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Mayo Clinic scientists say JAK inhibitors may help treat patients with myelofibrosis

Two janus kinase (JAK) inhibitors are substantially improving treatment of myelofibrosis in patients, say Mayo Clinic researchers who are presenting results of several clinical trials at the 52nd annual meeting of the American Society of Hematology (ASH) Dec. 4 in Orlando.

7 Dec 2010

New drug appears to help patients with chronic myeloid leukemia

A new drug appears to help chronic myeloid leukemia patients who are out of treatment options after first- and second-line drugs have failed them or because their cancer cells have a mutation that makes them resistant from the start, researchers reported at the 52nd Annual Meeting of the American Society of Hematology.

7 Dec 2010

HDAC inhibitors may be used to fight against skin cancers

In a study published in the journal Developmental Cell, Sarah Millar PhD, professor of Dermatology and Cell & Developmental Biology at the University of Pennsylvania School of Medicine, and colleagues demonstrate that a pair of enzymes called HDACs are critical to the proper formation of mammalian skin.

7 Dec 2010

No evidence of toxicity in hemophilia B patients enrolled in gene therapy trial

Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Children's Research Hospital and UCL (University College London) to correct the inherited bleeding disorder.

7 Dec 2010

Screening for CCDC40 gene mutations may help determine risk for congenital heart defects

A gene that can cause congenital heart defects has been identified by a team of scientists, including a group from Princeton University. The discovery could lead to new treatments for those affected by the conditions brought on by the birth defect.

7 Dec 2010

ArQule initiates enrollment in ARQ 736 Phase 1 clinical trial for advanced solid tumors

ArQule, Inc. today announced the commencement of patient dosing in a Phase 1 clinical trial with ARQ 736, a small molecule inhibitor of the RAF kinases.

7 Dec 2010

$Updated Phase 1 study data of ponatinib in patients with refractory CML, Ph+ ALL presented at ASH$

Updated Phase 1 study data of ponatinib in patients with refractory CML, Ph+ ALL presented at ASH

ARIAD Pharmaceuticals, Inc. today announced updated clinical data from a fully enrolled and ongoing Phase 1 study of its investigational pan-BCR-ABL inhibitor, ponatinib, in patients with resistant and refractory chronic myeloid leukemia and Philadelphia-positive acute lymphoblastic leukemia.

6 Dec 2010

Research on BCR-ABL inhibitors for treating ALL, CML presented at ASH Annual Meeting

Over the past decade, significant advances have been made in the treatment of leukemia through the ongoing development of gene-based targeted therapies. Research that will be presented today at the 52nd Annual Meeting of the American Society of Hematology provides greater understanding of the optimal use of several BCR-ABL inhibitors for the treatment of acute lymphoblastic leukemia and chronic myeloid leukemia, and how a new gene target functions for several myeloid malignancies.

6 Dec 2010

Research highlighting treatment for pediatric SCD, malaria presented at ASH 2010

Red blood cells have the very important role of carrying oxygen from the lungs to the rest of the body; therefore, disorders that affect RBCs can have a significant impact on quality of life. Sickle cell disease and malaria are two common RBC diseases that affect a significant portion of the U.S. and global populations. Sickle cell disease, a genetic blood disorder that causes deformed and dysfunctional red blood cells, affects an estimated 70,000 to 100,000 Americans, while malaria affects approximately 1,500 Americans annually and 190 to 311 million people worldwide.

6 Dec 2010

Research reveals new standard treatment options for various forms of lymphoma

The next generation of drug therapies and enhanced treatment approaches for various forms of lymphoma are evolving as researchers continue to better understand how these cancers progress. Research will be presented today at the 52nd Annual Meeting of the American Society of Hematology introducing promising new options for the standard treatment of advanced asymptomatic follicular lymphoma, mantle cell lymphoma, and early, unfavorable (referring to patients with clinical stage I or II disease and one or more risk factors) Hodgkin disease.

6 Dec 2010

New studies answer questions related to stem cell transplant procedures in treating hematologic malignancies

Results from four innovative studies help answer outstanding questions about stem cell transplant procedures in treating various hematologic malignancies. Research that will be presented today at the 52nd Annual Meeting of the American Society of Hematology reveals that there is no increase in overall cancer risk in people who donate stem cells and that the utilization of a double cord blood stem cell transplant is associated with better overall outcomes when used early in the treatment of acute leukemias.

6 Dec 2010

MDS Foundation: Significant advances made in understanding, treating MDS, AML

The Myelodysplastic Syndromes Foundation today said significant advances are being made in understanding and treating MDS and a related condition, AML (acute myeloid leukemia). Key studies being presented this week include data on new markers and genetic mutations to predict susceptibility to MDS and response to treatment.

4 Dec 2010

Gene mutation can lead to different mental illnesses when influenced by same environmental factor

Male mice born with a genetic mutation that's believed to make humans more susceptible to schizophrenia develop behaviors that mimic other major psychiatric illnesses when their mothers are exposed to an assault to the immune system while pregnant, according to new Johns Hopkins research.

4 Dec 2010

Alteration in gene causes male embryos to develop as females

Scientists have discovered that the alteration of a single gene could cause some male embryos to develop as females.

4 Dec 2010

Research findings could provide breakthrough for development of effective therapies for NSCLC

Research at Virginia Commonwealth University Massey Cancer Center led by Charles E. Chalfant, Ph.D., associate professor of biochemistry and molecular Biology, discovered a previously unknown mechanism in non-small cell lung cancer cells that contributes to their ability to maintain and grow tumors. Narrowing in on this mechanism could provide a breakthrough for the development of effective therapies for NSCLC and other cancers.

4 Dec 2010

New study offers hope for children with MPS I genetic disorder

A new study offers hope for children born with a rare genetic disease, according to a paper published by the American Association for the Advancement of Science.

3 Dec 2010

New clue in leukemia mystery

There is new hope for people with acute myelogenous leukemia (AML), a fast-growing cancer of the blood and bone marrow. Research led by Weill Cornell Medical College and published today in the online edition of the journal Cancer Cell reveals a surprising and unexpected cancer-causing mechanism. The investigators discovered that newly identified mutant enzymes in AML create a chemical poison to cause leukemia. Their findings should prove useful in treating patients by providing a molecular target against which to develop new drugs against one subset of AML as well as other cancers.

2 Dec 2010

Researchers identify blip in gene that causes JMP syndrome

UT Southwestern Medical Center researchers have identified a gene responsible for a rare disease that results in severe joint stiffness, muscle loss, anemia and panniculitis-induced lipodystrophy, or JMP syndrome.

2 Dec 2010

Study finds duplicated region of DNA in major depressive disorder

A large genetic study of people with major depression has found that a duplicated region of DNA on chromosome 5 predisposes people to the disorder. The gene involved plays an important role in the development of nerve cells, adding to evidence that disruptions in neurotransmission networks form a biological basis for depression.

1 Dec 2010

Pfizer to present data on hematology portfolio at ASH Annual Meeting

Pfizer said today that new data on investigational compounds in its hematology portfolio will be presented at the 52nd Annual Meeting of the American Society of Hematology (ASH) in Orlando, December 4-7.

1 Dec 2010

Mutation News and Research

Mayo Clinic scientists say JAK inhibitors may help treat patients with myelofibrosis

New drug appears to help patients with chronic myeloid leukemia

HDAC inhibitors may be used to fight against skin cancers

No evidence of toxicity in hemophilia B patients enrolled in gene therapy trial

Screening for CCDC40 gene mutations may help determine risk for congenital heart defects

ArQule initiates enrollment in ARQ 736 Phase 1 clinical trial for advanced solid tumors

Updated Phase 1 study data of ponatinib in patients with refractory CML, Ph+ ALL presented at ASH

Research on BCR-ABL inhibitors for treating ALL, CML presented at ASH Annual Meeting

Research highlighting treatment for pediatric SCD, malaria presented at ASH 2010

Research reveals new standard treatment options for various forms of lymphoma

New studies answer questions related to stem cell transplant procedures in treating hematologic malignancies

MDS Foundation: Significant advances made in understanding, treating MDS, AML

Gene mutation can lead to different mental illnesses when influenced by same environmental factor

Alteration in gene causes male embryos to develop as females

Research findings could provide breakthrough for development of effective therapies for NSCLC

New study offers hope for children with MPS I genetic disorder

New clue in leukemia mystery

Researchers identify blip in gene that causes JMP syndrome

Study finds duplicated region of DNA in major depressive disorder

Pfizer to present data on hematology portfolio at ASH Annual Meeting

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