Involvement of Patient Organisations in Research and Development of Orphan Drugs for Rare Diseases in Europe.

Involvement of patient organisations in research and development of orphan drugs for rare diseases in europe.

Filed under: Drug and Alcohol Rehabilitation

Mol Syndromol. 2012 Nov; 3(5): 237-43
Mavris M, Le Cam Y

Patients’ representatives have an increasingly present voice in all aspects of drug development from fundamental research through regulatory processes to health technology assessment. Although major advances have been made in raising awareness and increasing funding for rare diseases, important challenges remain in terms of best use of resources, coordinating efforts and improving policy. This article describes actions taken by rare disease patients’ organisations as well as initiatives at the national and European levels to promote research into rare diseases. A survey conducted by EURORDIS (European Organisation for Rare Diseases) on the support (financial and non-financial) provided by patients’ organisations in rare disease research is described as well as the involvement of patients’ representatives in regulatory processes for medicinal products at the European Medicines Agency. The importance of including patients’ groups in fundamental and clinical research as equal partners has become a fact that clearly contributes to the success of an application and the research conducted.
HubMed – drug


Lipoxygenase and Cyclooxygenase Pathways and Colorectal Cancer Prevention.

Filed under: Drug and Alcohol Rehabilitation

Curr Colorectal Cancer Rep. 2012 Dec; 8(4): 316-324
Rao CV, Janakiram NB, Mohammed A

Colorectal cancer is one of the commonest malignancies in both men and women. In spite of significant progress in screening and in surgical and therapeutic interventions, colorectal cancer (CRC) is still a major public health problem. Accumulating evidence suggests that targeting inflammatory pathways may provide protection against the development of CRC. Eicosanoids derived from the enzymes cyclooxygenase (COX) and lipoxygenase (LOX) may contribute to CRC carcinogenesis. Approaches for targeting COX-1 and COX-2 with traditional nonsteroidal anti-inflammatory agents or targeting COX-2 with specific inhibitors are highly successful at the preclinical and clinical levels; however, large-scale clinical applicability of these agents is limited owing to unwanted side effects. Emerging studies suggests that 5-LOX-derived leukotrienes may contribute to colon tumor development and risk of thrombotic events. Thus, developing drugs that target both 5-LOX and COX-2 may provide a safer strategy. In this review, we discuss evidence for the involvement of 5-LOX in colon tumor development and targeting 5-LOX and COX-2 with synthetic and naturally occurring agents for CRC prevention.
HubMed – drug


Clinical factors related to long-term administration of sorafenib in patients with hepatocellular carcinoma.

Filed under: Drug and Alcohol Rehabilitation

Cancer Manag Res. 2012; 4: 423-9
Nojiri S, Kusakabe A, Fujiwara K, Shinkai N, Matsuura K, Iio E, Miyaki T, Nomura T, Sobue S, Sano H, Hasegawa I, Ohno T, Takahashi Y, Orito E, Joh T

Sorafenib has been approved in the indication of unresectable hepatocellular carcinoma, but there are many cases in which administration of the drug is discontinued due to severe side effects. In this study, we compared the characteristics of patients who continued and discontinued sorafenib.Ninety-six patients (75 men and 21 women) were initiated on sorafenib from July 2009 through September 2011. The patient characteristics of interest included gender, age, etiology, Child-Pugh classification, treatment history and frequency, and levels of ?-fetoprotein, des- gamma-carboxy prothrombin, aspartate amino acid transferase, and alanine aminotransferase. Duration of administration of sorafenib and reasons for its discontinuation were compared.Median overall survival was 11.8 months. Discontinuation of sorafenib within 90 days was identified as an independent prognostic factor for overall survival on multivariate analysis (P < 0.0001). Transarterial chemoembolization performed six times or more (P = 0.013) was also identified as an independent factor contributing to discontinuation of sorafenib within 90 days in multivariate analysis. Patients who received sorafenib for ?90 days had significantly longer overall survival than those who discontinued it (P < 0.0001).Prolonged treatment with sorafenib is an important factor in achieving extended overall survival. We recommend starting sorafenib before latent liver damage has occurred as a result of too many transarterial chemoembolization procedures. HubMed – drug



9969204.mp4 – Downtown Tacoma Campus Making lasting changes means addressing the complex issues that result in homelessness or addiction. Our Downtown Campus provides complete services to help stabilize and transition people to recovery and self-sufficiency. The Good Neighbor Cafe is the “front door” to many of the Rescue Mission’s facilities and programs, offering healthy, well-balanced meals to hundreds of homeless men and women every day. Short-term shelter is offered at the Downtown Campus. And because homelessness is frequently linked to other problems such as drug or alcohol addiction, rehabilitation services are housed at this location as well. Personal support, educational resources part of holistic approach To help move people to permanent change, case managers work one-to-one with clients, identifying needs and setting goals. Our community classroom, Challenge Learning Center, helps people with GED preparation, adult basic education and resume assistance. Life transformation is supported by hopeful, helpful staff, resources for recovery and job training and our New Life program, with bible study, daily prayer and other spiritual support. In a typical winter month, the Downtown Campus in Tacoma: Serves over 16500 meals Conducts nearly 1500 hours of education and job training classes Houses 100 homeless people every night and up to 150 people during the cold winter months Provides more than 2000 hours of drug and alcohol rehabilitation services Offers one-to-one case management


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