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To bring us closer to curing cancer, a combination of effective drugs with non-overlapping mechanisms of action is required. 6 In all these examples, an effective backbone drug was first developed, before adding one or more drugs to establish the new regimens. 2012);12(4):237-251. Front Pharmacol. 2018;9:1300.
I work in the Centre for Cancer Drug Discovery (CCDD) at The Institute of Cancer Research in London, which is an academic drug discovery centre. The mission of the CCDD is to discover novel small-molecule therapeutics for the treatment of cancer and progress them to hypothesis testing phase 1 clinical trials.
These results, along with data from another clinical trial expected to begin later this year, will be used by Vertex to select which next-generation targeted drug or combination of drugs will move forward into larger scale Phase III studies. About 30,000 Americans have CF.
After a whirlwind tour of the cutting-edge robotics facility for high throughput screening of smallmolecules, she joined me and NCATS Director Dr. Chris Austin in announcing that, thanks to an innovative public-private partnership, an experimental drug originally developed to fight cancer is now showing promise against Alzheimer’s disease.
“LY2456302 is a novel, potent, orally-bioavailable smallmolecule kappa-selective antagonist with activity in animal models predictive of efficacy in mood and addictive disorders” Neuropharmacology. Rankovic Z, Hargreaves R, Bingham M (2012). Drug Discovery for Psychiatric Disorders. Drug Discovery.
Although therapeutics have contributed to improvements in survival, the five-year relative survival rate for patients with MM was just 57% between 2012 and 2018. Note: Both analyses focused only on studies of smallmolecules, antibodies and biologics; studies of CAR T therapies were not included.
BRUKINSA (zanubrutinib) is a smallmolecule inhibitor of Bruton’s tyrosine kinase (BTK), discovered by BeiGene scientists, that is currently being evaluated globally in a broad pivotal clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. 2012 Aug 2;120(5):947-59.
What are macrocycles and why are they interesting for drug discovery? Traditionally, drug discovery has focused on small-molecule therapeutics, typically with a molecular weight of less than 500 Daltons. 2 Typically, small-moleculedrugs target active sites buried inside proteins.
Strategies for the synthesis and development of complex active pharmaceutical ingredients While the prevalence of biologics has grown within the pharmaceutical sector over recent decades, smallmolecules remain important and can offer significant advantages. References [1] Clemons et al.
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