Study shows efficacy of YONDELIS (trabectedin) in patients with soft-tissue sarcoma

In continuation of my update on trabectedin

PharmaMar announced that the European Journal of Cancer published online data from a large retrospective study with soft-tissue sarcoma (STS) patients carried out at 25 French centers confirming that in routine practice YONDELIS® (trabectedin) shows comparable or better clinical outcomes than those observed in clinical trials . The results add to previous evidence from other studies with trabectedin , including the compassionate expanded access program of 1895 patients with incurable disease , demonstrating that response and disease control rates are higher than expected. The authors describe that long-term treatment of multiple types of STS patients without progressive disease delayed progression and improved survival compared to those who stopped it after six cycles, as recently suggested in the T-DIS studyiii. In this routine real-life scenario, the median progression-free survival (PFS) and overall survival (OS) were 4.4 and 12.2 months, respectively after a median follow-up of 22 month. Of the 304 patients who achieved non-progressive disease after six cycles, 227 continued receiving trabectedin and obtained significant superior PFS (11.7 versus 7.6 months) and OS (24.9 versus 16.9 months) than those who did not. The recent study reinforces an approach of early administration of YONDELIS® (trabectedin) to optimize its efficacy as second-line treatment in patients that will probably benefit from this drug. Despite the limitations of the study, the investigators emphasize that this clinical strategy may achieve longer disease control without compromising the safety profile of the treatment, given that a third of the patients received more 6 or more cycles of the drug and were able to tolerate longer treatments.

Study shows efficacy of YONDELIS (trabectedin) in patients with soft-tissue sarcoma

Bionomics to present data from DisrupTOR-1 trial at ASCO Genitourinary Cancers Symposium

Bionomics Limited (ASX:BNO, ADR:BMICY) is to present important additional data from the DisrupTOR-1 trial of BNC105 in patients with metastatic renal cancer at the ASCO Genitourinary Cancers Symposium in Orlando, Florida. The data will be presented by Dr. Sumanta Pal of the City of Hope Comprehensive Cancer Center in California in his poster presentation.

The new data identifies Ferritin and IL-8 as two baseline biomarkers that correlate with an improved progression free survival (PFS) in patients. Elevated baseline Ferritin and lower baseline IL-8 were associated with improved PFS (P=0.0291 and P=0.0149, respectively). Eighty nine percent of patients expressing elevated plasma levels of Ferritin and lower plasma levels of IL8 at baseline were disease progression free (PFS) at 6 months.

"The DisrupTOR-1 trial offered a unique opportunity to assess biomarkers related to the activity of BNC105. Interestingly our findings suggest that baseline levels of Ferritin and IL-8 may predict an excellent clinical outcome with the combination of Afinitor with BNC105. Plans are underway already to validate this finding in a biomarker-driven trial," said Dr. Pal, Co-Director of the Kidney Cancer Program at the Department of Medical Oncology & Experimental Therapeutics of the City of Hope Comprehensive Cancer Center.

"This is a compelling improvement compared to the 34% PFS observed at six months in the unselected population," said Dr. Deborah Rathjen, Bionomics' CEO and Managing Director.

"These results indicate that moving forward, biomarker-based patient selection has the potential to provide guidance and optimise clinical outcomes in the treatment of renal cancer, presenting a range of new possibilities for BNC105. We will continue to explore further data and the options for the advancement of the compound.

"Bionomics to present data from DisrupTOR-1 trial at ASCO Genitourinary Cancers Symposium

Sorafenib, sunitinib provide no benefit to patients with locally advanced kidney cancer

Findings from a federally funded study suggest that patients with locally advanced kidney cancer should not be treated with either adjuvant (post-surgery) sorafenib or sunitinib. The average period to disease recurrence was similar between those who received sorafenib or sunitinib after surgery (5.6 years) and those treated with placebo (5.7 years). The study will be presented at the upcoming 2015 Genitourinary Cancers Symposium in Orlando.

"These drugs didn't reduce disease recurrence, but on average they did not appear to worsen patient outcomes either," said lead study author Naomi B. Haas, MD, an Associate Professor of Medicine at the Abramson Cancer Center of the University of Pennsylvania in Philadelphia, Pa. "We are still analyzing the various groups of patients enrolled on this trial, and we hope that analysis of patient specimens collected on this study may provide clues into subsets of patients who might still benefit from these therapies."

Sorafenib, sunitinib provide no benefit to patients with locally advanced kidney cancer

Treatment reduces risk of recurrence of C. difficile infection

In continuation of my update on metronidazole  &  vancomycin

Among patients with Clostridium difficile infection (CDI) who recovered following standard treatment with the antibiotics metronidazole or vancomycin, oral administration of spores of a strain of C difficilethat does not produce toxins colonized the gastrointestinal tract and significantly reduced CDI recurrence, according to a study in the May 5 issue of JAMA.

C difficile is the cause of one of the most common and deadly health care-associated infections, linked to 29,000 U.S. deaths each year. Rates of CDI remain at unprecedented high levels in U.S. hospitals. Clinical infection also has a recurrence rate of 25 percent to 30 percent among affected patients. Not all strains of C difficile produce toxins. Nontoxigenic C difficile strains that lack the genes for toxin production are also found in the hospital environment and can colonize hospitalized patients, although patients are usually asymptomatic. Gastrointestinal colonization by these nontoxigenic C difficilestrains (in both humans and hamsters) has shown promising results as a potential way to prevent CDI, according to background information in the article.

Dale N. Gerding, M.D., of the Edward Hines Jr. VA Hospital, Hines, Il., and Loyola University Chicago, Maywood, Il., and colleagues randomly assigned 173 adult patients who were diagnosed as having CDI (first episode or first recurrence) to receive 1 of 4 treatments: oral liquid formulation of nontoxigenic C difficile strain M3 (VP20621; NTCD-M3), 104 spores/d for 7 days (n = 43), 107 spores/d for 7 days (n = 44), 107 spores/d for 14 days (n = 42), or placebo for 14 days (n = 44). Prior to enrollment, these patients had all successfully completed treatment with metronidazole, oral vancomycin, or both at 44 study centers in the United States, Canada, and Europe.

Treatment reduces risk of recurrence of C. difficile infection 

Taiho Oncology announces acceptance of TAS-102 NDA for review by FDA

Taiho Oncology, Inc., a subsidiary of Taiho Pharmaceutical Co., Ltd. (Japan), today announced the New Drug Application (NDA) for TAS-102 (nonproprietary names: trifluridine and tipiracil hydrochloride), has been accepted for review by the U.S. Food and Drug Administration (FDA). TAS-102 is an oral combination investigational anticancer drug for the treatment of refractory metastatic colorectal cancer (mCRC).

TAS-102 is an investigational drug candidate for metastatic colorectal cancer. It is a combination of two active pharmaceutical ingredients: trifluridine, (see left structure)  

a nucleoside analog (see right structure), and tipiracil hydrochloride, a thymidine phosphorylase inhibitor. Tipiracil hydrochloride prevents rapid metabolism of trifluiridine, increasing the bioavailability of trifluiridine.

"The FDA's filing of the TAS-102 NDA represents a significant milestone for our company and underscores the need for new treatment options for patients with refractory metastatic colorectal cancer," said Eric Benn, Taiho Oncology's president and chief executive officer. "Today, we are one step closer to our ultimate goal of gaining regulatory approval for TAS-102 and making it available to patients in the USA with this serious medical condition. We look forward to working closely with the FDA during the NDA review."

Taiho Oncology announces acceptance of TAS-102 NDA for review by FDA

Anticoagulant fondaparinux lowers risk of major bleeding events, death in heart attack patients

 

Patients who experienced a certain type of heart attack who received the anticoagulant fondaparinux had a lower risk of major bleeding events and death both in the hospital and after six months compared to patients who received low-molecular-weight heparin (LMWH), although both groups had similar rates of subsequent heart attack or stroke, according to a study in the February 17 issue of JAMA.

Reducing bleeding events in patients receiving antithrombotic therapy is important since bleeding events are associated with increased mortality. Fondaparinux was associated with reduced major bleeding events and improved survival compared with LMWH (a class of anticoagulant medications) in a large randomized clinical trial involving patients with non-ST-segment elevation myocardial infarction (NSTEMI; a certain pattern on an electrocardiogram following a heart attack). Large-scale experience of the use of fondaparinux vs LMWH outside of a clinical trial setting has been lacking, according to background information in the article.

Karolina Szummer, M.D., Ph.D., of the Karolinska Institutet, Stockholm, Sweden, and colleagues analyzed data from a Swedish registry that included 40,616 patients with NSTEMI who received in-hospital treatment with fondaparinux or LMWH between September 2006 through June 2010, with follow-up through December 2010.

Anticoagulant fondaparinux lowers risk of major bleeding events, death in heart attack patients

New compound appears to play role in development of opioid tolerance

While opioids are a widely used treatment for pain, patients who take them on a regular basis can become tolerant, requiring a higher dose for continued pain relief. In a study published in Anesthesiology, the official medical journal of the American Society of Anesthesiologists® (ASA®), researchers identified a compound that appears to play a role in the development of opioid tolerance. It may be possible to lessen the development of opioid tolerance if that compound is neutralized or blocked in patients taking opioids chronically for severe pain.

"Opioid tolerance is a growing problem among chronic pain patients and cancer patients in particular," said Chih-Peng Lin, M.D., assistant professor, Department of Anesthesiology, National Taiwan University College of Medicine. "We found that CXCL1, a protein produced by spinal cord tissue, contributes to opioid tolerance. By neutralizing CXCL1 in patients, we might help solve the problem of opioid tolerance."

New compound appears to play role in development of opioid tolerance

Alcon Receives FDA Approval of Pazeo (olopatadine HCl) Ophthalmic Solution for Allergic Conjunctivitis

Olopatadine hydrochloride is an antihistamine (as well as anticholinergic and mast cell stabilizer), sold as a prescription eye drop (0.2% solution, Pataday (or Patanol S in some countries), manufactured by Alcon). It is used to treat itching associated with allergic conjunctivitis (eye  allergies). Olopatadine hydrochloride 0.1% is sold as Patanol (or Opatanol in some countries). A decongestant nasal spray formulation is sold as Patanase, which was approved by the FDA on April 15, 2008.  It is also available as an oral tablet in Japan under the tradename Allelock, manufactured by Kyowa Hakko Kogyo.

Alcon, the global leader in eye care and a division of Novartis, has received approval from the U.S. Food and Drug Administration (FDA) of Pazeo (olopatadine hydrochloride ophthalmic solution) 0.7%, for the treatment of ocular itching associated with allergic conjunctivitis. Pazeo solution is dosed one drop daily, and was approved with efficacy data at 24 hours, post dose.

Alcon Receives FDA Approval of Pazeo (olopatadine HCl) Ophthalmic Solution for Allergic Conjunctivitis

Eliglustat drug improves liver, spleen size and hemoglobin level in adults with Gaucher disease type 1

In continuation of my update on eliglustat  

Among previously untreated adults with Gaucher disease type 1, a genetic disease in which there is improper metabolism due to a defect in an enzyme, treatment with the drug eliglustat resulted in significant improvements in liver and spleen size hemoglobin level, and platelet count, according to a study in the February 17 issue of JAMA.

Gaucher disease type 1 is characterized by enlargement of the spleen and liver, anemia, low blood platelets, chronic bone pain, and the failure to grow properly. Untreated Gaucher disease type 1 is a chronic and progressive disorder associated with disability, reduced life expectancy, and, in some patients, life-threatening complications. The current standard of care is enzyme replacement therapy, which requires lifelong intravenous infusions every other week. A safe, effective oral therapy is needed, according to background information in the article. 

Pramod K. Mistry, M.D., Ph.D., F.R.C.P., of the Yale University School of Medicine, New Haven, Conn., and colleagues randomly assigned 40 untreated adults with Gaucher disease type 1 to receive eliglustat (twice daily; n = 20) or placebo (n = 20) for 9 months. Eliglustat is a novel oral medication, which showed favorable results for patients with this disease in a phase 2 trial. This phase 3 trial was conducted at 18 sites in 12 countries.

The researchers found that administration of eliglustat resulted in a reduction in spleen volume of approximately 30 percent compared with placebo, as well as improvements in hemoglobin level, decreased liver volume (-6.6 percent), and increased platelet count (41 percent). No serious adverse events occurred. No patient discontinued treatment over the course of the 9-month study because of a treatment-emergent adverse event.

Eliglustat drug improves liver, spleen size and hemoglobin level in adults with Gaucher disease type 1

New molecule shows promise in controlling HIV without using daily antiretroviral drugs

Scientists have created a new molecule that shows promise for controlling HIV without daily antiretroviral drugs. The molecule foils a wider range of HIV strains in the laboratory than any known broadly neutralizing HIV antibody and is more powerful than some of the most potent of these antibodies. In addition, the molecule safely protected monkeys from infection with an HIV-like virus during a 40-week study period. Together, the data suggest that the molecule could, with further research, be used to subdue HIV in humans. The authors note that the molecule potentially could be used as both a preventative drug and as a treatment. The new findings appear in the February 18 issue of the journal Nature.

"This innovative research holds promise for moving us toward two important goals: achieving long-term protection from HIV infection, and putting HIV into sustained remission in chronically infected people," said Anthony S. Fauci, M.D., director of the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

Ref : http://www.nature.com/nature/journal/vaop/ncurrent/full/nature14264.html

New molecule shows promise in controlling HIV without using daily antiretroviral drugs

Eli Lilly accepts committee recommendation to extend evacetrapib Phase 3 trial

We know that, Evacetrapib  is a  drug under  development by  Eli Lilly  and  company (investigational   name   LY2484595)      that inhibits cholesterylester transfer protein (CETP), which  transfers and thereby increases high-density lipoprotein and     lowers   low-density lipoprotein. It is thought that modifying lipoprotein levels modifies the    risk     of     cardiovascular   disease.   The  first  CETP inhibitor, torcetrapib, was unsuccessful because it increased levels of the hormone aldosterone and increased blood pressure, which led to excess cardiac events when it was studied.  Evacetrapib does not have the same effect. When studied in a small clinical trial in people with elevated LDL and low HDL, significant improvements were noted in their lipid profile.

A recent report states that,  Eli Lilly and Company (NYSE: LLY) has accepted the recommendation of the ACCELERATE study academic executive committee, based on emerging science in the cardiovascular field, to extend the Phase 3 trial of the investigational medicine evacetrapib by approximately six months. The decision is not based on any data from ACCELERATE, as both the academic committee and the company remain blinded to efficacy results............

Eli Lilly accepts committee recommendation to extend evacetrapib Phase 3 trial

Palbociclib extends progression-free survival in advanced breast cancer patients

In continuation of my update on Palbociclib

Palbociclib, an investigational oral medication that works by blocking molecules responsible for cancer cell growth, is well tolerated and extends progression-free survival (PFS) in newly diagnosed, advanced breast cancer patients, including those whose disease has stopped responding to traditional endocrine treatments. Results of the phase II study, led by researchers in the Abramson Cancer Center and the Perelman School of Medicine at the University of Pennsylvania , were published this month in Clinical Cancer Research. Earlier phase I results by researchers at Penn Medicine contributed to the development of palbociclib, which was recently approved by the U.S. Food and Drug Administration (FDA) for metastatic breast cancer patients just beginning to undergo endocrine therapy.

"The FDA approval has expanded treatments options for many metastatic breast cancer patients, but these new results are showing how effective the drug can also be for breast cancer patients who have already tried endocrine therapies and may be running out of options," said lead investigator Angela DeMichele, MD, MSCE , associate professor in the division of Hematology/Oncology and Epidemiology and co-leader of the Breast Cancer Research Program at the Abramson Cancer Center. "Combined with the promising results from other trials looking at the effectiveness of this drug, our results indicate that palbociclib can extend the duration of disease control and produce tumor shrinkage in patients with estrogen-receptor positive (ER+) breast cancer, without the debilitating side effects of chemotherapy."

The newly-published phase II trial primarily sought to evaluate disease response and control, while monitoring for the presence of side effects such as neutropenia, an abnormally low white blood cell count. Patients enrolled in the trial had previously undergone several prior chemotherapy and hormonal regimens for metastatic disease. Palboclib was administered once daily for 21 days each month.

Overall, researchers noted a median PFS, the time before a tumor worsens or the patient dies, of 3.7 months for patients taking the drug. However, patients with hormone receptor-positive (HR+) breast cancer -where the breast cancer cells depend on the hormones estrogen and progesterone to grow - had significantly longer PFS (5.1 months) compared to that of the HR-negative group (84 percent and 11 percent of the enrolled population, respectively). And those who had previously progressed through at least two rounds of hormonal therapy saw significantly greater benefits, suggesting substantial activity in the setting of acquired endocrine resistance.

Palbociclib extends progression-free survival in advanced breast cancer patients

Mayo Clinic researchers identify molecule that lays groundwork for development of pancreatic cancer

A research team led by investigators from Mayo Clinic's campus in Jacksonville, Florida, and the University of Oslo, Norway, have identified a molecule that pushes normal pancreatic cells to transform their shape, laying the groundwork for development of pancreatic cancer -- one of the most difficult tumors to treat.

Their findings, reported in Nature Communications, suggest that inhibiting the gene, protein kinase D1 (PKD1), and its protein could halt progression and spread of this form of pancreatic cancer, and possibly even reverse the transformation.

"As soon as pancreatic cancer develops, it begins to spread, and PKD1 is key to both processes. Given this finding, we are busy developing a PKD1 inhibitor that we can test further," says the study's co-lead investigator, Peter Storz, Ph.D., a cancer researcher at Mayo Clinic.

http://www.nature.com/ncomms/2015/150220/ncomms7200/full/ncomms7200.html

Mayo Clinic researchers identify molecule that lays groundwork for development of pancreatic cancer

Lenvatinib trial offers hope for thyroid cancer patients

A new targeted therapy called lenvatinib has been shown to improve progression-free survival among patients with advanced thyroid cancer that is not responsive to iodine-131.

In a clinical trial of almost 400 patients from 21 different countries, patients who took lenvatinib survived for a median of 18.3 months without displaying any signs of disease progression, while those who were given placebo only had a median progression-free survival of 3.6 months.

"The median progression-free survival in the placebo group in this study was shorter than the 8 months expected, indicating that these patients had aggressive thyroid cancer," write the authors of the study, which was published in the New England Journal of Medicine.

Given the results of this trial, lenvatinib may become the standard treatment for patients resistant to idoine-131, says lead author Martin Schlumberger from the Department of Nuclear Medicine and Endocrine Oncology at Gustave Roussy in France.

Lenvatinib trial offers hope for thyroid cancer patients

New antibody shows promise in increasing survival for patients suffering from influenza, pneumonia

Scientists from NTU Singapore, the world's No. 1 young university, have developed an antibody which boosts the survival chances for patients suffering from influenza and pneumonia.

Proven effective in lab tests, the antibody is now being made suitable for use in humans. The scientists are also using the new antibody to develop a diagnostic kit which can help doctors accurately track the recovery progress of flu and pneumonia patients.

The patent-pending antibody has generated much interest globally. Two biotech multi-national corporations, Abcam based in the United Kingdom and Adipogen International based in the United States, have won the rights to license the antibody. The two multinational companies will produce the antibody for sale to global organisations doing research in vaccine and drug development.

The breakthrough finding was published in the latest issue of the prestigious international peer-reviewed journal Cell Reports.

Ref : http://www.cell.com/cell-reports/abstract/S2211-1247(15)00024-8

New antibody shows promise in increasing survival for patients suffering from influenza, pneumonia