Small Molecules are naturally occurring or synthetic organic compounds that interfere or augment biological processes.
Small Molecules. Taiga is in the process of identifying novel drugs that selectively inhibit growth or viability of cancer cells while sparing normal cells. Taiga is currently pursuing IND-enabling research on lead candidates that have shown promise in the specific targeting of stem leukemic cells and leukemias, but did not affect normal blood stem cells. Our goal is to reduce the impact of chemotherapy on the patients while undergoing treatment, and greatly increase their quality of life.
Small molecules represent a bridging of historical scientific observation and cutting edge chemical synthesis. Historically, the most effective drugs we have had until recently have been natural products – these are all small molecules. We now have the ability to harness combinatorial chemistry to synthesize novel species of small molecules that do not commonly occur in nature. Furthermore, once a candidate is identified, medicinal chemistry allows for the subsequent modification of the small molecule to better suit our therapeutic goals.
Limitations of current small molecule development:
1. Top-down approaches. By focusing on a specific enzymatic function, and targeting a disease later, issues arise regarding delivery in vivo such as solubility, half-life and targeting the malignant cell type.
2. Side effects. If a small molecule can be generated against a specific target, unwanted side effects are often discovered only after several expensive stages of development:
Our cellular products enable these screens – the ability to produce many blood stem cells has been an enabling breakthrough in this setting. Until now, the logistics, expense and resources required for performing such screens have all been rate-limiting steps that have prohibited the identification of such compounds. Our early screening efforts have already identified candidates that are being moved into IND-enabling studies.
Our proprietary screening platform allows us to perform differential screens to identify novel agents that will specifically kill leukemic stem cells, but spare normal blood stem cells. We can also search for novel drugs that can induce differentiation of normal or cancerous blood stem cells in order to curb their ability to reproduce. The drug candidates will then be used to determine the specific molecular mechanism of action. To date, none of the natural products that have been developed were kept out of clinical use for lack of understanding of the molecular mode of action.
We have committed to producing new treatments for blood cancers that are effective and have minimal effects on normal or non-cancerous cells. Our high standards for pre-clinical testing in genetically validated animal models of disease help assure that drugs with serious side effects can be eliminated from consideration for further human use.
