The MDS Foundation is a multi disciplinary, international organization devoted to the prevention, treatment, and study of the myelodysplastic syndromes. The organization is based upon the premise that international cooperation will accelerate the process leading to the control and cure of these diseases.

If you wish to support the work of the Foundation in the battle against myelodysplastic syndromes, please remember us and consider donating all year long. All donations are tax-deductible.

MAKE A DONATION NOW.

Clinical Research Trial with Eltrombopag – Now Open for Accrual
Thrombocytopenia in patients with Myelodysplastic Syndromes

We would like to announce a phase I/II study (PMA112509) of eltrombopag in thrombocytopenic subjects with advanced Myelodysplastic Syndromes (MDS) or secondary Acute Myeloid Leukemia after MDS (sAML/MDS), or de novo AML.

For further information regarding key eligibility criteria, please contact the MDS Foundation at 1-800-MDS-0839 or email ahassan@mds-foundation.org.

Amgen Global Phase 2 MDS Study with Romiplostim – Open for Accrual Thrombocytopenia in patients with Myelodysplastic Syndromes (MDS)

This is a Phase 2, multicenter, randomized, double blind, placebo controlled study designed to assess the efficacy and safety of romiplostim (formerly, AMG 531) treatment in thrombocytopenic MDS subjects. The study is composed of a 26-week placebo controlled test treatment period (romiplostim versus Placebo), a 4 week interim wash-out period, a 24-week placebo controlled extended treatment period, and a 4-week follow-up period. During the interim wash-out period, a bone marrow biopsy will be performed in the absence of growth factor to assess changes in the marrow. In the extended treatment period, safety assessments will continue and subjects will be allowed to receive any standard of care treatments for MDS.

Contact the Amgen Call Center at 866-572-6436 or click here for additional information and to view the active sites.

Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study (TELESTO)

FDA Approves Five-Day Dosing Regimen for Dacogen^® (decitabine) for Injection, Offering a
New Outpatient Dosing Option for Myelodysplastic Syndromes (MDS)

Woodcliff Lake, NJ, March 11, 2010 – Eisai Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved a five-day dosing regimen for Dacogen^® (decitabine) for Injection to treat patients with myelodysplastic syndromes (MDS), a group of bone marrow diseases that alter the production of functional blood cells.

The new outpatient dosing option provides physicians and patients with the flexibility of a dosing regimen with a reduced infusion time. Dacogen is the only hypomethylating agent approved for a five-day dosing regimen.

Click here to read the full press release

Petition calling for cancer drug Vidaza to be made available on the NHS was presented to the Prime Minister of London

From left to right:
Dr Samir Agrawal, Senior Lecturer and Honorary Consultant in Hematology, Barts and the London NHS Trust
and representative from UK MDS Forum who led the petition
Sophie Wintrich, Patient Liaison at the MDS Foundation
Professor Rodney Taylor, MDS Patient and representative of MDS UK Patient Support Group
Janet Hayden, Myeloid Clinical Nurse Specialist, Kings College Hospital

17th May 2010 - Representatives from UK MDS Forum, King’s College Hospital, MDS Foundation and MDS UK Patient Support Group delivered a petition to the Prime Minister in London, to demand care comparative with that available in most other European countries. They collected 688 signatures.

Click here to read the full press release

MDS Patient and Family Support
Stanford Cancer Center in Palo Alto, CA

Click here for more information

A new bone marrow drug alternative to chemotherapy is saving my life.
So why has it been rejected by NICE?

Click here to read the article

THE MDS FOUNDATION SAYS NICE DECISION AGAINST VIDAZA^® DENIES ESSENTIAL TREATMENT OPTION AND DENIGRATES THE VALUE OF PATIENTS’ LIVES

VIDAZA is the only licensed drug shown in clinical trials to improve survival
Crosswicks, NJ, USA (4 March 2010) — The Myelodysplastic Syndromes (MDS) Foundation today issued a statement highly critical of a draft decision not to make available to UK patients the only drug approved by the European Medicines Agency (EMEA) for MDS.

Click here to read the full press release

In The News…

Professor Rodney Taylor, an MDS Patient, was interviewed on BBC Radio’s Today Programme (4 March 2010) where he spoke about the need for the NICE HTA process to be more flexible in the evaluation of medicines for rarer cancers that affect only small patient groups.

NICE says no to bone marrow disease drug  BBC News

Life extending cancer drug to be banned on NHS as too expensive Daily Telegraph

Charity disappointed by drugs news Press Association

UK cost body spurns Celgene rare blood cancer drug Reuters

UK’s NICE Says Celgene Drug Too Expensive For NHS Wall Street Journal

NICE Appraisal of Azacitidine for Myelodysplastic Syndromes PharmaLive

Vidaza’s future in the NHS bleak after cost watchdog says ‘no’ PharmaTimes

Research Studies Seeking Volunteers

The Behavioral Medicine Research Team at Eastern Michigan University is researching whether adult bone marrow or stem cell transplant survivors feel a need for help from mental health professionals, where they turn to for help, and how beneficial that help is. They are also conducting research about the experiences, challenges and reactions of spouses or partners who are caregivers for BMT and stem cell transplant patients.

Click here for further information about being a Research Study Volunteer

Click here for further information about the Spouse or Partner of a Bone Marrow or Stem Cell Patient Study

The MDS Foundation says Newly Published Study Finds VIDAZA^®
Prolongs Survival in Acute Myeloid Leukemia

Publication Could Encourage Physicians to Offer Treatment to Patients with this Difficult Cancer
Crosswicks, NJ (February 2, 2010) — The Myelodysplastic Syndromes (MDS) Foundation says a study published this week in the Journal of Clinical Oncology concludes VIDAZA (azacitidine) “prolongs survival and is well tolerated” in patients with acute myeloid leukemia (AML) - an aggressive form of leukemia that in many cases progresses from MDS. The study looked at older patients with a median age of 70. These are patients who have had “no truly adequate treatments,” however the study found that half of the patients treated with VIDAZA survived at least two years, compared to only 16% of patients who received conventional care.

Click here for more information

Celgene Corp (CELG: News ) said Health Canada has approved VIDAZA, an important treatment option for advanced forms of a group of serious blood cancers.

According to the company, the pivotal clinical trial, AZA-001 demonstrated that the two-year survival rate almost doubled with VIDAZA treatment compared to conventional care regimens. This data prompted the Joint Oncology Drug Review, the interprovincial body that reviews and makes listing recommendations for cancer drugs, to grant VIDAZA priority review status - only the second such designation to be granted this year.

The company also noted that VIDAZA will be commercially available in Canada in January 2010.

NICE Publication preliminary decision to not recommend Azacitidine to NHS Patients

On 3rd August 2009, NICE published their preliminary decision not to recommend the use of Azacitidine to treat NHS patients experiencing Myelodyplastic Syndromes, Chronic Myelomonocytic Leukaemia, and Acute Myeloid Leukaemia.

Click here to find out how you can help and sign the UK MDS Forum petition.

FDA Accepts sNDA for Alternative Dosing Regimen for Dacogen® (decitabine for injection) to Treat Patients with Myelodysplastic Syndromes (MDS)

Woodcliff Lake, NJ, July 8, 2009 – Eisai Corporation of North America today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s supplemental new drug application (sNDA) for an alternative five-day dosing regimen for Dacogen^® (decitabine for injection) to treat patients with myelodysplastic syndromes (MDS). MDS is a potentially life-threatening group of bone marrow diseases that limit the production of functional blood cells.

Click here for more information

New Phase III Trial in North America Currently Enrolling Patients with Low- or Intermediate-1 – Risk MDS

Contact Lisa Nardelli for further information; e-mail: LISA.NARDELLI@MOFFITT.ORG or phone: 813-745-4731

Click here for details

The MDS Foundation Says that NCCN Upgrades Epigenetic Drug Vidaza^®
As First "Preferred" Treatment For High-Risk Patients
National Comprehensive Cancer Network says new low-intensity therapies prolong stable disease and decrease need for transfusions
VIDAZA shown in clinical trials to improve survival

Click here for more information

MDS Quality-of-Life Survey
Please help us by completing the survey now!  

Click here to complete the survey

THE INTERNATIONAL MYELOMA FOUNDATION, THE MDS FOUNDATION AND A COALITION OF PATIENT ADVOCACY ORGANIZATIONS CALL FOR UPDATED RULES FOR REIMBURSEMENT, ACCESS AND APPROVALS FOR NEW AND EXISTING CANCER TREATMENTS

--Patient “Statement of Principles” to be Unveiled at the Global ASCO Cancer Meeting in Orlando, Florida--

To read more click here

Healthcare policy makers are meeting in the nation’s capital and around the country to discuss health care reform, and the need to provide appropriate care, including preventive care, for all those who need it.  Recently, at the largest meeting of oncology doctors, the 45th Annual Meeting of the American Society for Clinical Oncology (ASCO), one of the key themes was the impact of financial issues on the use of cancer therapies, including issues of cost, access, reimbursement and how they affect compliance.

There is a vast inequity of access to and reimbursement for cancer medications. Every cancer patient is unique and a treatment that works for one patient may not work for another. Patients should have access to the treatments that are recommended by their physician and equally reimbursed regardless of the type of therapy or the mechanism of delivery.

On behalf of the MDS Foundation and other blood cancer groups, including the International Myeloma Foundation and the Tackle Myeloma Foundation, we propose the following principles to advance access to treatments while reducing healthcare costs, improving prevention, and encouraging innovation.  Please join us in our efforts by signing the petition. This petition will be sent to key healthcare policy makers in the hopes of encouraging legislation that moves these important principles forward.

THE CANCER PATIENT STATEMENT OF PRINCIPLES: Prevention, Innovation, Access, and Early Approvals Please show your support by signing the petition

Your Help is Needed for Important Research Investigating the Genetics of Familial MDS/AML

If you or anyone you know has a familial history of MDS/AML please contact us at 1-800-MDS-0839 or email patientliaison@mds-foundation.org. While this is extremely rare, there is an ongoing study and we are trying to help locate these rare cases. Thank you.

100 Questions & Answers About Myelodysplastic Syndromes By Jason Gotlib, MD, MS & Lenn Fetcher, RN, BSN Now available from the Foundation. For your copy please call 1-800-MDS-0839

PUBLISHED DATA CONFIRMS VIDAZA^® SIGNIFICANTLY EXTENDS
SURVIVAL IN PATIENTS WITH THE MALIGNANT CONDITION MDS

VIDAZA Restores Gene Function to Double Survival and Increase Transfusion Independence

Click here to view the press release

FDA GRANTS ORPHAN DRUG DESIGNATION TO CHEMGENEX’S OMACETAXINE FOR THE TREATMENT OF MYELODYSPLASTIC SYNDROMES

MELBOURNE, Australia, and MENLO PARK, California U.S.A. (January 20, 2009). ChemGenex Pharmaceuticals Limited (ASX:CXS and NASDAQ:CXSP) announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation to omacetaxine for the treatment of Myelodysplastic Syndromes (MDS).

Orphan drug designation is intended to support the clinical development of new drugs in diseases affecting less than 200,000 people in the United States. The FDA often provides technical and financial assistance to expedite and optimize drug development and on approval, grants a seven year period of market exclusivity.

Click here to view the complete article

EISAI WILL INITIATE FIRST HEAD-TO-HEAD STUDY COMPARING DACOGEN® (DECITABINE FOR INJECTION) AND VIDAZA® (AZACITIDINE) IN PATIENTS WITH MYELODYSPLASTIC SYNDROMES
Randomized Comparator Study to Evaluate Efficacy of Commercially Available Hypomethylating Agents

Eisai Corporation of North America today announced that it plans to initiate the first clinical trial evaluating the activity of Dacogen® (decitabine for injection) compared to Vidaza® (azacitidine) in adult patients with intermediate-1, intermediate-2 or high-risk myelodysplastic syndromes (MDS), a potentially life-threatening group of bone marrow diseases that limit the production of functional blood cells. The head-to-head trial will be conducted in the United States and will directly compare Dacogen® to Vidaza® with a primary endpoint of complete response rate (including marrow complete response).

Click here to view the complete article

DACOGEN^® (DECITABINE FOR INJECTION) DATA PRESENTED ON A PHASE II CLINICAL TRIAL IN ELDERLY PATIENTS WITH ACUTE MYELOID LEUKEMIA (AML)
Response Observed Across All Subtypes of AML, Including Those with Poorest Prognoses

San Francisco, CA, December 8, 2008 – Eisai Corporation of North America announced data from a Phase II trial evaluating a five-day dosing regimen of Dacogen^®(decitabine for injection) in acute myeloid leukemia (AML), the most common form of leukemia. The study involved elderly patients with AML, who often have limited options due to comorbidities and are typically considered ineligible for standard induction chemotherapy. These data were presented today at the American Society of Hematology (ASH) 50th Annual Meeting.

Dacogen^®is indicated for treatment of patients with myelodysplastic syndromes (MDS), including those with refractory anemia with excess blasts (immature or unformed blood cells) in transformation (RAEB-T - now re-classified by World Health Organization [WHO] as AML). Phase II and III clinical trials evaluating Dacogen^®in patients with AML are currently underway.

Click here to view the complete article

DATA PRESENTED AT ASH ILLUSTRATES THAT
CONTINUED TREATMENT WITH VIDAZA CAN BENEFIT MDS PATIENTS

The Myelodysplastic Syndromes (MDS) Foundation announced today that a new analysis of the AZA-001 phase III clinical trial demonstrates that continued treatment with VIDAZA (azacitidine) can improve response rates for higher-risk MDS patients.

Leading hematologist Dr. Lewis Silverman of Mount Sinai Medical Center presented the analysis at the 50th Annual Meeting of the American Society of Hematology. The results showed that 51% of patients in the trial responded to treatment with Vidaza. Of those patients, almost half achieved an improved response when treatment with VIDAZA was continued for an additional four cycles.

Click here to view the complete article

Exjade^® benefits chronically transfused patients by significantly reducing toxic iron that can damage key organs, according to landmark trial

East Hanover, NJ, December 8, 2008 — New data from the largest prospective trial in iron chelation demonstrate the efficacy and safety of Exjade® (deferasirox) in treating chronic transfusional iron overload, a potentially life-threatening condition for patients who have had multiple blood transfusions to treat underlying anemias, including beta-thalassemia and myelodysplastic syndromes (MDS).

The EPIC cardiac substudy showed that Exjade removed iron from the heart in beta-thalassemia patients, based on a statistically significant improvement in T2* magnetic resonance imaging, a validated technique to assess cardiac iron content (P<0.0001).  The one-year substudy included 114 beta-thalassemia patients with cardiac iron overload, the leading cause of death in these patients.

Click here to read complete press release

AMGEN TO PROVIDE NEW EVIDENCE AS PART OF A FORMAL RECONSIDERATION
OF CMS’ NATIONAL COVERAGE DETERMINATION ON ESAs

Revised Class Labeling and Data on Increased Transfusions are Part of the Growing New Evidence to Revise the Policy

Click here to read complete press release

THE MDS FOUNDATION SUPPORTS VIDAZA’S RECOMMENDATION FOR EUROPEAN APPROVAL

Vidaza Can Help Patients Throughout Europe Live Longer with a Better Quality of Life

Click here to read complete press release

THE MDS FOUNDATION SUPPORTS THE FDA’S DECISION TO
EXPAND VIDAZA LABEL TO INCLUDE OVERALL SURVIVAL DATA

Results from Clinical Trial Found that VIDAZA is the Only Agent to
Extend Survival in Patients with Higher-Risk Myelodysplastic Syndromes

Click here to read complete press release

Genzyme Corporation is conducting a global phase 2 study to further understand
the effect Rabbit Antithymocyte Globulin (rATG) has on MDS.

A Phase II Study of Efficacy of Rabbit Anti-thymocyte Globulin (rATG) in Patients with
Low and Intermediate-1 Risk Myelodysplastic Syndrome

Click here to read complete press release

FDA APPROVES NPLATE™ FOR LONG-TERM
TREATMENT OF ADULT CHRONIC ITP

First and Only Approved Platelet Producer Represents New Treatment Approach for Serious Chronic Autoimmune Disorder

Amgen to Launch Nplate™ NEXUS Program to Provide Treatment Access and Patient Support Programs
THOUSAND OAKS, Calif., (August 22, 2008) – Amgen Inc. (NASDAQ: AMGN) today announced that the United States (U.S.) Food and Drug Administration (FDA) has approved Nplate™ (romiplostim), the first and only platelet producer for the treatment of thrombocytopenia in splenectomized (spleen removed) and non-splenectomized adults with chronic immune thrombocytopenic purpura (ITP). Nplate, the first FDA-approved peptibody protein, works by raising and sustaining platelet counts, representing a novel approach for the long-term treatment of this chronic disease.

Click here for the full media details.

Virtual Microscopy in MDS-AML: The Key to Enhancing Classification This CME Accredited Program is now available! Supported by a grant from Celgene Corporation Click here to view the program

Once-Daily Exjade^® Shown to Remove Iron From the Heart, According to Data Presented at ASH

• Approximately 78% of beta-thalassemia patients had decreases in cardiac iron and 90% had decreases in liver iron after six months, interim data show
• Study in sickle cell disease (SCD) patients with iron overload showed continued safety and efficacy over two years
• Safety and efficacy demonstrated in lower-risk myelodysplastic syndromes (MDS) patients
  

Click here for complete media release

Epigenetics: Playing the Genetic Score

Epigenetics is challenging long-held notions that our genes are our destiny and radically changing the way researchers think about the development of certain illnesses. As science come to understand the way epigenetic processes silence genes, it is given rise to promising new therapies for diseases such as MDS.

Watch The Journal of Life Sciences webcast

MDS Practice and Treatment Survey

The MDS Foundation recognizes that data on many aspects of MDS worldwide is sketchy or nonexistent. While individual investigators have developed databases to track MDS within their individual sites or working groups, that information is not located within one easily accessible database.

To assist in the development of useful information, The Foundation has recently initiated the first Patient Registry and data from the Foundation’s Centers of Excellence are currently being entered.

Since it will be some time before these data are mature and usable, The Foundation has attempted to design a survey that we hope will assist in describing some of the issues related to MDS worldwide as well as the treatments being utilized in this disease. A pilot of this survey has already been completed with some selected Centers of Excellence. While we know that this information is, in most instances, based on subjective criteria it can assist in identifying educational and research opportunities in the near term and until more accurate data is available.

The results of this expanded survey will be shared with each of our Centers of Excellence and used by the Foundation to assess new educational and research opportunities. Thank you in advance for your consideration in completing this form.

Click here for the Nursing Practice & Treatment print survey

Click here for the Physician print survey

Click here to complete the Physician survey online

Click on a flag to take the Nursing Pratice & Treatment survey in your language

Insurance and Reimbursement Resources for MDS Patients in the USA

We have assembled a listing of assistance programs available to MDS patients. It is important to know that there is support for those who cannot afford medicine or other healthcare costs. Click on the item of interest below for more information or click here to request an Insurance and Reimbursement Booklet. We hope this new resource will be beneficial in helping you with your medical needs.

Click here view and print the insurance and Reimbursement Resources for MDS Patients guide

Ask about iron is an education program for those at most risk for iron overload disease due to blood transfusions.

Click here for more information

MDS FOUNDATION RESOURCE CENTER
Understanding The Myelodysplastic Syndromes

Visit This New Resource for Healthcare Professionals

Understanding MDS: A Primer for Practicing Clinicians

Segment 1, The Past & Present in MDS is available free of charge. This segment introduces the group of bone marrow disorders called MDS and provides a history of myelodysplastic syndromes up to the present time.

Segment 2, Clinical Presentation, Diagnosis, and Pathology is available free of charge. This segment provides an insightful overview of the clinical picture of adult and pediatric patients with primary or secondary MDS. Diagnosing MDS based on morphologic, cytogenetic, and pathologic criteria are also discussed.

Segment 3, Ineffective Hematopoiesis: Considerations in Diagnosis and Treatment is available free of charge. This segment provides insight into the pathogenic mechanisms that contribute to the development of MDS, including the altered bone marrow microenvironment of MDS in terms of cells, cytokines, growth factors, receptors, and microvasculature; dyserythropoiesis in MDS, and therapeutic targets and approved drugs for the treatment of MDS.

Segment 4, Anemia in MDS: Survival, QoL, and Treatment Options
Segment 4 is an overview of supportive care with a focus on RBC transfusions and its effect on the morbidity and mortality of MDS patients. This segment also looks at the quality of life issues from the perspectives of the physical, functional, emotional, social and cost impacts on the patient with MDS.

This multi-segment program is available in the following languages: English, French, German, Italian, Japanese and Spanish.

Click here to register

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