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Motixafortide
Motixafortide, sold under the brand name Aphexda, is a medication used for the treatment of multiple myeloma. Motixafortide is a hematopoietic stem cell mobilizer and a CXCR4 antagonist. It is given by subcutaneous injection. The most common side effects include injection site reactions, pruritus, flushing, and back pain. Motixafortide was approved for medical use in the United States in September 2023. Medical uses Motixafortide is indicated, in combination with filgrastim, a granulocyte-colony stimulating factor (G-CSF), to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in people with multiple myeloma. Adverse effects Motixafortide can cause injection site reactions, anaphylactic shock, and hypersensitivity reactions. Tumor cell mobilization in people with leukemia, leukocytosis, potential for tumor cell mobilization, and embryo-fetal toxicity can also be caused by motixafortide. History The US ...
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Multiple Myeloma
Multiple myeloma (MM), also known as plasma cell myeloma and simply myeloma, is a cancer of plasma cells, a type of white blood cell that normally produces antibody, antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, anemia, Kidney failure, renal insufficiency, and infections may occur. Complications may include hypercalcemia and amyloidosis. The cause of multiple myeloma is unknown. Risk factors include obesity, radiation exposure, family history, age and certain chemicals. There is an increased risk of multiple myeloma in certain occupations. This is due to the occupational exposure to aromatic hydrocarbon solvents having a role in causation of multiple myeloma. Multiple myeloma is the result of a multi-step malignant transformation, and almost universally originates from the pre-malignant stage monoclonal gammopathy of undetermined significance (MGUS). As MGUS evolves into MM, another pre-stage of the disease is reached, known as Smouldering ...
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Subcutaneous Administration
Subcutaneous administration is the insertion of medications beneath the skin either by injection or infusion. A subcutaneous injection is administered as a bolus into the subcutis, the layer of skin directly below the dermis and epidermis, collectively referred to as the cutis. The instruments are usually a hypodermic needle and a syringe. Subcutaneous injections are highly effective in administering medications such as insulin, morphine, diacetylmorphine and goserelin. Subcutaneous administration may be abbreviated as SC, SQ, subcu, sub-Q, SubQ, or subcut. Subcut is the preferred abbreviation to reduce the risk of misunderstanding and potential errors. Subcutaneous tissue has few blood vessels and so drugs injected into it are intended for slow, sustained rates of absorption, often with some amount of depot effect. Compared with other routes of administration, it is slower than intramuscular injections but still faster than intradermal injections. Subcutaneous infusion (as ...
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Medication
Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmacotherapy) is an important part of the medicine, medical field and relies on the science of pharmacology for continual advancement and on pharmacy for appropriate management. Drugs are Drug class, classified in many ways. One of the key divisions is by level of controlled substance, control, which distinguishes prescription drugs (those that a pharmacist dispenses only on the medical prescription) from over-the-counter drugs (those that consumers can order for themselves). Medicines may be classified by mode of action, route of administration, biological system affected, or therapeutic effects. The World Health Organization keeps a list of essential medicines. Drug discovery and drug development are complex and expensive endeavors undertake ...
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Hematopoietic Stem Cell
Hematopoietic stem cells (HSCs) are the stem cells that give rise to other blood cells. This process is called haematopoiesis. In vertebrates, the first definitive HSCs arise from the ventral endothelial wall of the embryonic aorta within the (midgestational) aorta-gonad-mesonephros region, through a process known as endothelial-to-hematopoietic transition. In adults, haematopoiesis occurs in the red bone marrow, in the core of most bones. The red bone marrow is derived from the layer of the embryo called the mesoderm. Haematopoiesis is the process by which all mature blood cells are produced. It must balance enormous production needs (the average person produces more than 500 billion blood cells every day) with the need to regulate the number of each blood cell type in the circulation. In vertebrates, the vast majority of hematopoiesis occurs in the bone marrow and is derived from a limited number of hematopoietic stem cells that are multipotent and capable of extensive ...
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CXCR4 Antagonist
A CXCR4 antagonist is a substance which blocks the CXCR4 receptor and prevent its activation. Blocking the receptor stops the receptor's ligand, CXCL12, from binding which prevents downstream effects. CXCR4 antagonists are especially important for hindering cancer progression because one of the downstream effects initiated by CXCR4 receptor activation is cell movement which helps the spread of cancer, known as metastasis. The CXCR4 receptor has been targeted by antagonistic substances since being identified as a co-receptor in HIV and assisting the development of cancer. Macrocyclic ligands have been utilised as CXCR4 antagonists. Plerixafor is an example of a CXCR4 antagonist, and has approvals (e.g. US FDA 2008) for clinical use (to mobilize hematopoietic stem cells). BL-8040 is a CXCR4 antagonist that has undergone clinical trials (e.g. in various leukemias), with one planned for pancreatic cancer (in combination with pembrolizumab). Previously called BKT140, it is a synthetic c ...
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Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is ...
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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 ...
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Filgrastim
Filgrastim, sold under the brand name Neupogen among others, is a medication used to treat low neutrophil count. Low neutrophil counts may occur with HIV/AIDS, following chemotherapy or radiation poisoning, or be of an unknown cause. It may also be used to increase white blood cells for gathering during leukapheresis. It is given either by injection into a vein or under the skin. Filgrastim is a leukocyte growth factor. Common side effects include fever, cough, chest pain, joint pain, vomiting, and hair loss. Severe side effects include splenic rupture and allergic reactions. It is unclear if use in pregnancy is safe for the baby. Filgrastim is a recombinant form of the naturally occurring granulocyte colony-stimulating factor (G-CSF). It works by stimulating the body to increase neutrophil production. Filgrastim was approved for medical use in the United States in 1991. It is on the World Health Organization's List of Essential Medicines. Filgrastim biosimilar med ...
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Granulocyte-colony Stimulating Factor
Granulocyte colony-stimulating factor (G-CSF or GCSF), also known as colony-stimulating factor 3 (CSF 3), is a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream. Functionally, it is a cytokine and hormone, a type of colony-stimulating factor, and is produced by a number of different tissues. The pharmaceutical analogs of naturally occurring G-CSF are called filgrastim and lenograstim. G-CSF also stimulates the survival, proliferation, differentiation, and function of neutrophil precursors and mature neutrophils. Biological function G-CSF is produced by endothelium, macrophages, and a number of other immune cells. The natural human glycoprotein exists in two forms, a 174- and 177-amino-acid-long protein of molecular weight 19,600 grams per mole. The more-abundant and more-active 174-amino acid form has been used in the development of pharmaceutical products by recombinant DNA (rDNA) technology. ...
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Autologous Transplantation
Autologous stem-cell transplantation (also called autogenous, autogenic, or autogenic stem-cell transplantation and abbreviated auto-SCT) is the autologous transplantation of stem cells—that is, transplantation in which stem cells ( undifferentiated cells from which other cell types develop) are removed from a person, stored, and later given back to that same person. Although it is most frequently performed with hematopoietic stem cells (precursors of blood-forming cells) in hematopoietic stem cell transplantation, cardiac cells have also been used successfully to repair damage caused by heart attacks. Autologous stem-cell transplantation is distinguished from allogenic stem cell transplantation where the donor and the recipient of the stem cells are different people. It can be also used as an Assisted reproductive technology to improve the reproductive outcomes. In a first step the bone marrow derived stem cells are mobilized. After that they will be isolated from the periph ...
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