Drug Pipeline
32 drugs associated with X-linked severe combined immunodeficiency
| Drug | Phase | Sponsor | |
|---|---|---|---|
approaches remain | Preclinical | ||
correction is | Preclinical | ||
Alemtuzumab 0.2 mg CAMPATH-1 antigen inhibitor | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
Alemtuzumab 0.3 mg CAMPATH-1 antigen inhibitor | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
anti-thymocyte globulin The mechanism of action by which polyclonal antilymphocyte preparations suppress immune responses is not fully understood. Possible mechanisms by which THYMOGLOBULIN may induce immunosuppression in vivo include: T-cell clearance from the circulation and modulation of T-cell activation, homing, and cytotoxic activities. THYMOGLOBULIN includes antibodies against T-cell markers such as CD2, CD3, CD4, CD8, CD11a, CD18, CD25, CD44, CD45, HLA-DR, HLA Class I heavy chains, and ß2 micro-globulin. In vit | unknown | Fairview University Medical Center | ↗ |
Cyclophosphamide Alkylating agents work by three different mechanisms: 1) attachment of alkyl groups to DNA bases, resulting in the DNA being fragmented by repair enzymes in their attempts to replace the alkylated bases, preventing DNA synthesis and RNA transcription from the affected DNA, 2) DNA damage via the formation of cross-links (bonds between atoms in the DNA) which prevents DNA from being separated for synthesis or transcription, and 3) the induction of mispairing of the nucleotides leading to mutations | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
data collection | unknown | Paul Szabolcs | ↗ |
etoposide Etoposide inhibits DNA topoisomerase II, thereby inhibiting DNA re-ligation. This causes critical errors in DNA synthesis at the premitotic stage of cell division and can lead to apoptosis of the cancer cell. Etoposide is cell cycle dependent and phase specific, affecting mainly the S and G2 phases of cell division. Inhibition of the topoisomerase II alpha isoform results in the anti-tumour activity of etoposide. The drug is also capable of inhibiting the beta isoform but inhibition of this targ | unknown | Fairview University Medical Center | ↗ |
MESNA Mesna reacts chemically with the urotoxic ifosfamide metabolites, acrolein and 4-hydroxy-ifosfamide, resulting in their detoxification. The first step in the detoxification process is the binding of mesna to 4-hydroxy-ifosfamide forming a non-urotoxic 4-sulfoethylthioifosfamide. Mesna also binds to the double bonds of acrolein and to other urotoxic metabolites and inhibits their effects on the bladder. | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
methylprednisolone The short term effects of corticosteroids are decreased vasodilation and permeability of capillaries, as well as decreased leukocyte migration to sites of inflammation. Corticosteroids binding to the glucocorticoid receptor mediates changes in gene expression that lead to multiple downstream effects over hours to days. Glucocorticoids inhibit neutrophil apoptosis and demargination; they inhibit phospholipase A2, which decreases the formation of arachidonic acid derivatives; they inhibit NF-Kapp | unknown | Fairview University Medical Center | ↗ |
prednisone Prednisone is first metabolized in the liver to its active form, prednisolone, a glucocorticoid agonist corticosteroid. The short term effects of corticosteroids are decreased vasodilation and permeability of capillaries, as well as decreased leukocyte migration to sites of inflammation. Corticosteroids binding to the glucocorticoid receptor mediates changes in gene expression that lead to multiple downstream effects over hours to days. Glucocorticoids inhibit neutrophil apoptosis and demargin | unknown | Fairview University Medical Center | ↗ |
Stem Cell Transplantation Removal of necrotic tissue | unknown | Masonic Cancer Center, University of Minnesota | ↗ |