The MDS Beacon™

Treatment for MDS will vary depending on symptoms, diagnosis, risk category, disease stage, age, pre-existing conditions, general health, and availability of bone marrow donors. A doctor knowledgeable in cancer and blood can determine the best type and combination of therapy on an individual basis.

Supportive Care

Supportive care is not a cure, but the management of symptoms caused by low blood cell counts (cytopenias). To counteract the shortage of red blood cells (anemia) and platelets, a patient’s abnormal blood cells are often replaced with healthy blood from a matching donor, a procedure called a blood transfusion. To reduce the need of transfusions, patients often receive growth factors – such as erythropoietin (EPO) and granulocyte-colony stimulating factor (G-CSF) – that simulate more blood cell development. To counteract low white blood cells (neutropenia) and a weak immune system, patients typically receive antibiotics.

Drug Therapy

In the US, three drugs have been approved for MDS treatment. Each of these drugs was approved for use individually, and not in combination with the other two drugs.

• Vidaza (azacitidine) kills rapidly dividing cells in bone marrow and removes methyl groups from DNA (demethylation). Methyl groups that bind to DNA sequences prevent the regulation of cellular growth, causing uncontrollable cell division. Demethylating agents, such as Vidaza, allow the DNA sequences to function normally by removing these methyl groups. It is approved for all MDS subtypes and is administered by subcutaneous injection or intravenous infusion for seven days every 28 days.
• Revlimid (lenalidomide) inhibits new blood vessel growth while stimulating both cell death and the immune system. It is effective for treating Low- or Intermediate-1 risk MDS, specifically those who require red blood cell transfusions or have 5q-syndrome. It is taken orally as a capsule once a day for as long as the treatment lasts.
• Dacogen (decitabine) is a DNA demethylating agent and is approved for all MDS sub-types. It is administered through intravenous infusion for three consecutive days every six weeks, requiring a hospital stay.

Chemotherapy

Chemotherapy is the use of drugs to kill cancerous cells, while also helping bone marrow to produce healthy blood cells. Unlike radiation or surgery, chemotherapy is not localized to one area of the body and can affect every cell. Intensive chemotherapy treatment runs between six to nine months and may bring short-term reduction of symptoms for as high as 60 percent of MDS patients. Chemotherapy may help MDS patients more permanently if bone marrow transplantation is an option.

Drugs used commonly in chemotherapy for MDS include:

• Cytarabine (Cytosar-U)
• Idarubicin (Idamycin)
• Daunorubicin (Cerubidine)
• Mitoxantrone (Novantrone)

Bone Marrow Transplantation

Bone marrow transplantation is the only curative treatment currently available. The bone marrow of a patient is initially destroyed by chemotherapy or radiation, and then replaced with donated bone marrow. The risks of the procedure may be aggravated by age, and a transplant does not guarantee full recovery.

• Allogeneic stem cell transplant (Allotransplantation) patients receive bone marrow from a matched donor. Graft vs. host disease is a possible risk, as functional immune cells in the transplanted marrow may discriminate the recipient body as foreign and attack it.
• Reduced-intensity / Non-myeloablative allogeneic stem cell transplantation is a lower-risk procedure for older patients and involves lower doses of chemotherapy and radiation in preparation for the transplant, supplemented by immunosuppressive drugs that prevent the body from rejecting the foreign transplanted marrow.
• Autologous stem cell transplant patients receive their own stem cells after the bone marrow is destroyed. However, this type of transplant is not a standard treatment for patients with MDS because their bone marrow contains abnormal stem cells.

Future Treatments

Clinical trials are currently being held to test and approve new treatments for MDS, especially combination treatments of FDA-approved drugs and chemotherapy. All trials are focused on developing methods to improve patient quality of life, effectiveness of treatments, and survival rates for MDS patients.

Current investigational studies are targeting cancer cells through many different mechanisms. New treatments may be able to reduce the number of blasts, while producing less-toxic side effects of standard treatments.

In addition, antithymocyte globulin (ATG), thalidomide (Thalomid), and anticancer agents called histone deacetylase inhibitors are being investigated for their role in stopping cellular proliferation and causing differentiation or death of tumor cells. Differentiation therapy aims to restrain growth of malignant cells by forcing immature tumor cells to resume the process of maturation and eventually differentiate into mature cells.

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