According to a recent MoneyTree Report by PricewaterhouseCoopers and the National Venture Capital Association, investments in biotech’s life sciences sector declined 15 percent in 2008 compared to 2007. “Funding for early-stage drug development is limited, especially for rare diseases like multiple myeloma, and continues to decline in today’s economy. We have maintained our commitment to MMRF Biotech Investment Awards in 2008 at the same level as 2007 because they help rapidly advance important research and early drug development that may not have otherwise been possible,” said Louise M. Perkins, Ph.D., Chief Scientific Officer of the MMRF.

The 2008 MMRF Biotech Investment Awards will support the early development of two novel approaches: Aileron Therapeutics’ Stapled Peptide, borrows from nature a small piece of a protein that restores programmed cell death in multiple myeloma cells; and Astex Therapeutics’ proprietary cyclin-dependent kinase (CDK) inhibitor, a type of drug that blocks the uncontrolled proliferation of multiple myeloma cells.

“We are honored to be selected for a MMRF Biotech Investment Award to support the advancement of our Stapled Peptide drugs for the treatment of multiple myeloma. Support of respected organizations like the MMRF plays a critical role in advancing breakthrough technologies such as ours into the clinic,” said Joseph A. Yanchik III, CEO of Aileron Therapeutics.

“The MMRF BIA grant bridges a critical funding gap that will support the clinical development in myeloma of our promising novel compound AT7519, our proprietary CDK inhibitor,” said Harren Jhoti, Ph.D., CEO of Astex Therapeutics and a recipient of the MMRF Biotech Investment Award.

The MMRF Biotech Investment Awards program has already seen impressive results. Semafore, one of the first grantees of the Biotech Investment Awards, was awarded funding to support the early development of its targeted PI3 kinase (PI3K) inhibitor, SF1126, in multiple myeloma. This promising compound is now being studied in a clinical trial through the MMRF’s sister organization, the Multiple Myeloma Research Consortium (MMRC).

About Multiple Myeloma

Multiple myeloma is an incurable cancer of the plasma cell. The five-year relative survival rate for multiple myeloma is approximately 35%, one of the lowest of all cancers. In 2008, an estimated 19,920 adults (11,190 men and 8,730 women) in the United States were diagnosed with multiple myeloma and an estimated 10,690 people died from the disease.

About Aileron Therapeutics

Aileron Therapeutics is a biopharmaceutical company developing a breakthrough class of drugs called Stapled Peptides. This new class of drugs represents the first general solution for modulating intracellular protein-protein interactions, which have been identified as critical control points for most human diseases. Aileron’s new therapeutic modality creates a unique opportunity to exploit potentially thousands of currently “undruggable” targets with applications in all human diseases. For further information on Aileron, please visit the company’s website: http://www.aileronrx.com.

About Astex Therapeutics

Astex Therapeutics is a UK-based biotechnology company that discovers and develops novel small molecule therapeutics. Using its pioneering fragment-based drug discovery platform Pyramid™, Astex has built a pipeline of five molecularly-targeted oncology drugs, of which three are currently being tested in clinical trials and two are in pre-clinical development. In addition to its proprietary research programs, Astex’s productivity in lead discovery has been endorsed through numerous partnerships with major pharmaceutical companies, including AstraZeneca, Bayer-Schering, Boehringer Ingelheim, Novartis and Johnson and Johnson. For further information on Astex, please visit the company’s website: http://www.astex-therapeutics.com.

About the Multiple Myeloma Research Foundation (MMRF)

The Multiple Myeloma Research Foundation (MMRF) was established in 1998 as a 501(c)3 non-profit organization by twin sisters Karen Andrews and Kathy Giusti, soon after Kathy’s diagnosis with multiple myeloma. The mission of the MMRF is to relentlessly pursue innovative means that accelerate the development of next-generation multiple myeloma treatments to extend the lives of patients and lead to a cure. As the world’s number-one private funder of multiple myeloma research, the MMRF has raised over $110 million since its inception to fund nearly 100 laboratories worldwide. An outstanding 93% of funds raised go toward research and related programming. The MMRF has supported 40 new compounds and approaches in clinical trials and pre-clinical studies and has facilitated 17 clinical trials through its sister organization, the Multiple Myeloma Research Consortium (MMRC). For more information about the MMRF, please visit http://www.themmrf.org.

The three-pronged regimen of Velcade, lenalidomide (Revlimid) and dexamethasone – referred to as Rev/Vel/Dex – has achieved an overall response rate of 98 percent in 42 patients evaluated thus far in a Phase 1-2 trial, said Paul Richardson, MD, of Dana-Farber and the study’s principal investigator. He added that 52 percent of the patients had high quality responses (very good partial response or better), with 30 percent achieving complete response to date.

“These may be some of the best response rates we’ve seen to date with up-front therapies, and although these are preliminary results, they are extremely promising,” Richardson said. The patients were previously untreated when they received the Rev/Vel/Dex combination.

Velcade is a “smart” drug known as a proteasome inhibitor that blocks the myeloma cells’ waste disposal system, creating an accumulation of toxic compounds that poison the cell. Revlimid is a chemical relative of thalidomide that affects several pathways in cancer cells, including immune mechanisms and blood vessel growth to tumors. Dexamethasone is a steroid hormone that counters inflammation and is used to treat hematologic malignancies such as myeloma. Studies leading to the trial of the three drugs in combination were carried out at Dana-Farber.

While these are the first results from trials of Rev/Vel/Dex given as initial, first-line therapy for the blood cancer, the combination has already been shown effective for multiple myeloma patients who had relapsed following successful treatment or who had not responded to standard therapies.

Richardson also reported at the ASH meeting preliminary data from a Dana-Farber led multicenter Phase 2 trial of the combination in relapsed or refractory myeloma. “These data confirm the favorable side effect profile,” said Richardson, “and the response rate of 72 percent – including complete, partial, and minor responses – is very encouraging.”

The responses, he added, appear to be holding up well, with a duration of more than one year for some patients to date. Both trials will continue to enroll patients, and final results are expected next year.

In the study, lenalidomide and pomalidomide, immunomodulatory anticancer drugs, were both shown to be more effective than hydroxyurea at inducing HbF expression by erythrocytes derived in vitro from CD34+ cells from healthy individuals. In addition, the effects of pomalidomide and hydroxyurea on HbF expression were synergistic. As pomalidomide was able to induce HbF expression in CD34+ cells from patients with SCD, the authors suggested that it might provide a new therapy for SCD, either alone or in combination with hydroxurea. Furthermore, because the induction of HbF has been shown to be of some benefit to individuals with beta-thalassemia (a hereditary anemia caused by decreased beta-globin production), the authors also suggested that pomalidomide might be a good therapeutic for the treatment of beta-hemoglobinopathies other than SCD, such as beta-thalassemia.

“ASH promises to be a very exciting meeting for us, with a focus on the detailed presentation of data from the Vidaza overall survival study, which demonstrate an unprecedented survival benefit compared to conventional care regimens, as well as other compelling study data on Vidaza, Thalidomide and MGCD0103,” said Patrick J. Mahaffy, president and chief executive officer of Pharmion. “The data from 36 abstracts, including 23 posters and 13 oral presentations, to be presented demonstrates the progress of our pipeline and we are very enthusiastic about our participation this year.”

Studies presented at this year’s ASH meeting will report the complete Phase 3 data set from a study demonstrating that Vidaza® (azacitidine for injection) provides a significant survival benefit beyond that provided by conventional care regimens for higher-risk MDS patients. Other data to be presented at the meeting include alternate dosing schedules of Vidaza and the use of Vidaza as a maintenance therapy for patients with higher-risk MDS and AML.

Additionally, data from a study of Vidaza in combination with MGCD0103 in MDS and AML will be presented at ASH, as will poster presentations featuring study results from two ongoing single-agent Phase 2 MGCD0103 trials, one in refractory/relapsed classical HL patients and the other in diffuse large B cell lymphoma and follicular lymphoma, two forms of NHL.

“The data on Pharmion’s products being presented at ASH clearly demonstrate the importance of epigenetic therapy in the treatment of hematologic malignancies and Pharmion’s leadership position in the field of epigenetics,” said Andrew R. Allen, chief medical officer of Pharmion. “In particular, the data being presented confirming the clinical responses with Vidaza, MGCD0103 and their combination suggest a promising synergistic treatment approach using epigenetic therapies.”

Pharmion’s portfolio of epigenetic therapies includes three developmental programs: Vidaza and oral azacitidine, both DNA demethylating agents; MGCD0103, a HDAC inhibitor; and sirtuin inhibitors, Pharmion’s newest epigenetic research program. Pharmion currently markets Vidaza, the parenteral formulation of Azacitidine, in the U.S. and several additional countries for the treatment of patients with MDS, and as previously mentioned the full data set from the Phase 3 study of Vidaza’s effect on overall survival in higher-risk MDS patients will be presented at the meeting.

Data from a study evaluating the addition of Thalidomide to front-line melphalan/prednisone (MP) therapy in newly diagnosed elderly patients with multiple myeloma will be the subject of an oral presentation at ASH, where two oral presentations and three posters on Thalidomide studies will be presented.

The following data will be presented during the 49th Annual ASH Meeting and Exposition:

Vidaza®

Date / Time / Location: December 11, 2007; 7:30-9:00 a.m., Thomas B Murphy Ballroom 4, Georgia World Congress Center
Session: Myelodysplastic Syndromes: Advances in Therapeutic Options
Oral Session 7:30 a.m.: Azacitidine (AZA) Treatment Prolongs Overall Survival (OS) in Higher-Risk MDS Patients Compared with Conventional Care Regimens (CCR): Results of the AZA-001 Phase III Study (Abstract #817).
Oral Session 7:45 a.m.: Maintenance Treatment with Azacytidine for Patients with High Risk Myelodysplastic Syndromes or Acute Myeloid Leukemia in Complete Remission after Intensive Chemotherapy (Abstract #818).
Oral Session 8:00 a.m.: Results of the Initial Treatment Phase of a Study of Three Alternative Dosing Schedules of Azacitidine (Vidaza) in Patients with Myelodysplastic Syndromes (MDS) (Abstract #819).

Date / Time / Location: December 8, 2007; Viewing 9:00 a.m.-7:30 p.m.,
Presentation 5:30 -7:30 p.m., Hall B4 of Georgia World Congress Center
Session: Acute Myeloid Leukemias: Therapy, Excluding Transplantation I
Poster Board No. 65-I: Report of a Phase I/II Study of 5-Azacitidine and Cytarabine in Patients with Relapsed, Refractory Acute Myelogenous Leukemia (Abstract #911).

Date / Time / Location: December 8, 2007; Viewing 9:00 a.m.-7:30 p.m., Presentation 5:30-7:30 p.m., Hall B4 of Georgia World Congress Center
Session: Myeloproliferative Syndromes: Biological
Poster Board No. 699-I: Hypermethylation of CXCR4 Promoter, and Its Reactivation by Hypomethylating Agent, in CD34+ Cells from Primary Myelofibrosis Patients (Abstract #1545).

Date / Time / Location: December 8, 2007; Viewing 9:00 a.m.-7:30 p.m., Presentation 5:30-7:30 p.m., Hall B4 of Georgia World Congress Center
Session: Myelodysplastic Syndromes: Clinical Studies, Including Transplantation
Poster Board No. 603-I: Outcomes of MDS Patients with Chromosome 7 Abnormalities Treated with 5-Azacytidine (Abstract #1449).
Poster Board No. 605-I: A Phase II Study of Intravenous Azacitidine Alone in Patients with Myelodysplastic Syndromes (Abstract #1451).
Poster Board No. 606-I: Therapy of Myelodysplastic Syndrome (MDS) with Azacitidine Given in Combination with Etanercept: A Phase II Study (Abstract #1452).
Poster Board No. 612-I: Preliminary Results from a Phase I Study of Revlimid (Lenalidomide) in Combination with Vidaza (Azacitidine) in Patients with Advanced Myelodysplastic Syndromes (MDS) (Abstract #1458).

Date / Time / Location: December 9, 2007; Viewing 9:00 a.m.-8:00 p.m., Presentation 6:00-8:00 p.m., Hall B4 of Georgia World Congress Center
Session: Acute Myeloid Leukemias: Therapy, Excluding Transplantation II
Poster Board No. 25-II: 5-Azacytidine Augments the Cytotoxicity of Mylotarg toward AML Blasts In Vitro and In Vivo (Abstract # 1835).
Poster Board No 39-II: Treatment of AML with Azacytidine (AZA): Current Results of the French ATU Program (Abstract #1849).

Date / Time / Location: December 9, 2007; Viewing 9:00 a.m.-8:00 p.m., Presentation 6:00-8:00 p.m., Hall B4 of Georgia World Congress Center
Session: Myelodysplastic Syndromes: Molecular Biology and Pathogenesis
Poster Board No 638-II: PI-PLCbeta1 Expression in Patients with High-Risk Myelodysplastic Syndromes Is Affected by Azacitidine Treatment (Abstract #2448).

Date / Time / Location: December 9, 2007; Viewing 9:00 a.m.-8:00 p.m., Presentation 6:00-8:00 p.m., Hall B4 of Georgia World Congress Center
Session: Myelodysplastic Syndromes: Prognosis and Clinical Correlative Studies

Poster Board No. 656-II: FISH-Analyses of Circulating CD34+ Cells in MDS-Patients – A Suitable Method to Measure and Predict Response to 5-Azacytidine (Abstract #2466).

Date / Time / Location: December 9, 2007; Viewing 9:00 a.m.-8:00 p.m., Presentation 6:00-8:00 p.m.., Hall B4 of Georgia World Congress Center
Session: Disordered Epigenetic Regulation in Hematologic Malignancy
Poster Board No. 312-II: DNA Methylation Analysis of 807 Genes in Chronic Myeloid Leukemia and Acute Promyelocytic Leukemia (Abstract #2122).
Poster Board No. 402-III: Up-Regulation of miR-195 Expression Leads to Decreased Expression of Basic Fibroblast Growth Factor in CLL Patients Treated with DNA Methylation Inhibitors (Abstract #3183).

Date / Time / Location: December 10, 2007; 1:30-3:00 p.m., Rooms B216-B217, Georgia World Congress Center
Session: Thalassemia: Pre-Clinical and Clinical Advances:
Oral Session 1:45 p.m.: Neither DNA Hypomethylation or Changes in the Kinetics of Erythroid Differentiation Account for 5-Azacytidine’s Ability To Induce Human Fetal Hemoglobin (Abstract 572).

Date / Time / Location: December 10, 2007; 1:30 -3:00 p.m., Rooms A411-A412, Georgia World Congress Center
Session: Acute Myeloid Leukemias: Therapy, Excluding Transplantation-Novel Therapies
Oral Session 2:45 p.m.: Phase I/II Study of MGCD0103, an Oral Isotype-Selective Histone Deacetylase (HDAC) in Combination with 5-Azacitidine in Higher-Risk Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML) (Abstract #444).

Date / Time / Location: December 10, 2007; Viewing 10:30 a.m.-7:00 p.m., Presentation 5:00-7:00 p.m., Hall B4 of Georgia World Congress Center
Session: Clinical Care: Transplantation Regimen Toxicities and Engraftment II
Poster Board No. 231-III: A Dose and Schedule Finding Study of Maintenance Therapy with Low-Dose 5-Azacitidine (AZA) after Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for High-Risk AML or MDS (Abstract #3012).
Poster Board No. 232-III: Efficacy of Azacytidine (5-AC) Given as Maintenance of Salvage Therapy for Patients with Acute Leukemia Post Allogeneic Stem Cell Transplantation (HSCT) (Abstract #3013).

MGCD0103

Date / Time / Location: December 9, 2007; Viewing 6:00 p.m.-8:00 p.m., Presentation 6:00 p.m., Hall B4 of Georgia World Congress Center
Session: New Agents and Treatment Approaches in Non-Hodgkin Lymphoma
Poster Board No. 756-II: Isotype Selective HDAC Inhibitor MGCD0103 Decreases Serum TARC Concentrations and Produces Clinical Responses in Heavily Pretreated Patients with Relapsed Classical Hodgkin Lymphoma (Abstract #2566).
Poster Board No. 761-II: Treatment of Relapsed or Refractory Lymphoma with the Oral Isotype-Selective Histone Deacetylase Inhibitor MGCD0103: Interim Results from a Phase II Study (Abstract #2571).

Date / Time / Location: December 10, 2007; 1:30 -3:00 p.m., Rooms A411-A412, Georgia World Congress Center
Session: Acute Myeloid Leukemias: Therapy, Excluding Transplantation-Novel Therapies
Oral Session 2:45 p.m.: Phase I/II Study of MGCD0103, an Oral Isotype-Selective Histone Deacetylase (HDAC) in Combination with 5-Azacitidine in Higher-Risk Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML) (Abstract #444).

Date / Time / Location: December 9, 2007; Viewing 9:00 a.m.-8:00 p.m., Presentation 6:00-8:00 p.m., Hall B4 of Georgia World Congress Center
Session: Disordered Epigenetic Regulation in Hematologic Malignancy
Poster Board No. 320-II: Analysis of Class I and II Histone Deacetylase Fails To Identify a Human Leukemia Specific Expression Profile (Abstract # 2130).

Thalidomide

Date / Time / Location: December 9, 2007; 4:30-6:00 p.m.; Rooms C303-C305, Georgia World Congress Center
Session: Myeloma: Firstline Phase III Trials in Multiple Myeloma
Oral Presentation – 4:30 p.m.: Velcade-Thalidomide-Dexamethasone (VTD) vs Thalidomide-Dexamethasone (TD) in Preparation for Autologous Stem-Cell Transplantation (ASCT) in Newly Diagnosed Multiple Myeloma (Abstract #73).
Oral Presentation – 5:00 p.m.: Melphalan-Prednisone-Thalidomide (MP-T) Demonstrates a Significant Survival Advantage in Elderly Patients ?75 years with Multiple Myeloma Compared with Melphalan-Prednisone (MP) in a Randomized, Double-blind, Placebo-controlled Trial, IFM 01/01 (Abstract #75).
Oral Presentation – 5:45 p.m.: Melphalan-Prednisone-Thalidomide to Newly Diagnosed Patients with Multiple Myeloma: A Placebo Controlled Randomized Phase 3 Trial (Abstract #78).

Date / Time / Location: December 10, 2007; 7:30-9:00 a.m.; Rooms C303-C305, Georgia World Congress Center
Session: Myeloma: Frontline Therapy in Newly Diagnosed Multiple Myeloma
Oral Presentation – 7:45 a.m.: A Phase II Study of Velcade, Cyclophosphamide, Thalidomide and Dexamethasone as First-Line Therapy for Multiple Myeloma (Abstract #188).

Date / Time / Location: December 10, 2007; 1:30-3:00 p.m.; Rooms C306-C308, Georgia World Congress Center
Session: Autologous Transplantation for Myeloma: Induction, Mobilization, and Biologic Correlates
Oral Presentation – 2:00 p.m.: Incorporation of Thalidomide into Up-Front Double Autologous Stem Cell Transplantation (ASCT) for Multiple Myeloma Improves Outcome in Comparison with Double ASCT without Thalidomide. Analysis of Baseline Factors Predictive of Outcome (Abstract #447).

Date / Time / Location: December 10, 2007; 1:30-3:00 p.m.; Rooms C303-C305, Georgia World Congress Center
Session: Myeloma: Maintenance, Consolidation and Bone Disease in Multiple Myeloma
Oral Presentation – 1:45 p.m.: Consolidation with Bortezomib, Thalidomide, and Dexamethasone Induces Molecular Remissions in Autografted Multiple Myeloma Patients (Abstract #530).
Oral Presentation – 2:15 p.m.: Thalidomide-Dexamethasone vs Interferon-Dexamethasone as Maintenance Treatment after ThaDD induction for Multiple Myeloma: Final Analysis of a Prospective, Randomized Study (Abstract #532).

Date / Time / Location: December 9, 2007; Viewing 9:00 a.m.-8:00 p.m., Presentation 6:00-8:00 p.m., Hall B4 of Georgia World Congress Center Session: Myeloma: Relapsed and Refractory Multiple Myeloma
Poster Board No. 915-II: Longer Duration of Thalidomide Monotherapy Results in Improved Outcome in Relapsed/refractory Multiple Myeloma (Abstract #2725).
Poster Board No. 905-II: A Phase I/II Trial on Melphalan, Prednisone, Thalidomide, and Defribotide Combination in Relapsed/Refractory Multiple Myeloma Patients (Abstract #2715).
Poster Board No. 908-II: Prolonged Progression Free Survival Does Not Relate to Quality of Response to Treatment with Thalidomide in Patients with Relapsed Multiple Myeloma (Abstract #2718).
Poster Board No. 919-II: ThaDD-V Treatment for Patients with Relapsed/Refractory Multiple Myeloma: A Feasibility/Activity Study (Abstract #2729).
Poster Board No. 924-II: Effect of Thrombotic Events on Overall Survival in Patients with Newly Diagnosed Myeloma: Analysis from a Randomized Phase III Trial of Thalidomide plus Dexamethasone vs Dexamethasone in Newly Diagnosed Multiple Myeloma (E1A00) (Abstract #2734).

Date / Time / Location: December 10, 2007; Viewing 10:30 a.m.-7:00 p.m., Presentation 5:00-7:00 p.m., Hall B4 of Georgia World Congress Center
Session: Myeloma: Novel Therapies
Poster Board No 812-III: Thalidomide Combinations Improve Response Rates; Results from the MRC IX Study (Abstract 3593).

About Pharmion

Pharmion is a leading global oncology company uniquely focused on acquiring, developing and commercializing innovative products for the treatment of hematology and oncology patients in the U.S., Europe and additional international markets. Pharmion has a number of products on the market including the world’s first approved epigenetic drug, Vidaza(R), a DNA demethylating agent. For additional information about Pharmion, please visit the company’s website at http://www.pharmion.com.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995

This release contains forward-looking statements, including summary statements relating to the interim or preliminary results of clinical trials involving Vidaza, Thalidomide Pharmion, and MGCD0103. Such statements are based on current expectations and involve a number of known and unknown risks and uncertainties that could cause the final results to differ significantly from the results summarized by such statements. Preliminary results may not be confirmed upon final analysis of the detailed results of a trial and additional information relating to the safety, efficacy or tolerability of Pharmion’s products may be discovered upon further analysis of clinical trial data and upon review and analysis of additional clinical trial data from this or other clinical trials. Additional risks and uncertainties relating to Pharmion and its business can be found in the “Risk Factors” section of Pharmion’s Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2007, its Annual Report on Form 10-K for the year ended December 31, 2006 and in Pharmion’s other filings with the U.S. Securities and Exchange Commission. Forward-looking statements speak only as of the date on which they are made, and Pharmion undertakes no obligation to update publicly or revise any forward-looking statement, whether as a result of new information, future developments or otherwise. Pharmion also disclaims any duty to comment upon or correct information that may be contained in reports published by the investment community.

“The discoveries made by Drs. Croce, Vainchenker, and Beutler have altered the paradigm of medical thinking not only in hematology, but many other fields as well, and impacted the lives of many patients for the better,” said ASH President Andrew Schafer, MD, of the New York Presbyterian-Weill Cornell Medical Center. “It is my honor to recognize these outstanding individuals for their achievements.”

With his discovery of the workings of the MYC oncogene in Burkitt lymphoma, Dr. Croce was one of the first to make a case for the genetic basis of cancer. A prolific researcher, he later identified several other genes and their mechanisms of action responsible for the pathogenesis of lymphoma, leukemia, and many other cancers. Dr. Croce is currently Professor and Chairman of the Department of Molecular Virology, Immunology, and Medical Genetics and Director of the Human Cancer Genetics Program at The Ohio State University School of Medicine in Columbus.

Dr. Vainchenker will be recognized for his research on the genetic basis of myeloproliferative diseases, which result in an overproduction of blood cells in the bone marrow. He was the first to find that a single mutation of the JAK2 gene commonly occurred in patients with three distinct disorders: polycythemia vera, idiopathic myelofibrosis, and essential thrombocythemia, a discovery which has profound implications for potential therapies. Dr. Vainchenker is currently INSERM Directeur de Recherche Exceptionnel and Director of the INSERM Unit 790 at the Institut Gustave Roussy in Villejuif, France.

Dr. Beutler is a physician-scientist whose career has spanned more than half a century. Among his many accomplishments, Dr. Beutler made significant contributions to the understanding of the biochemical and genetic causes underlying disorders of the red blood cell, originated the concept of X-chromosome inactivation in human females independently of mouse geneticist Mary Lyon, and provided the first formal proof of this phenomenon – an insight that has become one of the cornerstones of mammalian genetics. He also developed screening tests for the genetic disorders galactosemia and Gaucher disease. He is currently Professor and Chairman, Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, CA.

Dr. Beutler’s award will be presented prior to the Plenary Scientific Session on Sunday, December 9, at 1:30 p.m. EST in Hall A1. Drs. Croce and Vainchenker will be presented with their awards at the Presidential Symposium to be held on Tuesday, December 11, from 9:30 a.m. to 12:00 p.m. in Hall A1. For the complete annual meeting schedule and additional information, please visit http://www.hematology.org/meetings/2007.

One technique a bloodless surgery can employ is called “cell salvage” in which blood lost during surgery is siphoned from the body, passed through a filter for cleaning and returned to the body. It can also be used by the physician during surgery to limit blood loss and to avoid the need for transfusion of blood from sources other than the patient.

Jehovah’s Witness patients believe that accepting blood from a source other then themselves defies the scriptural teachings that their religion holds steadfast. Originally developed to meet the needs of the Jehovah’s Witness community, bloodless surgery is transfusion-free and is acceptable to Jehovah Witness followers because they are being reinfused with their own blood. Bloodless surgery and medicine is a viable and life-saving option for these patients and those wary of the safety of the blood supply, and it is safe for a growing number of surgical and medical conditions, except for acute leukemia and traumatic injury.

“Bloodless procedures have proven to be safer than blood transfusion because they help eliminate complications resulting from transfusions such as immunosuppression, infection, diseases from emerging pathogens for which our blood supply is not yet tested,” said Dr. Ford. “The hospital stay is also shorter for our bloodless patients, a cost savings for the patient and the institution,” she continues. Pennsylvania Hospital in Philadelphia is one of the pioneering centers for bloodless medicine and surgery.

Dr. Ford likens the weeks-old blood often used for transfusions to “water from a dirty fish tank.” Depleted of most of its oxygen-carrying capacity, the stored blood is not maximally beneficial to any patient.

Prior to surgery, Dr. Ford prepares patients carefully – using medicines to build red blood cells, and managing their hemoglobin count. A higher hemoglobin level lowers the risk of transfusion.

Dr. Ford has performed the largest number of successful stem cell transplants without blood transfusion of anyone in the world. Among the procedures for which Dr. Ford has prepared patients for bloodless medicine and surgery are cardiothoracic surgery; radical hysterectomies, prostatectomies, cystectomies, and repair of aneurysms, chemotherapy management, and total hip and knee surgery.

The bloodless team at Pennsylvania Hospital has saved the lives of many Jehovah’s Witness patients who otherwise would not have received care. “We see patients from all over the country who come to us for our expertise in bloodless medicine. The needs of the Jehovah’s Witness community have helped us develop practices that can not only save their lives, but can also benefit the entire patient community,” explained Dr. Ford.

Co-authored by researchers from the University of Rochester Medical Center and University of Michigan Health System, the study raises another red flag about transfusions, an ancient medical practice that some doctors now believe is overused.

Blood transfusions were once reserved for only the sickest patients, but have evolved from a life-saving therapy to an elective treatment for many illnesses. Patients today receive donor blood, for example, to prevent severe anemia and improve oxygen delivery due to heart failure.

“For 100 years we’ve assumed blood transfusions are good for people, but most of these clinical practices grew before we had the research to support it,” said co-author Neil Blumberg, M.D., professor of Pathology and Laboratory Medicine and director of Transfusion Medicine at the University of Rochester Medical Center.

In the current study, Blumberg and corresponding author Mary Rogers, Ph.D., of the University of Michigan, Department of Internal Medicine, analyzed the data of 380 adult Rochester, N.Y., patients who had primary coronary artery bypass graft surgery, primary valve replacement, or both, in 1997 or 1998 at Strong Memorial Hospital. Researchers looked at in-hospital deaths, lengths of stay, number of days of infection and fever, and whether any patients developed pulmonary dysfunction, a serious side effect of heart surgery. No external funding was received for the study.

Sixty percent of the patients were men and about 40 percent were women. However, the women were 44.6 percent more likely to receive a blood transfusion than the men. Of the 150 women studied, 149 (99 percent) received donor blood during their hospitalization, compared to 77 percent of the men.

Reasons for the gender gap are unclear. Doctors typically measure a patient’s hematocrit value, or red blood cell count, before ordering a blood transfusion. Women tend to have lower concentrations of red blood cells than men throughout their lives, Blumberg said. This does not always warrant a transfusion, as the red cell concentration alone does not determine the likelihood of complications from anemia. The study showed that when men and women had equivalent, normal preoperative red blood counts, 99 percent of the women still received transfusions, compared to 62 percent of the men. This suggests a reliance on the red cell concentration as the prime factor in determining when a transfusion is given, the authors said.

Although a direct connection between blood transfusions and infections is being debated among scientists, several studies support the notion that donor blood can provoke a negative response from the patient’s immune system.

Of the 380 patients, 13 died while in the hospital; all of the 13 patients received blood transfusions, and infection was strongly related to death. Blood transfusions correlated with more days of fever, more days in intensive care, and a longer hospital stay, particularly if the patient got more than four units of blood. Women were more likely to die in the hospital (6.7 percent) than men (1.3 percent), and 11 percent of the women in the study developed pulmonary dysfunction after surgery, compared with 3.9 percent of the men.

Blood transfusions are very common. The study reports that 41 percent to 71 percent of all Americans have a blood transfusion within their lifetimes. For the year 2003 (the most recent year data was available) a transfusion was the most common procedure performed in U.S. hospitals, according to the Healthcare Cost and Utilization Project (HCUP), a government/industry database widely used by scientists.

Judgments among doctors and hospital transfusion policies can vary greatly across the country. Blumberg and colleagues advocate using donor blood from which the white cells have been removed. This process, called leukoreduction, is believed to diminish the chances of an inflammatory response or infection. (Pall Biomedical, which manufactures leukoreduction filters, has paid lecture fees and awarded other research grants to Blumberg.)

In 1998 the University of Rochester’s Strong Memorial Hospital was among the first hospitals in the country to begin using leukoreduced blood for cardiac surgery cases. In 2000, the hospital extended its leukoreduction practice to all other patients.

New research from the lab of Harvard Medical School professor of pathology Ulrich von Andrian, published in the November 30 edition of Cell, now suggests that HSCs’ biological role is far more versatile and dynamic. He and his colleagues have found that HSCs can travel from the bone marrow, through the blood system, and enter visceral organs where they perform reconnaissance missions in search of pathogenic invaders. Upon encountering an invader they immediately synthesize a defense, divide and mature, churning out new immune system cells such as dendritic cells and other leukocytes, right on the spot.

“This process changes the way we look at blood stem cells,” says von Andrian.

For almost five decades scientists have known that a fraction of HSCs will sometimes migrate from the bone marrow into the bloodstream. And while scientists have observed this phenomenon, they haven’t known exactly why the stem cells would do this, and what sort of itinerary they might follow once they entered the blood.

A group in von Andrian’s lab, led by postdoctoral researcher and cardiologist Steffen Massberg, decided to explore this question.

They began be extracting lymph samples from the thoracic duct of a mouse. The thoracic duct, a major component of the lymphatic system, routes the body’s excess fluids into the circulation, fluids that normally accumulate in organs. In that sense, it’s a kind of physiological storm drainage system. The group reasoned that any itinerary would eventually bring these cells into the lymph system, so it marked a logical starting point.

After screening large samples of thoracic fluid, they discovered an extremely small population of cells that, after rigorous testing, behaved identically to blood stem cells. Further tests, which involved mice genetically engineered so that their blood stem cells could be detected through fluorescent microscopy, revealed that these cells were also scattered throughout visceral organs, such as liver, heart, and lung.

“Taken all together, a picture developed suggesting that these cells migrated from the marrow and into the circulation where they would then leak out and enter the tissue,” says Massberg. “After that, the thoracic duct would empty them back into the circulation, where they could reenter the marrow. But the question was, why” What exactly are they doing?”

The group had found that the stem cells remain in the tissue for thirty-six hours before exiting into the thoracic duct. This suggested that they were conducting some kind of surveillance. To test this, Massberg and his colleagues injected a bacterial endotoxin into the mouse tissue. Within a matter of days, clusters of specialized immune cells formed in the infected areas.

“Typical immune responses deplete local specialized immune cells,” says Massberg. “It appears that the hematopoietic stem cells initiate an immune response and replenish these specialized immune cells. It’s a way of sensing local environmental disturbances and responding locally.”

But finally, the researchers identified the molecular mechanism that explained these observational data.

After residing for a while in the organ tissue, the stem cells receive a lipid signal that enables them to exit into the thoracic duct. However, the presence of endotoxin disrupts the normal signaling cascade. When the receptors on the stem-cell surface that detect the pathogens become active, the cell’s ability to receive the lipid signal is blocked. The stem cells literally get stuck in the tissue, where they are then triggered to proliferate into immune cells.

“That stem cells are actually a part of the immune system, rather than just giving rise to it, is a very provocative idea,” says von Andrian. “This opens up a number of new avenues for us to explore ways that our bodies fight pathogens.”

The researchers are now looking at ways that other common diseases, like cancer, may exploit this process.

Published in today’s New England Journal of Medicine and representing the largest ever clinical trial of its kind for ITP patients, the Phase 2 study found that eltrombopag doses of 50 and 75 mg daily elevated platelet counts to a safe level (? 50,000 per cubic millimeter) in more than 75 percent of chronic ITP patients within two weeks — compared to 11 percent of patients receiving placebo. As long as the therapy was administered, platelet counts continued to rise or remain increased, and bleeding symptoms decreased. Incidence and severity of adverse effects was similar in the placebo and eltrombopag treated groups.

“These findings represent an important step in the development of a new treatment option for those living with chronic ITP. The fact that eltrombopag elevated platelet counts in this study within one week could be very useful to chronic ITP patients in need of short-term treatment,” says the study’s principal investigator, Dr. James Bussel, director of the platelet research and treatment program at the Phyllis and David Komansky Center for Children’s Health at NewYork-Presbyterian Hospital/Weill Cornell Medical Center and professor of pediatrics in obstetrics and gynecology and in medicine at Weill Cornell Medical College.

The study represents the second major clinical study of a platelet growth factor for ITP. In October 2006, Dr. Bussel was the lead author of publication reporting the results of a Phase I, II study of AMG 531, also in the New England Journal of Medicine.

Current and future studies will evaluate the safety and efficacy of eltrombopag as a long-term treatment for ITP, and its efficacy and safety in populations like the four million Americans with hepatitis C or patients receiving myelosuppressive chemotherapy.

Co-authors included Weill Cornell’s Dr. Bethan Psaila, and physician-scientists from Prince of Wales Hospital in Hong Kong; George Cancer Specialists in Tucker, Ga.; Hematology Research Center in Moscow, Russian Federation; Hôpital La Rabta in Tuhis, Tunisia; Akademia Medyczna in Bialystok, Poland; GlaxoSmithKline in Collegeville, Pa. and Greenford, U.K.; and Barts & The London School of Medicine, London.

The study was financially supported by GlaxoSmithKline. Dr. Bussel is an Advisory Board Member for GlaxoSmithKline and has received research grant support, lecture fees, and consulting fees from GlaxoSmithKline and reports equity ownership in GlaxoSmithKline.

Immune thrombocytopenic purpura (ITP) is an autoimmune disease in which anti-platelet antibodies accelerate destruction of platelets. ITP patients commonly have platelet counts of less than 30,000 per cubic millimeter, compared to normal platelet counts of between 150,000 and 440,000. ITP affects women of child bearing age at 2-to-3 times the rate of men.

NewYork-Presbyterian Hospital/Weill Cornell Medical Center

NewYork-Presbyterian Hospital/Weill Cornell Medical Center, located in New York City, is one of the leading academic medical centers in the world, comprising the teaching hospital NewYork-Presbyterian and Weill Cornell Medical College, the medical school of Cornell University. NewYork-Presbyterian/Weill Cornell provides state-of-the-art inpatient, ambulatory and preventive care in all areas of medicine, and is committed to excellence in patient care, education, research and community service. Weill Cornell physician-scientists have been responsible for many medical advances — from the development of the Pap test for cervical cancer to the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial for gene therapy for Parkinson’s disease, the first indication of bone marrow’s critical role in tumor growth, and, most recently, the world’s first successful use of deep brain stimulation to treat a minimally-conscious brain-injured patient. NewYork-Presbyterian, which is ranked sixth on the U.S.News & World Report’s list of top hospitals, also comprises NewYork-Presbyterian Hospital/Columbia University Medical Center, Morgan Stanley Children’s Hospital of NewYork-Presbyterian, NewYork-Presbyterian Hospital/Westchester Division and NewYork-Presbyterian Hospital/The Allen Pavilion. Weill Cornell Medical College is the first U.S. medical college to offer a medical degree oversees and maintains a strong global presence in Austria, Brazil, Haiti, Tanzania, Turkey and Qatar. For more information, visit http://www.nyp.org and http://www.med.cornell.edu.

This hereditary fault (Hereditary antithrombin Deficiency, HD) means that the patients e.g. when in surgery can be in danger of dying from thrombosis if not treated for the lacking antithrombin III.

With ATryn®, the HD patients now get access to a new, safe and technological remarkable treatment without the use of donor blood. ATryn® is the world’s first approved drug which is produced in transgenic goats. Human antithrombin is expressed in the milk of these goats and then processed into a highly purified product.

In 2005, LEO Pharma and the American GTC Biotherapeutics Inc. (NASDAQ: GTCB, http://www.gtc-bio.com) concluded a development and marketing agreement for Europe, Canada and the Middle East concerning ATryn®.

LEO is responsible for the marketing and sales in the mentioned areas for the HD indication as well as further developing ATryn® for new indications. GTC is responsible for supplying the product.

In August, LEO Pharma began a clinical phase II study in which patients with DIC (Disseminated Intravascular Coagulation) are treated with ATryn®. DIC is serious condition during which the blood coagulates in the blood vessels and occurs in connection with serious sepsis. In the EU and Canada, the number of patients stricken by sepsis and DIC each year is estimated to be about 125,000 – 30-60% of these ends fatally.

“ATryn® is a new, exciting drug with interesting perspectives if it can be used for the treatment of other indications. This could e.g. be for the treatment of DIC at patients with severe sepsis. ATryn® fits well with our existing anticoagulation business which among other things includes Innohep®, Heparin LEO® and Protaminsulfat LEO Pharma®,” says Ernst Lunding, CEO of LEO Pharma.

The ATryn® phase II study concerning DIC will prove the drug’s safety, effect and optimal dose against future phase III study.

About LEO Pharma

LEO Pharma is an independent research based company with headquarter located in Ballerup, Denmark. LEO Pharma is 100% owned by the LEO Foundation and is one of the world’s leading companies in dermatology and parenteral treatment of tromboemboli. LEO invents, develops and manufactures safe and efficacious pharmaceutical drugs and markets them globally – 96% of the turnover is generated outside Denmark. LEO Pharma is represented in more than 90 countries and has around 3,000 employees around the world; 1,200 of these in Denmark.