Dear Alexander,

I try find drug name with trehalose, I found only 'Cabaletta'. But when I was checking approved drugs in FDA database cant find it. For now I not sure its approved by FDA.

FDA Approved Drug Products:

https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=browseByLetter.page&productLetter=C&ai=0

In clinicaltrials.gov I found only 3 studies related to Cabaletta (2 phase 2 and one from phase 3) and some other by Bioblast Pharma Ltd. with trehalose.

https://clinicaltrials.gov/ct2/results?cond=&term=cabaletta&cntry=&state=&city=&dist=

I not sure that trehalose is approved as a 'drug product'? If its not approved and not present in the market then data from every day practice (not clinical trials) could be not available.

Best regards,

Tomasz

The FDA commented on this compound - https://www.fda.gov/media/113175/download

It concluded-

Based on the information that DuPont provided, as well as other information available to FDA, we have no questions at this time regarding DuPont’s conclusion that trehelase enzyme preparation produced by T. reesei expressing a trehalase gene from T. reesei is GRAS under its intended conditions of use. This letter is not an affirmation that trehelase enzyme preparation produced by T. reesei expressing a trehalase gene from T. reesei is GRAS under 21 CFR 170.35. Unless noted above, our review did not address other provisions of the FD&C Act. Food ingredient manufacturers and food producers are responsible for ensuring that marketed products are safe and compliant with all applicable legal and regulatory requirements.

In accordance with 21 CFR 170.275(b)(2), the text of this letter responding to GRN 000727 is accessible to the public at www.fda.gov/grasnoticeinventory.

Trehalose is of particular interest having been shown to reduce protein aggregates in models of other neurodegenerative diseases (Tanaka et al., 2004; Davies et al., 2006). Trehalose is a naturally occurring disaccharide, which appears to play an important role in stress responses in yeast (Singer and Lindquist, 1998). Whilst it has been suggested that its ability to reduce protein aggregation occurs due to a chaperone activity, or through binding and stabilization of abnormal proteins (Tanaka et al., 2004; Davies et al., 2006), it has also been shown to act via an mTOR-independent pathway to increase autophagy (Sarkar et al., 2007; Rodríguez-Navarro et al., 2010). These studies have clearly prompted interest in this compound, though a recent study of mouse primary cortical neurons found that trehalose did not prevent toxicity from exposure to α-synuclein pre-formed fibrils (Redmann et al., 2017).

Mini Review ARTICLE Front. Neurosci., 08 October 2018 | https://doi.org/10.3389/fnins.2018.00693 Emerging Treatment Approaches for Parkinson’s Disease Thomas B. Stoker1,2,3*†, Kelli M. Torsney1,4† and Roger A. Barker1,2,3