption, low market penetration since a limited number of companies are making an analog of marketed drugs and increasing health awareness among people. According to the American Cancer Society, around 13,000 new cases of myelodysplastic syndrome are diagnosed each year in the U.S. It usually occurs in people with age of 65 years and above and is more prevalent among males as compared with females.
Myelodysplastic syndrome is a group of disorders in which unhealthy blood cells are formed with poor function. The cause of myelodysplastic syndrome is still unknown but it is assumed that myelodysplastic syndrome is caused by exposure to chemicals and radiations. The risk factors of myelodysplastic syndrome include smoking, environmental exposures, other cancers and genetic syndromes. The symptoms of myelodysplastic syndrome are anaemia, shortness of breath, unusual bleeding, petechiae and frequent infections. The symptoms of myelodysplastic syndrome usually occur in late stages, which results in late diagnosis of the disease. There are several types of myelodysplastic syndrome including refractory cytopenia with unilineage dysplasia, refractory anemia with ringed sideroblasts, refractory cytopenia with multilineage dysplasia, refractory anemia with excess blasts and myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality. The diagnosis of myelodysplastic syndrome is done by blood tests and bone marrow testing. Myelodysplastic syndrome is treated by blood transfusions, medications and bone marrow stem cell transplant. Also, changing lifestyle habits can help in improving the life of patient.
Many companies are developing drug for myelodysplastic syndrome which have shown promising result in clinical trials. Celgene Corporation is developing a drug by the name Luspatercept for the improvement of myelodysplastic syndrome. Luspatercept is being developed as a recombinant protein which acts as a TGF-beta superfamily protein inhibitor. It has also received an orphan drug designation for myelodysplastic syndrome and beta-thalassaemia by U.S. Food and Drug Administration (USFDA). Janssen Research & Development, LLC is developing Imetelstat, which is currently in a Phase III clinical trial. Imetelstat is a telomerase inhibitor and has received an orphan drug designation for myelodysplastic syndrome and myelofibrosis by USFDA. Kura Oncology, Inc. is developing Tipifarnib for myelodysplastic syndrome patients. It is currently in Phase II stage of development and is a small molecule that inhibits farnesyltranstransferase. Pfizer Inc. is developing PF-04449913, which is in Phase II clinical trial and is a smoothened (SMO) protein inhibitor.
Some of the companies having a pipeline of myelodysplastic syndrome therapeutics include Celgene Corporation, Janssen Research & Development, LLC, Onconova Therapeutics, Inc., Astex Pharmaceuticals, Inc., Alexion Pharmaceuticals, Inc., GlaxoSmithKline Plc, AbbVie, Inc., Genentech, Inc., Kura Oncology, Inc., Boehringer Ingelheim GmbH, Novartis AG, Syros Pharmaceuticals, Inc., Millennium Pharmaceuticals, Inc., Acceleron Pharma, Inc., Pfizer Inc.