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Children’s Tumor Foundation-Funded Research Shows Promise of Dual Drug Tumor Therapy

By September 15, 2011December 18th, 2023Science & Research

Malignant peripheral nerve sheath tumors (MPNSTs) are among the tumors that can occur in NF1, but these malignancies are particularly challenging to treat and control. One reason for this is that MPNST cells appear to undergo changes that make them reistant to traditional treatments such as chemotherapy. Cuurent Children’s Tumor Foundation Young Investigator Awardee Dr. Thomas de Raedt, a postdoctoral fellow in  the laboratory of Dr. Karen Cichowski at Harvard Medical School, has published a paper this week in leading journal ‘Cancer Cell’describing his work in mice, showing a new and seemingly highly effective way to treat MPNSTs via a ‘double-hit’ – using two drugs in combination.  Dr. de Raedt and his colleagues combined the drug rapamycin, which targets the cell signaling element mTOR, with the drug IPI-504, which targets another cell signalling element called HSP90.  In the face of this treatment, the mouse MPNSTs underwent dramatic shrinkage. The tumors also underwent what is termed autophagy, where the cells die and collapse under stress so rapidly that they create spaces where the tumors were.  The success of this approach comes from the fact that combining rapamycin and IPI-504 leads to a collapse of cell structures called the endoplasmic reticulum (ER) and mitochondria which together, constitute the energy source or ‘engine’ of the cell.  By shutting down the ER and mitochondria, the cell dies.  We look forward to seeing the next steps of this exciting finding.

Dr. Cichowski and Dr. de Raedt are also members of the Children’s Tumor Foundation Neurofibromatosis Preclinical Consortium.