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Related Experiment Video

Updated: Jul 7, 2026

Chemical Inactivation of the E3 Ubiquitin Ligase Cereblon by Pomalidomide-based Homo-PROTACs

Chemical Inactivation of the E3 Ubiquitin Ligase Cereblon by Pomalidomide-based Homo-PROTACs

Published on: May 15, 2019

Thalidomide in advanced mastocytosis.

Gandhi Damaj¹, Emmanuelle Bernit, David Ghez

¹Department of Haematology, Centre Hospitalier Universitaire, Amiens, France. damaj.gandhi@chu-amiens.fr

British Journal of Haematology

|March 1, 2008

Summary

Thalidomide shows promise for treating advanced systemic mastocytosis (SM), an orphan disease. Two patients achieved partial remission, with rapid symptom improvement, suggesting thalidomide

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Chemical Inactivation of the E3 Ubiquitin Ligase Cereblon by Pomalidomide-based Homo-PROTACs

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Area of Science:

Hematology
Oncology
Rare Diseases

Background:

Mastocytosis is a rare, acquired disorder involving abnormal mast cell proliferation.
Current treatments offer limited long-term remission for advanced systemic mastocytosis (SM).

Observation:

Two patients with advanced SM received maximal tolerated doses of thalidomide.
Treatment led to rapid improvement in clinical symptoms and specific diagnostic findings (B and C findings).

Findings:

Both patients achieved partial remission lasting over one year.
Thalidomide demonstrated significant activity in advanced systemic mastocytosis.

Implications:

Thalidomide represents a potential therapeutic option for advanced SM.
Further clinical trials are necessary to confirm efficacy and safety.