New animal model for Gastrointestinal Stromal Tumor

Gastrointestinal stromal tumor (GIST) is one of the most common mesenchymal tumors of the gastrointestinal tract. GISTs occur in 10-20 per one million people. Thus, GIST has been named as an orphan drug disease. Scientists at the University of Göttingen developed a new animal drug screening model for GIST, which is caused by dysfunction of the Hedgehog pathway.

Challenge

Typically, GIST formation is driven by mutations in the KIT/CD117 gene. However, not all tumors are driven by those mutations. Hence, there is a need for animal models reflecting this situation.

Our Solution

Gross appearance of tumors of KO mice. Source: Penelope Pelczar.

Scientists at the University of Göttingen developed a new animal drug screening model for GIST, which is caused by dysfunction of the Hedgehog pathway. This is the first immunological GIST mouse model with an aberrant Hedgehog signaling independent of Kit, demonstrating the importance of this particular pathway in GIST formation.

Advantages

Tumors of this novel mouse model have a GIST-like histology. Source: Walter J. Schulz-Schaeffer.

Novel GIST animal model caused by aberrant Hedgehog pathway independent of KIT mutations.
Conditional gene knock-out of a key player within Hedgehog pathway in monocytes (LysM-positive cells).
Severe gastrointestinal phenotype without affecting otherwise normal development.
Good and effective breeding.

Applications

Excellent drug screening model for new targets within Hedgehog signaling.
Efficacy testing of potential new therapeutics for GIST.

Developmental Status

Fully characterized new mouse model for GIST.
Proof of concept with Imatinib treatment.

Tumors of GIST-mice are responsive to Imatinib, which is used in the treatment of human GIST. Source: Penelope Pelczar.

Patent Status

No IPR. We are selling/licensing this mouse model.

References

Pelczar et al.: Inactivation of Patched1 in Mice Leads to Development of Gastrointestinal Stromal-Like Tumors that Express Pdgfr alpha but not Kit. Gastroenterology, 2013, 144:134 –144.

Contact

Dr. Stefan Uhle
Patent Manager Life Science
E-Mail: suhle(at)sciencebridge.de
Tel.: +49 551 30724 154
Reference: BioC-1466-UMG

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