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Unmet Needs
Unmet Needs 2016: Myelodysplastic Syndromes (MDS)
October 27, 2016
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Current State of the Art: Management of Higher Risk Myelodysplastic Syndromes (MDS). Komrokji RS. Clin Lymphoma Myeloma Leuk. 2016 Aug;16 Suppl:S39-43. doi: 10.1016/j.clml.2016.02.019.

https://www.ncbi.nlm.nih.gov/pubmed/27521322

Hypomethylating agents are the only medications that alter the natural history of the disease. Azacitidine is the only drug reported to improve overall survival in higher risk MDS patients. Appropriate use and assessment of response is key for assuring patients benefit of such limited options. Treatment after failure of hypomethylating agents is an unmet need. The role of detectable somatic gene mutations in prognosis and tailoring therapy continue to emerge.

Growing Awareness and High-Unmet Medical Needs of Patients to Propel the Global Central Nervous System Market, According to Technavio

http://www.businesswire.com/news/home/20160526005461/en/Growing-Awareness-High-Unmet-Medical-Patients-Propel-Global

The global myelodysplastic syndrome (MDS) market which will grow at a steady CAGR of around 10% during 2016-2020. This market is envisaged to grow at a moderate pace due to the high-unmet medical needs of patients, growing public awareness, and the increase in the ageing patient population. Companies that have the ability to manufacture and market drugs that can effectively treat and cure MDS will gain prominence in this market during the forecast period.

According to Barath Palada, a lead research analyst at Technavio for healthcare and life sciences research, “The presence of diagnostic tools is the key driver for the growth of this market. Diagnosis techniques like blood count, peripheral blood smear test, microscopic exams, cytochemistry, flow cytometry and immunotherapy, cytogenetics, FISH, and PCR help in the detection of MDS. Consequently, these diagnostic tools are expected to have a positive impact on the prescription of drugs for people suffering from MDS.

Myelodysplastic Syndrome Market to Grow at 10% CAGR to 2019

http://www.marketwatch.com/story/myelodysplastic-syndrome-market-to-grow-at-10-cagr-to-2019-2016-02-02-72033117

For growth of myelodysplastic syndrome market, presence of diagnostic tools is considered a key driver with diagnosis techniques like blood count, peripheral blood smear test, microscopic exams, cytochemistry, flow cytometry and immunotherapy, cytogenetics, FISH, and PCR help in the detection of MDS. Consequently, these diagnostic tools are expected to have a positive impact on the usage of drugs for people suffering from MDS. The analysts forecast global myelodysplastic syndrome market to grow at a CAGR of 10% during the period 2015-2019.

Government organizations and pharmaceutical companies are conducting awareness programs to spread knowledge about the disease and associated drugs. In addition, they are also providing patient assistance programs to give low-income people with myelodysplastic syndrome access to these drugs. These two factors will surge the number of people availing treatment services, increasing the sales and consumption of drugs.

According to the myelodysplastic syndrome market report, the immune system of an individual deteriorates as aging progresses. For this reason, people aged 60 and above are more susceptible to this disease. A surge in the population of this age group will increase the need for treatment drugs, positively influencing the latter’s sales and revenue.

Alkylating agents are used to treat different types of cancer and function by attaching an alkyl group to the guanine base of the DNA. Dacogen by Otsuka and Vidaza by Celgene are examples of alkylating agents that are used for the treatment of MDS.

The global myelodysplastic syndrome market is envisaged to grow at a moderate pace due to the high-unmet medical needs of patients, growing public awareness, and the increase in the ageing patient population. Companies that have the ability to manufacture and market drugs that can effectively treat and cure MDS will gain prominence in myelodysplastic syndrome market during the forecast period.

Leading vendors in the global myelodysplastic syndrome market are Celgene, Novartis and Otsuka. Other prominent vendors in this market are Acceleron Pharma, Amgen, Astellas, Astex , Actinium Pharmaceuticals, Bellicum Pharmaceuticals, BioLineRx, Celator Pharmaceuticals, Cornerstone Pharmaceuticals, CTI BioPharma, Eli-lilly, Gamida Cell , GlaxoSmithKline, KaloBios Pharmaceuticals, Kiadis Pharma, Mirati Therapeutics, Onconova Therapeutics, Strategia Therapeutics, Sumitomo Dainippon Pharma , Sunesis Pharmaceuticals, Targazyme, and TetraLogic Pharmaceuticals.

Further, the report states that the high cost of drugs used for treating myelodysplastic syndrome can deter people from consuming them.

Molecular and Cellular Mechanisms of Myelodysplastic Syndrome: Implications on Targeted Therapy

www.mdpi.com/1422-0067/17/4/440/pdf

Better understanding of the molecular landscape of MDS has important clinical implications. Firstly, prognosticating MDS based on molecular aberrations will supplement current models in stratifying patients for treatment including allogeneic HSCT. Somatic mutations of TP53, TET2, and DNMT3A have been shown to identify patients with MDS with shorter OS after allogeneic HSCT, suggesting that newer treatment strategies other than transplantation are needed for these patients. Secondly, molecular markers may better predict response and resistance to treatment with HMAs. Thirdly, detection of targetable molecular markers during treatment resistance or leukemic transformation may provide an opportunity for specific therapy, as exemplified by the use of FLT3 inhibitors in FLT3-ITD positive secondary AML.

Finally, understanding the molecular dynamism of myeloid cell mutation has important implications on treatment, particularly in detecting clonal hematopoiesis of indeterminate potential (CHIP) that precedes the development of clinical MDS. Currently, there is as yet no specific treatment for these mutations. However, rapid advances in molecular biology and drug development lead to the optimism that these mutations may soon be targetable, with a view to early treatment perhaps even at the stage of CHIP so as to avert the development of myeloid malignancies. Hence, future treatment strategies for MDS may involve exploitation of genetic information in designing more effective therapy encompassing single agents or combinatorial approaches.

Opsona Receives Orphan Designation For Myelodysplastic Syndrome (MDS) With OPN-305, A First-In-Class Monoclonal Antibody That Blocks Toll-Like Receptor 2

https://www.biospace.com/News/opsona-receives-orphan-designation-for/436820

Opsona Therapeutics Ltd (‘Opsona’), the innate immune drug and development company focused on novel therapeutic approaches to treat oncology, autoimmune and other inflammatory diseases, today announced that it has received orphan drug designation (ODD) from United States Food and Drug Administration for myelodysplastic syndromes (MDS).

Mary Reilly, VP Pharmaceutical Development and Operations at Opsona Therapeutics, said: “We are pleased to receive FDA Orphan Drug Designation for OPN-305 in MDS. This is an important regulatory milestone for the company and a significant step forward in our clinical development of OPN-305 targeting this rare disease associated with an unmet medical need for safe and effective therapeutics.”