Imetelstat (development code GRN163L) is an experimental
anticancer drug
Chemotherapy (often abbreviated to chemo and sometimes CTX or CTx) is a type of cancer treatment that uses one or more anti-cancer drugs (chemotherapeutic agents or alkylating agents) as part of a standardized chemotherapy regimen. Chemotherap ...
. The first
telomerase
Telomerase, also called terminal transferase, is a ribonucleoprotein that adds a species-dependent telomere repeat sequence to the 3' end of telomeres. A telomere is a region of repetitive sequences at each end of the chromosomes of most euka ...
inhibitor to enter
clinical trial
Clinical trials are prospective biomedical or behavioral research studies on human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel vaccines, drugs, diet ...
s,
As of early 2023, it was in
Phase 2/
3 trials for various cancer types.
[
Chemically, imetelstat is a synthetic conjugate consisting of three parts: GRN163, a thio ]phosphoramide
Phosphoramide is a chemical compound with the molecular formula O=P(NH2)3. It is a derivative of phosphoric acid in which each of the hydroxyl groups have been replaced with an amino group. Phosphoramide arises from the reaction of phosphoryl ch ...
oligonucleotide
Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics. Commonly made in the laboratory by solid-phase chemical synthesis, these small bits of nucleic acids ...
, and a palmitoyl lipid group.[ GRN163 is the pharmacological component with telomerase inhibition. The palmitic acid moiety is conjugated via a phosphothioate linkage to the backbone of the antisense oligonucleotide. Telomere shortening and lower cell viability are observed after inhibition of telomerase activity in vitro. IC50 values ranged from 50 to 200nM for 10 different pancreatic cell lines. ]
References
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Experimental cancer drugs