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Promacta Clinical Trial Begins Recruiting MDS Patients – A Phase 1/2 clinical trial studying Promacta (eltrombopag) for the treatment of low platelet counts is now recruiting participants with myelodysplastic syndromes (MDS) or acute myeloid leukemia. The study will evaluate the efficacy and safety of Promacta in patients who are ineligible to receive Vidaza (azacitidine), Dacogen (decitabine), intensive chemotherapy, or stem cell transplantation. Promacta is currently approved for patients with chronic immune thrombocytopenic purpura who have low platelet counts and are at increased risk for bleeding. For more information, please see the clinical trial description for the study or call the MDS Foundation at 1-800-637-0839.

Arno Therapeutics Receives Funding To Support Development Of MDS Treatment – The pharmaceutical company Arno Therapeutics raised $15 million in investment funding to support clinical testing of its developmental compounds. One of these drugs, AR-67, is currently being tested in Phase 2 clinical trials for the treatment of MDS and brain cancer. AR-67 is a camptothecin-derivative like Hycamptin (topotecan) and Camptosar (irinotecan), which have been approved for the treatment of several cancers. Hycamptin is also being tested for MDS. For more information, please see the Arno Therapeutics website or the clinical trial descriptions for the AR-67 trials.

Living With MDS Conference For Patients And Families – A one-day conference for MDS patients and their families will be held on September 25 in Chicago. Participants will learn about diagnostic tests and procedures, treatment options, preparing for doctor appointments, and emotional and physical wellness. The program will run from 8:45 a.m. to 3:45 p.m. with complimentary breakfast and lunch. The conference is free to attend, but registration is required. For more information or to register, please see the Aplastic Anemia &MDS International Foundation website.

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

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U.K. Agency To Re-Assess Vidaza Use In Myelodysplastic Syndromes – The National Institute for Health and Clinical Excellence (NICE), the agency that decides which treatments will be funded for English and Welsh patients, will re-assess the use of Vidaza (azacitidine) in myelodysplastic syndromes (MDS) patients after Celgene, the manufacturer of Vidaza, and other organizations successfully appealed NICE’s recommendation that Vidaza not be used to treat MDS. The European Medicines Agency approved Vidaza for treatment of MDS in March 2009. For more information on the ruling, please see the related press release.

Webinar: What’s On The Horizon For MDS Treatment? – The Aplastic Anemia and MDS International Foundation will be holding a free webinar entitled “What’s on the Horizon for MDS Treatment?” at 3 p.m. EST today. Dr. David Steensma of Harvard University will be presenting new and emerging therapies for MDS and their implications on patient survival. For more information and to register, please see the Aplastic Anemia & MDS International Foundation website.

4^th Annual Jack Ingold Memorial Golf Outing – The 4^th Annual Jack Ingold Memorial Golf Outing will take place on August 16 at 10 a.m. at The Club at Nevillewood in Presto, PA, to raise funds for the Leukemia & Lymphoma Society. This event includes lunch, golfing, dinner, cocktails, live music, and more. For more information, please see the Leukemia & Lymphoma Society website.

For a more detailed listing of MDS related events, please check the MDS Beacon events calendar.

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U.K. Agency Does Not Recommend Vidaza For MDS Patients – The National Institute of Clinical Excellence (NICE), the agency that decides which treatments will be funded for patients in England and Wales, released a final draft guidance on March 3 recommending that Vidaza (azacitidine) not be used to treat MDS. Although Vidaza may extend survival by nine months compared to standard therapy for MDS, NICE concluded that due to the drug’s high cost it cannot be recommended as a cost-effective treatment. The final guidance deciding the use of Vidaza in the U.K. will be released in May. Celgene, the company that produces Vidaza, has stated it will appeal the NICE decision. The European Medicines Agency approved Vidaza for treatment of MDS in March 2009. For more information, see the NICE Web site and related Beacon news.

How to Understand Your Lab Results And What They Mean – The Leukemia & Lymphoma Society (LLS) is sponsoring an event to help blood cancer patients understand their blood test results. Dr. Thomas Butler will be leading the discussion on March 17 from 7 p.m. to 8 p.m. at the Virginia Hospital Center in Arlington, VA. This event is free, and light refreshments will be served. To attend, please register through the LLS by March 15.

MDS Webinar: Treating Lower-Risk MDS – The Aplastic Anemia & MDS International Foundation (AA&MDSIF) is sponsoring a webinar, including lecture and case studies, to present current treatment options for lower-risk MDS patients. Dr. Mikkael Sekeres of the Cleveland Clinic will explain the differences between higher-risk and lower-risk MDS, review the factors that influence specific treatment decisions, and discuss treatments that are under development. The webinar will be held on Thursday, March 18 beginning at 3 p.m. EST. For more information or to register, please see the AA&MDSIF Web site.

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

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Researchers Find Possible Gene Target for MDS Therapy - Researchers from the Van Andel Research Institute in Grand Rapids, Michigan, report that the gene RhoB plays an important role in the development of myelodysplastic syndromes (MDS).  They found, in particular, that MDS progresses more rapidly when levels of the protein produced by RhoB are low.  There is hope that examining RhoB levels in MDS advanced patients may help in the selection of the most appropriate treatments, and that future therapies will target this gene to prevent MDS progression.  For more details, please see the related press release.

FDA Advisory Committee Set to Review Ferriprox - (See important update immediately below) On October 6, the U.S. Food and Drug Administration (FDA) Oncology Drugs Advisory Committee (ODAC) will review the iron chelating agent Ferriprox (deferiprone) for possible marketing approval in the U.S.  Ferriprox is approved for sale in a number of countries outside of the U.S., and is used to treat iron overload in patients who require frequent blood transfusions.  The meeting will include presentations by Ferriprox manufacturer ApoPharma Inc. and the FDA staff as well as questions from the public.  The FDA usually follows the advice it receives from its advisory committees, but it is not required by law to do so.  For more information, please see the meeting’s draft agenda and the Ferriprox Web site (intended for non-U.S. visitors).

Update: The FDA has announced that the Ferrirprox review meeting has been cancelled “to allow time for the FDA to review and resolve several outstanding issues.”  The agency says that it intends to continue evaluating Ferriprox, and it will reschedule the ODAC meeting “as needed.”

Living With Myelodysplastic Syndromes One-Day Conference – The Moffitt Cancer Center  and the Aplastic Anemia & MDS International Foundation (AA&MDSIF) are sponsoring a free opportunity to learn about the latest MDS diagnostic and treatment information, and ask questions of leading MDS researchers.  This one-day conference will be on Saturday, October 10, at the Moffitt Cancer Center in Tampa, Florida, from 9 a.m. to 4 p.m.  Breakfast and lunch are provided.  Space is limited, so please register by October 2.  For more information, please visit the AA&MDSIF Web site.

MDS Patient & Family Forum – Participate in a forum organized by the Myelodysplastic Syndromes Foundation on Tuesday, October 6, in Ontario, Canada.  Dr. Karen Yee from Princess Margaret Hospital will be providing information pertinent to MDS patients and their families.  Individuals must register by calling 1-800-MDS-0839 or emailing dmurray@mds-foundation.org.  For a listing of other upcoming U.S. and international forums, please see the MDS Foundation Web site.

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