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Vidaza May Be More Cost Effective Than Dacogen – Results of a recent analysis indicate that Vidaza (azacitidine) may be more cost-effective than Dacogen (decitabine) for myelodysplastic syndromes (MDS) patients. The analysis makes use of U.S. health care treatment costs and assumptions about treatment outcomes based on previously published clinical trial results.  The total of all health care costs for a patient treated for two years with Vidaza is estimated to be $150,322, compared to $166,212 for Dacogen. The analysis was sponsored by Celgene, the manufacturer of Vidaza. For more information, please see the analysis in the journal Leukemia & Lymphoma (abstract).

Free Conference For MDS Patients And Their Families – On November 12, the MDS Foundation will host a free one-day conference for MDS patients and their families in Philadelphia. The conference will start at 10 a.m. with a patient support group discussion. In the afternoon, new therapies and treatment options for MDS will be presented and discussed.  Complimentary breakfast and lunch will be served. For more information, please see the MDS Foundation website.

Webinar: MDS And Health Insurance Coverage – On November 17, the Aplastic Anemia & MDS International Foundation (AA&MDSIF) is hosting a webinar to explain health insurance coverage rules to MDS patients and their families. The webinar, which starts at 3 p.m. ET, will also include information about prescription and co-pay assistance programs that may be of interest to MDS patients. Participants will have the opportunity to ask questions at the end of the webinar. For more information or to register, please see the AA&MDSIF website.

For a more detailed listing of MDS-related events, please check the MDS Beacon Events Calendar.

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FDA Advisory Committee Recommends Ferriprox For Accelerated Approval – The pharmaceutical company ApoPharma announced last week that the Oncologic Drugs Advisory Committee to the U.S. Food and Drug Administration (FDA) recommended that the FDA grant accelerated approval to the iron chelation agent Ferriprox (deferiprone). Specifically, the advisory committee recommended that Ferriprox receives accelerated approval for the treatment of patients with blood transfusion-related iron overload when current chelation therapy is inadequate. The FDA normally follows the recommendations of its advisory committees but is not obligated to do so. Ferriprox has already been approved for the treatment of blood transfusion-related iron overload in Europe since 1999. Current treatment options for myelodysplastic syndromes (MDS) patients with transfusional iron overload include Desferal (deferoxamine) and Exjade (deferasirox). For more information, please see the Apotex press release.

Webinar: The Latest Developments In MDS – On September 29, the Aplastic Anemia & MDS International Foundation (AA&MDSIF) is hosting a webinar summarizing the main findings of the State-of-the-Science Symposium on MDS, which the National Institutes of Health held at the beginning of the month. Dr. David Steensma of the Dana-Farber Cancer Institute in Boston will lead the webinar, which starts at 3 p.m. ET. Dr. Steensma will talk about the implications of these findings for patients and their family members. Participants will have the opportunity to ask questions at the end of the webinar. For more information or to register, please see the AA&MDSIF website.

Free Conference For MDS Patients And Their Families – On October 1, the MDS Foundation will host a free one-day conference for MDS patients and their families in Boston. The conference will start at 10 a.m. with a patient support group discussion. In the afternoon, new therapies and treatment options for MDS will be presented and discussed.  Complimentary breakfast and lunch will be served. For more information, please see the MDS Foundation website.

For a more detailed listing of MDS-related events, please check the MDS Beacon Events Calendar.

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Scotland Approves Use Of Vidaza In MDS – Earlier this week, the Scottish Medicines Consortium (SMC) approved the use of Vidaza (azacitidine) for patients with advanced myelodysplastic syndromes (MDS). The SMC decision, therefore, is in agreement with the guidance of the National Institute of Clinical Excellence (NICE), the agency that decides which treatments will be funded for patients in England and Wales. NICE recommended the use of Vidaza for high-risk MDS patients in February of this year. For more information, please see the related article in Pharma Times.

Acceleron Launches Phase 1 Trial For ACE-536 – The biopharmaceutical company Acceleron Pharma yesterday announced the start of a Phase 1 clinical trial of its investigational drug ACE-536 that is being developed for the treatment of low red blood cell counts (anemia) in people with MDS or other diseases. ACE-536 works by inhibiting certain proteins that negatively affect the late stages of red blood cell development. The goal of the study is to assess the maximum tolerated dose and the safety of ACE-536. For more information, please see the Acceleron press release.

Researchers Identify Gene Mutations For Hereditary MDS - Researchers from Australia recently found that mutations in a gene called GATA2 are linked to hereditary MDS. Specifically, the researchers found that the mutations in the GATA2 gene were passed on to multiple generations in three families in which MDS or acute myeloid leukemia ran in the family.  The GATA2 gene is used to produce a protein that is commonly found in blood stem cells and is required for the formation of mature blood cells. For more information, please see the article in Nature Genetics (abstract).

Living With MDS Conference For Patients And Families – The Aplastic Anemia and MDS International Foundation (AA&MDSIF) will hold a free, one-day conference about living with MDS in Minneapolis on September 24. Patients and their families are invited to learn about developments in the diagnosis and treatment of MDS from MDS experts. The program will run from 8:30 a.m. till 5 p.m., and a complimentary breakfast and lunch will be provided. Advance registration is required by September 19. For more information or to register, please see the AA&MDSIF website.

For a more detailed listing of upcoming MDS-related events, please check the MDS Beacon Events Calendar.

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Protein Interaction May Affect Revlimid Dosage – Results from a Phase 1 clinical trial indicate that P-glycoprotein, which removes certain drugs from cells into the bloodstream, interferes with Revlimid (lenalidomide) dosage in the body. High P-glycoprotein levels may cause drug resistance in cancerous cells. Revlimid is currently approved in the U.S. for use in patients with multiple myeloma and transfusion-dependent, lower-risk myelodysplastic syndromes (MDS) patients with a deletion in chromosome 5. In the study, researchers evaluated the activity and safety of Revlimid in combination with Torisel (temsirolimus) in multiple myeloma patients. They found that the concentration of Revlimid in patients’ bloodstreams was higher than expected, which experiments later showed was due to P-glycoprotein removing Revlimid from cancerous cells. The addition of Torisel resulted in a lower rate of Revlimid removal, which the study’s researchers pointed to as evidence of interaction between Revlimid and Torisel via P-glycoprotein. For more information, please see the study in the Journal of Clinical Oncology (abstract) or the Ohio State University Medical Center press release.

Modified Versions Of Ecstasy May Possess Anti-Cancer Activity – Researchers have made modified forms of the illegal drug ecstasy (MDMA) that kill white blood cell cancers 100 times more effectively than ecstasy itself.  Ecstasy has been known to kill cancers involving white blood cells, such as multiple myeloma, leukemia, and lymphoma.  However, the doses of ecstasy necessary to treat cancer would be lethal to the patient.  The new, more potent forms have the potential to be safe at therapeutic doses.  For more information, see the study in the journal Investigational New Drugs (abstract).

Webinar On Making Treatment Decisions In MDS – On September 1, the Aplastic Anemia & MDS International Foundation (AA&MDSIF) is hosting a webinar on making treatment decisions in MDS. Dr. Mikkael Sekeres of the Cleveland Clinic will lead the webinar, which starts at 1 p.m. EST. Dr. Sekeres will first review the diagnostic and prognostic criteria used for MDS and the available treatment options. He will then present a simulated patient journey to discuss his treatment decisions, starting at diagnosis. At the end of the webinar, there will be time for questions. For more information or to register, please see the AA&MDSIF website.

For a more detailed listing of upcoming MDS-related events, please check the MDS Beacon Events Calendar.

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Phase 1 Trial With BP-100-1.01 Continues As Researchers Recruit More Patients – The biotechnology company Bio-Path Holdings announced last week that the first patients have been treated with its developmental drug BP-100-1.01 (liposomal Grb-2) during a Phase 1 clinical trial. BP-100-1.01 is being investigated as a treatment for blood cancers, including myelodysplastic syndromes (MDS). According to Bio-Path Holdings, the drug was well tolerated and did not result in any treatment-related serious side effects. Despite using a low dose, the drug demonstrated anti-leukemia activity. The trial continues to recruit new patients, who will begin treatment with higher doses. For more information or to participate in the clinical trial, please see the Bio-Path press release and the clinical trial description.

MDS Experts Provide Recommendations On Prevention And Management Of Exjade-Related Gastrointestinal Side Effects – A panel of MDS experts have developed recommendations on the prevention and management of gastrointestinal problems caused by Exjade (deferasirox) treatment. Exjade removes excess iron from the body, which is a common complication associated with long-term transfusion dependence. The panel recommends that MDS patients take Exjade as a fasting dose 30 minutes prior to breakfast or dinner. In order to manage diarrhea, the most common Exjade-related side effect, MDS patients should remain hydrated, discontinue laxative use, take Exjade at night, and begin using the anti-diarrhea drug loperamide (Imodium). If the severity of diarrhea increases, the experts advise reducing the dosage or discontinuing Exjade treatment. To manage abdominal pain or vomiting, patients should take Exjade in the evening (not before bedtime) and refrain from solid foods two hours after treatment. For more information, please see the guidelines in Leukemia Research (abstract).

Free Conference for MDS Patients And Their Families In Chicago – On August 20, the Myelodysplastic Syndromes Foundation will offer a free one-day conference in Chicago for MDS patients and their caregivers. The conference starts at 10 a.m. with a patient support group discussion. After lunch, new therapies and patient treatment options will be discussed, with time for a question and answer segment. For more information or to register, please see the MDS Foundation website.

For a more detailed listing of MDS-related events, please check the MDS Beacon Events Calendar.