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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 antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, anemia, kidney dysfunction, and infections may occur. Complications may include hypercalcemia and amyloidosis.
Basiliximab, sold under the brand name Simulect, is a monoclonal antibody used to prevent rejection in kidney transplants. It is a chimeric mouse-human monoclonal antibody to the α chain (CD25) of the IL-2 receptor of T cells. It is used in combination with other medicines used to prevent organ rejection.
Hematopoietic stem-cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood in order to replicate inside of a patient and to produce additional normal blood cells. It may be autologous, allogeneic or syngeneic.
Graft-versus-host disease (GvHD) is a syndrome, characterized by inflammation in different organs. GvHD is commonly associated with bone marrow transplants and stem cell transplants.
A cord blood bank is a facility which stores umbilical cord blood for future use. Both private and public cord blood banks have developed in response to the potential for cord blood in treating diseases of the blood and immune systems. Public cord blood banks accept donations to be used for anyone in need, and as such function like public blood banks. Traditionally, public cord blood banking has been more widely accepted by the medical community. Private cord blood banks store cord blood solely for potential use by the donor or donor's family. Private banks typically charge around $2,000 for the collection and around $200 a year for storage.
Cord blood is blood that remains in the placenta and in the attached umbilical cord after childbirth. Cord blood is collected because it contains stem cells, which can be used to treat hematopoietic and genetic disorders such as cancer. There is growing interest from cell therapeutics companies in developing genetically modified allogenic natural killer cells from umbilical cord blood as an alternative to CAR T cell therapies for rare diseases.
Gemtuzumab ozogamicin, sold under the brand name Mylotarg, is an antibody-drug conjugate that is used to treat acute myeloid leukemia.
Maribavir, sold under the brand name Livtencity, is an antiviral medication that is used to treat post-transplant cytomegalovirus (CMV). Maribavir is a cytomegalovirus pUL97 kinase inhibitor that works by preventing the activity of human cytomegalovirus enzyme pUL97, thus blocking virus replication.
Subcutaneous T-cell lymphoma is a cutaneous condition that most commonly presents in young adults, and is characterized by subcutaneous nodules. Common symptoms include fever, fatigue, and pancytopenia.
Daratumumab, sold under the brand name Darzalex, is an anti-cancer monoclonal antibody medication. It binds to CD38, which is overexpressed in multiple myeloma cells. Daratumumab was originally developed by Genmab, but it is now being jointly developed by Genmab along with the Johnson & Johnson subsidiary Janssen Biotech, which acquired worldwide commercialization rights to the drug from Genmab.
Letermovir is an antiviral drug for the treatment of cytomegalovirus (CMV) infections. It has been tested in CMV infected patients with allogeneic stem cell transplants and may also be useful for other patients with a compromised immune system such as those with organ transplants or HIV infections. The drug was initially developed by the anti-infective division at Bayer, which became AiCuris Anti-infective Cures AG through a spin-out and progressed the development to end of Phase 2 before the project was sold to Merck & Co for Phase 3 development and approval.
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.
Venetoclax, sold under the brand names Venclexta and Venclyxto, is a medication used to treat adults with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), or acute myeloid leukemia (AML).
Andrew Louis Pecora is an American hematologist and oncologist involved in research on the use of stem cells and oncolytic viruses to treat diseases, including cancer. He is the CEO of Outcomes Matter Innovations. As of 2020, he is on the Board of Directors Celularity, Inc. (since 2017) and founder and Executive Chairman, COTA, Inc.. Previously, he was chief innovations officer, professor and vice president of cancer services at the John Theurer Cancer Center, part of the Hackensack University Medical Center. He is a professor of medicine and oncology at Georgetown University.
Tisagenlecleucel, sold under the brand name Kymriah, is a CAR T cells medication for the treatment of B-cell acute lymphoblastic leukemia (ALL) which uses the body's own T cells to fight cancer.
Lisocabtagene maraleucel, sold under the brand name Breyanzi, is a cell-based gene therapy used to treat large B-cell lymphoma.
Belumosudil, sold under the brand name Rezurock among others, is a medication used for the treatment of chronic graft versus host disease (cGvHD). It is in the class of drugs known as serine/threonine kinase inhibitors. Specifically, it is an inhibitor of Rho-associated coiled-coil kinase 2. Belumosudil binds to and inhibits the serine/threonine kinase activity of ROCK2. This inhibits ROCK2-mediated signaling pathways which play major roles in pro- and anti-inflammatory immune cell responses. A genomic study in human primary cells demonstrated that the drug also has effects on oxidative phosphorylation, WNT signaling, angiogenesis, and KRAS signaling.
Selinexor sold under the brand name Xpovio among others, is a selective inhibitor of nuclear export used as an anti-cancer medication. It works by blocking the action of exportin 1 and thus blocking the transport of several proteins involved in cancer-cell growth from the cell nucleus to the cytoplasm, which ultimately arrests the cell cycle and leads to apoptosis. It is the first drug with this mechanism of action.
Teclistamab, sold under the brand name Tecvayli, is a human bispecific monoclonal antibody used for the treatment of relapsed and refractory multiple myeloma. It is a bispecific antibody that targets the CD3 receptor expressed on the surface of T-cells and B-cell maturation antigen (BCMA), which is expressed on the surface of malignant multiple myeloma B-lineage cells.
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease.
References
- ↑ "Archived copy". Food and Drug Administration . Archived from the original on 21 April 2023. Retrieved 21 April 2023.
{{cite web}}: CS1 maint: archived copy as title (link) - ↑ "Omisirge". U.S. Food and Drug Administration. 17 April 2023. Archived from the original on 21 April 2023. Retrieved 20 April 2023.
- 1 2 3 4 5 6 7 8 9 10 11 12 13 14 "FDA Approves Cell Therapy for Patients with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation". U.S. Food and Drug Administration (FDA) (Press release). 18 April 2023. Archived from the original on 21 April 2023. Retrieved 20 April 2023.
This article incorporates text from this source, which is in the public domain . - 1 2 3 4 5 6 7 "FDA approves omidubicel to reduce time to neutrophil recovery and infection in patients with hematologic malignancies". U.S. Food and Drug Administration (FDA). 17 April 2023. Archived from the original on 21 April 2023. Retrieved 20 April 2023. This article incorporates text from this source, which is in the public domain .
