Targeting cancer via its genomic profile
Chemotherapy can be the standard of care for some cancer types; however the treatment does not completely distinguish between healthy or cancerous cells, and destroy both of them. For many patients these treatments can be debilitating and unfortunately provide little hope for full recovery from some types of cancer. We are continuously researching treatments that could provide patients with new, effective, and targeted options.
A novel treatment approach driven by cancer genomics
Together with our partner, the Broad Institute of MIT and Harvard, we are researching small molecules that selectively target cancer genome alterations in cancerous cells to stop the spread of cancer in the body.
In order for either healthy or cancerous cells to multiply, they require DNA building blocks. By removing the building blocks that cancerous cells require to multiply, they can be stopped from spreading in the body. Together with the Broad Institute, we have identified a substance which inhibits the enzyme dihydroorotate dehydrogenase (DHODH) which is involved in the production of these so-called DNA building blocks. This method only affects cancerous cells, rather than healthy cells, as they cannot obtain sufficient quantities of DNA blocks from the body to multiply and spread.
Could this provide new hope for patients with acute myeloid leukemia?
Acute myeloid leukemia (AML) is one such disease where the current standard of care is chemotherapy. We hope that our research into this new mechanism of targeting cancer could provide AML patients with new options. The project has now advanced into clinical development. You can hear first-hand from a lead chemist who worked on this novel approach in our interview with Steven Ferrara. Steven is a research scientist at the Broad Institute, with whom we collaborated on this project.