AML & Drug Discovery

Acute Myeloid Leukemia (AML) is the most common adult leukemia affecting the elderly. Due to the aggressive nature of this blood cancer, the overall survival rate of AML is less than 20-25% in the long term, thus represents an area of extreme unmet need in translational research.

Our lab is committed to development of new drugs, as well as the repurposing of already FDA approved drugs to patients with myeloid malignancies, including AML and MDS (myelodysplastic syndrome).

As an example of our translational work, we are currently funded by Bristol Myers & Squibb (BMS), to conduct an investigator initiated clinical trial (IIT) using enasidenib (IDHIFA) to improve erythropoiesis (red blood cell production) in patients with lower and intermediate risk MDS and chronic myelomonocytic leukemia (CMML) (NCT05282459). The idea of this clinical trial stemmed from collaborative work with colleagues in lab of Dr. Ravi Majeti, MD,PhD, where we showed that enasidenib can drive increased production of red blood cells (PMID: 33491527) independent of mutation status of isocitrate dehydrogenase type 2.

For more information on this IIT, please visit ClinicalTrials.gov: NCT05282459

https://clinicaltrials.gov/ct2/show/NCT05282459?term=enasidenib+stanford&cond=MDS&draw=2&rank=1