- McNally, Jonathan P;
- Millen, Scott H;
- Chaturvedi, Vandana;
- Lakes, Nora;
- Terrell, Catherine E;
- Elfers, Eileen E;
- Carroll, Kaitlin R;
- Hogan, Simon P;
- Andreassen, Paul R;
- Kanter, Julie;
- Allen, Carl E;
- Henry, Michael M;
- Greenberg, Jay N;
- Ladisch, Stephan;
- Hermiston, Michelle L;
- Joyce, Michael;
- Hildeman, David A;
- Katz, Jonathan D;
- Jordan, Michael B
Antigen-activated lymphocytes undergo extraordinarily rapid cell division in the course of immune responses. We hypothesized that this unique aspect of lymphocyte biology leads to unusual genomic stress in recently antigen-activated lymphocytes and that targeted manipulation of DNA damage-response (DDR) signaling pathways would allow for selective therapeutic targeting of pathological T cells in disease contexts. Consistent with these hypotheses, we found that activated mouse and human T cells display a pronounced DDR in vitro and in vivo. Upon screening a variety of small-molecule compounds, we found that potentiation of p53 (via inhibition of MDM2) or impairment of cell cycle checkpoints (via inhibition of CHK1/2 or WEE1) led to the selective elimination of activated, pathological T cells in vivo. The combination of these strategies [which we termed "p53 potentiation with checkpoint abrogation" (PPCA)] displayed therapeutic benefits in preclinical disease models of hemophagocytic lymphohistiocytosis and multiple sclerosis, which are driven by foreign antigens or self-antigens, respectively. PPCA therapy targeted pathological T cells but did not compromise naive, regulatory, or quiescent memory T-cell pools, and had a modest nonimmune toxicity profile. Thus, PPCA is a therapeutic modality for selective, antigen-specific immune modulation with significant translational potential for diverse immune-mediated diseases.