Local Company’s Promising Therapy for MDS

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At the Federation of American Societies of Experimental Biology meeting on Retinoids, Sephos Biotherapeutics presented novel findings in stem cell biology that can be used to induce differentiation of red blood cells.

Myelodysplastic syndrome is a fatal hematopoietic stem cell disorder that is characterized by the failure of bone marrow to produce blood cells. MDS is often termed pre-leukemia because of its propensity to develop in to acute leukemia.

abc_robin_roberts_dm_120611_wmainIn 2012 “Good Morning America” co-host Robin Roberts revealed  that she was diagnosed with Myelodysplastic Syndrome (MDS). MDS is malignant, but can affect all the blood cells in the body. Problems that occur with red blood cells can include anemia and low blood counts, while problems that occur with white blood cells can include increased number and severity of infections, since white blood cells are part of the body’s immune system.

In addition to abnormal blood components, MDS can cause immature cells called “blasts.” These cells can lead to leukemia, which is why MDS used to be referred to as “pre-leukemia” – a progression from MDS to leukemia is not uncommon, though it is not always a foregone conclusion.

The primary treatment for MDS is a bone marrow transplant, which means taking healthy marrow from a donor and replacing and repairing the abnormal bone marrow in an MDS patient.

“Each year 10,000 to 15,000 people are diagnosed with MDS in the United States. Despite advances in the efficacy of chemotherapy, radiation, targeted therapies and bone marrow transplantation, a cure for MDS has not been found,” said Dr. Dzana Dervovic, Scientific Officer at Sephos Biotherapeutics.

“The introduction of the drug Revlimid (Celgene), the first targeted therapy for myelodysplastic syndrome dramatically improved the treatment of this devastating disease,” said Dr. Elena Bogdanovic, Scientific Officer at Sephos Biotherapeutics. Sadly, however, Revlimid is only effective in 10-15% of MDS patients since it is only effective in patients with the mutation chromosome 5q deletion.

Sephos Biotherapuetics is committed to developing the next generation of targeted therapies for induction of differentiation of the acute myeloid leukemia and myelodysplastic syndrome stem cell.

“Using the molecular biology approach we are developing a novel targeted therapy, which targets abnormalities in a set of common cellular pathways for the treatment of MDS. This molecularly targeted agent offers the promise for improved MDS control without substantial toxicity,” said Dr. Dervovic

Sephos Biotherapeutics is a privately owned company focused on designing drugs for the treatment of cancer stem cells where the structure and biological activity of the drugs have been carefully modeled from studies of basic cellular mechanisms.