Brain tumor drug receives rare disease designations from the FDA

(Oklahoma Medical Research Foundation) The Food and Drug Administration has given Orphan Disease Designation and Rare Pediatric Disease Designation to OKN-007, an experimental brain cancer treatment currently in Phase 2 clinical trials at eight U.S. sites.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news

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Maurine Stuart credits the Affordable Care Act (ACA) for saving her family. In 2014, Stuart was diagnosed with HELLP syndrome, a rare disease that causes heart, liver and lymphatic problems. As a result, she was unable to continue working full time—which meant losing her employer-sponsored health insurance. But thankfully, she says, that same year, her home state of West Virginia opted in to the 2010 Affordable Care Act’s Medicaid-coverage expansion, and she qualified. Over the next few years, as bad news kept rolling in, ACA protections continued to keep Stuart’s family afloat. When Stuart was diagnosed ...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized Source Type: news
Conclusion Astroblastoma is an extremely rare CNS tumor. Morphological diagnosis is difficult, as the typical astroblastic rosettes may be present also in other CNS tumors, including some gliomas and ependymomas. In fact, AB can be considered as a morphologic pattern, which can be associated with a spectrum of molecular entities. Total resection is the best treatment; the precise role of chemotherapy and radiotherapy is still debated, particularly for high-grade tumors. We believe that DNA-methylation profiles represents an important instrument for confirming diagnosis, predicting prognosis and better defining the molec...
Source: Frontiers in Genetics - Category: Genetics & Stem Cells Source Type: research
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
Investigators at the NCI Laboratory of Immune Cell Biology  discovered an Anti-TNF Induced Apoptosis (ATIA) protein, which protects cells against apoptosis.  ATIA is highly expressed in glioblastoma and astrocytomas and its inhibition results in increased cell sensitivity to TNF-related apoptosis-inducing ligand induced cell death.  Hence, ATIA assays ma y enable clinicians to effectively stratify patients for appropriate treatment.  ATIA exists in a soluble form that can be detected in culture medium of ATIA expressing cells indicating it could be used to develop a non-invasive, blood based diagnostic ...
Source: NIH OTT Licensing Opportunities - Category: Research Authors: Source Type: research
High-grade glioma (HGG) is a group of primary malignant brain tumors with dismal prognosis. Whereas adult HGG has been studied extensively, childhood HGG, a relatively rare disease, is less well-characterized. Here, we present two novel platelet-derived growth factor (PDGF)-driven mouse models of pediatric supratentorial HGG. Tumors developed from two different cells of origin reminiscent of neural stem cells (NSC) or oligodendrocyte precursor cells (OPC). Cross-species transcriptomics showed that both models are closely related to human pediatric HGG as compared with adult HGG. Furthermore, an NSC-like cell-of-origin enha...
Source: Cancer Research - Category: Cancer & Oncology Authors: Tags: Tumor and Stem Cell Biology Source Type: research
Editor’s note: “Narrative Matters: On Our Reading List” is a monthly roundup where we share some of the most compelling health care narratives driving the news and conversation in recent weeks. In this month’s Narrative Matters essay, former Department of Health and Human Services (HHS) Secretary Louis Sullivan writes about growing up in rural Georgia and entering medical school as the only black student in his class. Sullivan graduated from Boston University School of Medicine in 1958 with only $500 in debt — hard to fathom when, today, med students might finish school owing some $150,00...
Source: Health Affairs Blog - Category: Health Management Authors: Tags: Elsewhere@ Health Affairs Featured Narrative Matters On Our Reading List opioids Veterans Source Type: blogs
By most measures, the United States' business-friendly environment has proven to be fertile for medical innovation. Compared to other countries, America has filed the most patents in the life sciences, is conducting most of the world's clinical trials and has published the most biomedical research. That's what makes the medical prominence of Cuba all the more surprising to those who view a free market as an essential driver of scientific discovery. Cuba is very poor, and yet the country has some of the healthiest, most long-lived residents in the world -- as well as a medical invention or two that could run circl...
Source: Science - The Huffington Post - Category: Science Source Type: news
Abstract Constitutional mismatch repair deficiency (CMMRD) syndrome is one of the rare diseases associated with a high risk of cancer. Causative mutations are found in DNA mismatch repair genes PMS2, MSH6, MSH2 or MLH1 that are well known in the context of Lynch syndrome. CMMRD follows an autosomal recessive inheritance trait and is characterized by childhood brain tumors and hematological malignancies as well as gastrointestinal cancer in the second and third decades of life. There is a high risk of multiple cancers, occurring synchronously and metachronously. In general, the prognosis is poor. About one third of...
Source: European Journal of Medical Genetics - Category: Genetics & Stem Cells Authors: Tags: Eur J Med Genet Source Type: research
By Stacy Simon The US Food and Drug Administration (FDA) has approved Alecensa (alectinib) to treat people with advanced ALK-positive non-small cell lung cancer (NSCLC) that has become worse after treatment with another targeted therapy drug called Xalkori (crizotinib). It’s also for people who could not tolerate taking Xalkori in the first place. About 5% of NSCLCs have been found to have a rearrangement in a gene called ALK. This change is most often seen in non-smokers (or light smokers) who have the adenocarcinoma subtype of NSCLC. Doctors may test cancers for changes in the ALK gene to see if drugs that target ...
Source: American Cancer Society :: News and Features - Category: Cancer & Oncology Tags: Lung Cancer - Non-Small Cell Source Type: news
Conclusion Fibrosarcoma is a rare disease with dismal prognosis. Surgery remains the cornerstone of therapy. Radiation confers long term disease control and survival. Chemotherapy needs to be evaluated for these tumours to improve survival.
Source: Journal of the Egyptian National Cancer Institute - Category: Cancer & Oncology Source Type: research
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