Ponatinib
Ponatinib, sold under the brand name Iclusig, is a medication used for the treatment of chronic myeloid leukemia and Philadelphia chromosome–positive (Ph+) acute lymphoblastic leukemia. It was developed by Ariad Pharmaceuticals. It is a multi-targeted tyrosine-kinase inhibitor. Some forms of chronic myeloid leukemia, those that have the T315I mutation, are resistant to current therapies such as imatinib. Ponatinib has been designed to be effective against these types of tumors. Ponatinib was approved for medical use in the United States in December 2012, and in the European Union in July 2013. Medical uses Ponatinib in indicated for the treatment of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia and chronic myeloid leukemia. In March 2024, the FDA expanded the indication to include the treatment, with chemotherapy, for adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia. Adverse effects The US Food and D ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Bcr-Abl Tyrosine-kinase Inhibitor
Bcr-Abl tyrosine-kinase inhibitors (TKI) are the first-line therapy for most patients with chronic myelogenous leukemia (CML). More than 90% of CML cases are caused by a chromosomal abnormality that results in the formation of a so-called Philadelphia chromosome. This abnormality was discovered by Peter Nowell in 1960 and is a consequence of fusion between the Abelson ( Abl) tyrosine kinase gene at chromosome 9 and the break point cluster ( Bcr) gene at chromosome 22, resulting in a chimeric oncogene (Bcr-Abl) and a constitutively active Bcr-Abl tyrosine kinase that has been implicated in the pathogenesis of CML. Compounds have been developed to selectively inhibit the tyrosine kinase. Before the 2001 U.S. Food and Drug Administration (FDA) approval of imatinib, no drugs were available to alter the natural progression of CML. Only cytotoxic drugs such as busulfan, hydroxyurea or interferon-alpha (rIFN-α) were utilized. Even though the first Bcr-Abl TK inhibitor was named "the mag ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Ariad Pharmaceuticals
ARIAD Pharmaceuticals, Inc. was an American oncology company, now part of Takeda Oncology, which was founded in 1991 by Harvey J. Berger, M.D. and headquartered in Cambridge, Massachusetts. ARIAD engaged in the discovery, development, and commercialization of medicines for cancer patients. ARIAD’s most prominent drug discoveries include Iclusig, designed for patients with all forms of Philadelphia chromosome-positive h+ chronic myeloid leukemia (CML) or Ph+ acute lymphoblastic leukemia (ALL) who are resistant to or unable to tolerate other tyrosine kinase inhibitors, and brigatinib, a lung cancer drug which has completed its registration trial in ALK fusion driven non-small cell lung cancer as of June 2016 and was approved in the U.S. in April 2017. In January 2017, Takeda announced it would acquire ARIAD for $5.2 billion, expanding the company's oncology and hematology business. On February 16, 2017, Takeda Pharmaceuticals, Ltd. announced it had completed its acquisition o ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Chronic Myeloid Leukemia
Chronic myelogenous leukemia (CML), also known as chronic myeloid leukemia, is a cancer of the white blood cells. It is a form of leukemia characterized by the increased and unregulated growth of myeloid cells in the bone marrow and the accumulation of these cells in the blood. CML is a clonal bone marrow stem cell disorder in which a proliferation of mature granulocytes (neutrophils, eosinophils and basophils) and their precursors is found; characteristic increase in basophils is clinically relevant. It is a type of myeloproliferative neoplasm associated with a characteristic chromosomal translocation called the Philadelphia chromosome. CML is largely treated with targeted drugs called tyrosine-kinase inhibitors (TKIs) which have led to dramatically improved long-term survival rates since 2001. These drugs have revolutionized treatment of this disease and allow most patients to have a good quality of life when compared to the former chemotherapy drugs. In Western countries, CM ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Oral Administration
Oral administration is a route of administration whereby a substance is taken through the Human mouth, mouth, swallowed, and then processed via the digestive system. This is a common route of administration for many medications. Oral administration can be easier and less painful than other routes of administration, such as Injection (medicine), injection. However, the onset of action is relatively low, and the effectiveness is reduced if it is not absorbed properly in the digestive system, or if it is broken down by digestive enzymes before it can reach the bloodstream. Some medications may cause gastrointestinal side effects, such as nausea or vomiting, when taken orally. Oral administration can also only be applied to conscious patients, and patients able to swallow. Terminology ''Per os'' (; ''P.O.'') is an adverbial phrase meaning literally from Latin "through the mouth" or "by mouth". The expression is used in medicine to describe a treatment that is taken orally (but not ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Indicated
In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis is the assessment that a particular medical condition is present while an indication is a reason for use. The opposite of an indication is a contraindication, a reason to withhold a certain medical treatment because the risks of treatment clearly outweigh the benefits. In the United States, indications for prescription drugs are approved by the FDA. Indications are included in the Indications and Usage section of the Prescribing Information. The primary role of this section of labeling is to enable health care practitioners to readily identify appropriate therapies for patients by clearly communicating the drug's approved indication(s). The Indications and Usage section states the disease or condition, or manifestation or symptoms thereof ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Alkyne Derivatives
\ce \ce Acetylene \ce \ce \ce Propyne \ce \ce \ce \ce 1-Butyne In organic chemistry, an alkyne is an unsaturated hydrocarbon containing at least one carbon—carbon triple bond. The simplest acyclic alkynes with only one triple bond and no other functional groups form a homologous series with the general chemical formula . Alkynes are traditionally known as acetylenes, although the name ''acetylene'' also refers specifically to , known formally as ethyne using IUPAC nomenclature. Like other hydrocarbons, alkynes are generally hydrophobic. Structure and bonding In acetylene, the H–C≡C bond angles are 180°. By virtue of this bond angle, alkynes are rod-like. Correspondingly, cyclic alkynes are rare. Benzyne cannot be isolated. The C≡C bond distance of 118 picometers (for C2H2) is much shorter than the C=C distance in alkenes (132 pm, for C2H4) or the C–C bond in alkanes (153 pm). : The triple bond is very strong with a bond strength of 839 kJ/mol. The si ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Drugs Developed By Takeda Pharmaceutical Company
A drug is any chemical substance other than a nutrient or an essential dietary ingredient, which, when administered to a living organism, produces a biological effect. Consumption of drugs can be via inhalation, injection, smoking, ingestion, absorption via a patch on the skin, suppository, or dissolution under the tongue. In pharmacology, a drug is a chemical substance, typically of known structure, which, when administered to a living organism, produces a biological effect. A pharmaceutical drug, also called a medication or medicine, is a chemical substance used to treat, cure, prevent, or diagnose a disease or to promote well-being. Traditionally drugs were obtained through extraction from medicinal plants, but more recently also by organic synthesis. Pharmaceutical drugs may be used for a limited duration, or on a regular basis for chronic disorders. Classification Pharmaceutical drugs are often classified into drug classes—groups of related drugs that have simila ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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National Institute For Health And Care Excellence
The National Institute for Health and Care Excellence (NICE) is an executive non-departmental public body of the Department of Health and Social Care (United Kingdom), Department of Health and Social Care. As the national health technology assessment body of England, it is responsible for judging the cost-effectiveness of medicines and making them available on the NHS through reimbursement, with its judgements informing decisions in Wales and Northern Ireland. It also provides a range of clinical guidance to the NHS in England and Wales, which are considered by Northern Ireland. History Organisational history It was set up as the National Institute for Clinical Excellence in 1999, and on 1 April 2005 joined with the Health Development Agency to become the new National Institute for Health and Clinical Excellence (still abbreviated as NICE). Following the Health and Social Care Act 2012, NICE was renamed the National Institute for Health and Care Excellence on 1 April ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Cancer Drugs Fund
The Cancer Drugs Fund (CDF) was introduced in England in 2011. It was established in order to provide a means by which National Health Service (NHS) patients in England could receive cancer drugs that had been rejected by National Institute for Health and Care Excellence because they were not cost effective. Its establishment was confirmed by the UK government's coalition agreement in 2010, and by the White Paper, Equity and excellence – Liberating the NHS. Starting in April 2011, the fund paid for nearly 100,000 people with cancer to access treatments. It was closed to new drugs from October 2015 to 29 July 2016 in line with the recommendation of the independent Cancer Taskforce report, which called for urgent reform to put the CDF on a more sustainable footing. Objectives Following the reforms in 2016 the objectives were updated. The new arrangements put it on a more sustainable footing with 3 key objectives: *patients have faster access to the most promising new cancer trea ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Bcr-abl Fusion Protein
The Philadelphia chromosome or Philadelphia translocation (Ph) is an abnormal version of chromosome 22 where a part of the '' Abelson murine leukemia'' 1 (''ABL1'') gene on chromosome 9 breaks off and attaches to the '' breakpoint cluster region'' (''BCR'') gene in chromosome 22. The balanced reciprocal translocation between the long arms of 9 and 22 chromosomes (9; 22) (q34; q11)results in the fusion gene ''BCR::ABL1''. The oncogenic protein with persistently enhanced tyrosine kinase (TK) activity transcribed by the ''BCR''::''ABL1'' fusion gene can lead to rapid, uncontrolled growth of immature white blood cells that accumulates in the blood and bone marrow. The Philadelphia chromosome is present in the bone marrow cells of a vast majority ''chronic myelogenous leukemia'' (CML) patients. The expression patterns off different BCR-ABL1 transcripts vary during the progression of CML. Each variant is present in a distinct leukemia phenotype and can be used to predict response to ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |