Discovering that a gene is associated with a disease, whether breast cancer or schizophrenia or Alzheimer’s disease, is only the beginning of the scientific quest.

Myriad questions follow:

How does this particular gene work to cause this particular disorder?
What other genes interact with it?
How can we interfere in the cascade of genetic events that lead to disease and set matters right?

After identifying mutations in a gene called NOTCH1 as an important contributor to T-cell leukemia, Columbia researchers started enrolling patients in a clinical trial to test the efficacy of targeting NOTCH signaling with a gamma-secretase inhibitor, a drug originally designed as treatment for Alzheimer’s disease.

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