Can Gaucher Drugs Treat COVID-19? The Medical Advisory Board Responds

Published: 6/2/2020; Updated: 1/12/21

The National Gaucher Foundation requested and received feedback from the Medical Advisory Board (MAB) regarding the recent ubiquitous press releases concerning the Israel Institute for Biological Research (IIBR) pre-peer reviewed paper. The IIBR’s study, run by Israel’s Defense Ministry, explores the possible use of analogues of eliglustat (brand name Cerdelga) and venglustat, a 3rd gen substrate inhibitor currently in clinical trials for Gaucher disease type 1 and 3, for either treatment and/or prevention of COVID-19 viral illness.

3 Key Points Our Experts Clarify About The IIBR’s Findings

  • Researchers who conducted the study tested analogues, NOT eliglustat (Cerdelga) nor venglustat. An analogue is a similar, but not identical molecule.
  • The study on the SARS-CoV-2 was only done IN-VITRO, meaning within cells outside of a living organism, not with mice, mammals, or humans.
  • There is no information regarding the potential toxicity of the doses that might be required to exert an anti-viral effect of these analogues in humans.

The findings of this study, therefore, while certainly interesting, do not remotely prove that eliglustat (brand name Cerdelga) and venglustat can be used to prevent or treat the novel coronavirus in human beings.

Main Take-Aways:

  1. Gaucher patients should continue their current treatment regimens.
  2. People who are doing well on ERT by no means should switch to Cerdelga concluding that doing so would protect against COVID-19. Any switch in treatment therapy should be discussed with your Gaucher specialist.

Prevalence of COVID-19 in the Gaucher Community

As a side note, our MAB has reported VERY FEW cases of COVID-19 among their patients with Gaucher disease. At the beginning of the pandemic, it was thought that Gaucher disease might increase the likelihood of a more severe presentation of the virus should one become infected. This has NOT materialized. The few Gaucher patients that indeed developed more severe COVID-19 symptoms ALL had other co-morbidities known to increase COVID-19 symptom severity, i.e. obesity, hypertension, cardiac conditions, and diabetes mellitus. To date, the globally accessible numbers are considerably less than what was expected. This leads to many interesting lines of inquiry, but NO practical changes in treatment regimens.

Additional Coronavirus Resources for Gaucher Patients

 

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