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Researchers discover new gene mutations associated with megalencephaly

A research team led by Seattle Children's Research Institute has discovered new gene mutations associated with markedly enlarged brain size, or megalencephaly.

30 Jun 2012

Bayer receives priority review designation from FDA for regorafenib NDA

Bayer HealthCare and Onyx Pharmaceuticals announced today that the U.S. Food and Drug Administration (FDA) has granted priority review designation to Bayer HealthCare's New Drug Application (NDA) filed end of April 2012 for the oral multi-kinase inhibitor regorafenib, for the treatment of patients with metastatic colorectal cancer (mCRC) whose disease has progressed after approved standard therapies.

29 Jun 2012

Data from Infinity’s IPI-145 pre-clinical study on inflammatory diseases

Infinity Pharmaceuticals, Inc. today reported preclinical data for IPI-145, the company's oral inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma. The data demonstrate potent PI3K-delta and -gamma inhibition in biochemical, cellular and whole blood assays and activity in multiple preclinical models of inflammatory disease, including rheumatoid arthritis and asthma.

28 Jun 2012

Protein kinase A activated in the nucleus as well as the cytoplasm

Using a new tool allowing proteins in a living cell to be manipulated in real time, researchers at Johns Hopkins have stumbled across the answer to a longstanding debate about where and how a certain protein is turned on in the cell. Reporting in the February 2012 issue of Nature Chemical Biology, scientists show that protein kinase A is also activated in the nucleus rather than inside the cell’s body, a challenge to traditional beliefs.

27 Jun 2012

Study investigates how glucose metabolism affects biochemical signals in cancer cells

Compared to normal cells, cancer cells have a prodigious appetite for glucose, the result of a shift in cell metabolism known as aerobic glycolysis or the "Warburg effect." Researchers focusing on this effect as a possible target for cancer therapies have examined how biochemical signals present in cancer cells regulate the altered metabolic state.

27 Jun 2012

Greater awareness of MH and consequences of ryanodine receptor gene mutations

From the MHAUS President's Blog by MHAUS President, Henry Rosenberg, MD. A few months ago, I received a call from the MH Hotline who connected me to an anesthesiologist and OR team who were desperately trying to manage a Malignant Hyperthermia crisis.

26 Jun 2012

WCMC researchers devise innovative boxer-like strategy to fight multiple myeloma

Weill Cornell Medical College researchers have devised an innovative boxer-like strategy, based on the serial use of two anti-cancer drugs, to deliver a one-two punch to first weaken the defenses of multiple myeloma and then deliver the final knock-out punch to win the fight.

23 Jun 2012

A potential drug target for NK/T-cell lymphoma

A discovery by Van Andel Institute researchers has identified a potential drug target for NK/T-cell lymphoma, a type of cancer responsible for claiming the life of a close friend and fellow researcher.

22 Jun 2012

Grb2 switch controls normal signaling through fibroblast growth factor receptor

Once considered merely a passive link between proteins that matter, Grb2 - pronounced "grab2" - actually lives up to its nickname with its controlling grip on an important cell signaling pathway, scientists at The University of Texas MD Anderson Cancer Center report in the June 22 issue of Cell.

22 Jun 2012

New signal transduction pathway might be fruitful target for cancer drugs

Researchers at the University of California, San Diego School of Medicine have uncovered a new signal transduction pathway specifically devoted to the regulation of alternative RNA splicing, a process that allows a single gene to produce or code multiple types of protein variants. The discovery, published in the June 27, 2012 issue of Molecular Cell, suggests the new pathway might be a fruitful target for new cancer drugs.

22 Jun 2012

Researchers discover protein required to regrow injured peripheral nerves

A protein required to regrow injured peripheral nerves has been identified by researchers at Washington University School of Medicine in St. Louis.

21 Jun 2012

Infinity voluntarily stops saridegib Phase 2 trial for chondrosarcoma

Infinity Pharmaceuticals, Inc. today announced interim data from its Phase 2, double-blind, randomized, placebo-controlled study of saridegib (IPI-926) in patients with metastatic or locally advanced, inoperable chondrosarcoma. The primary endpoint of the Phase 2 study was progression-free survival.

18 Jun 2012

Ascepion, Debiopharm partner to develop and commercialize ASP-08126 for solid tumors

Debiopharm Group, a global biopharmaceutical development specialist that focuses on serious medical conditions and particularly oncology, and Ascepion Pharmaceuticals, Inc., a privately-held, biopharmaceutical company dedicated to the discovery and development of breakthrough small molecule drugs for the treatment of oncology and related diseases, have entered into an exclusive worldwide license agreement concerning the development and commercialization of ASP-08126 (called Debio 1144 by Debiopharm) a multikinase inhibitor currently in pre-clinical development.

18 Jun 2012

Updated data from ARIAD’s ponatinib pivotal trial on CML or Ph+ ALL

ARIAD Pharmaceuticals, Inc. today announced updated clinical data from the pivotal PACE trial of its investigational pan-BCR-ABL inhibitor, ponatinib, in patients with chronic myeloid leukaemia (CML) or Philadelphia-positive acute lymphoblastic leukaemia (Ph+ ALL), who are resistant or intolerant to dasatinib or nilotinib or who have the T315I mutation.

18 Jun 2012

Updated results from two clinical trials of Bruton's ibrutinib for CLL/SLL

Pharmacyclics, Inc announced today at the 17th Congress of European Hematology Association updated results from two clinical trials of Bruton's tyrosine kinase (BTK) inhibitor, ibrutinib (PCI-32765), for the treatment of patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL).

18 Jun 2012

Otsuka, Bristol-Myers Squibb announce six-year data from SPRYCEL Phase 3 trial on CP-CML

Bristol-Myers Squibb Company and Otsuka Pharmaceutical Europe Ltd., today announced six-year follow-up results from a Phase 3 randomised, open-label, dose-optimisation study of SPRYCEL (dasatinib) in Philadelphia chromosome-positive (Ph+) chronic-phase chronic myeloid leukaemia (CP-CML) adult patients resistant or intolerant to Glivec (imatinib).

16 Jun 2012

Patients with NK/T-cell lymphomas might benefit from treatment with JAK inhibitors

A substantial proportion of NK/T-cell lymphomas harbor Janus Kinase 3 gene mutations. Patients with these lymphomas might benefit from treatment with a Janus Kinase inhibitor according to a study published in Cancer Discovery, a journal of the American Association for Cancer Research.

16 Jun 2012

AMPK enzyme promotes survival of cancer cells: Research

A molecule thought to limit cell proliferation also helps cancer cells survive during initial tumor formation and when the wayward cells spread to other organs in the body, researchers at the University of Illinois at Chicago College of Medicine have found.

15 Jun 2012

AB Science announces results from masitinib phase 2 study on MS

AB Science SA, a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors, announces the publication of results from the human phase 2 study of masitinib carried-out in the treatment of progressive multiple sclerosis.

14 Jun 2012

New method enhances adhesion of injected CECs for successful corneal transplantation

Regenerative medicine, or the use of specially grown tissues and cells to treat injuries and diseases, has been successful in treating disorders of a number of organs, including heart, pancreas, and cartilage. However, efforts to treat disorders of the corneal endothelium, a single cell layer on the inner surface of the cornea, with regenerative techniques have been less effective. Now, a group of scientists has developed a method that enhances the adhesion of injected corneal endothelial cells (CECs), allowing for successful corneal transplantation to repair pathological dysfunctions.

14 Jun 2012