Clinical trial suggests Parkinson's drug is safe in humans

[Abdiqani]

A new study finds an experimental drug that is significant for PD treatment " A large team of researchers from Denali Therapeutics, working with colleagues from multiple entities in the U.S. and one in Canada, has found that a LRRK2 inhibitor called DNL201 showed no ill effects to volunteers in a clinical trial. The researchers found that administration of the drug brought down levels of LRRK2 enzymes and also showed no signs of the side-effects that had been seen in some other animals." Study Notes .

More info :

medicalxpress.com/news/2022...

[jeffreyn]

See also this recent AlzForum article.

LRRK2 Inhibitor Hits Target, Appears Safe for Parkinson’s

alzforum.org/news/research-...

[Gymsack in reply to jeffreyn]

I read it a couple times and remain confusedPathogenic mutations in the LRRK2 gene . Did volunteers without mutation in this gene also show improvement ? How does one determine if they have such a mutation ? You23and me , I suppose.

[jeffreyn in reply to Gymsack]

The Phase 1 trial of DNL201 did not check for efficacy. Efficacy is being tested for in the ongoing Phase 2 trial (of DNL151).

It seems to me that 23andme would be a reasonable place to get a DNA test.

[Missy0202 in reply to Gymsack]

I was tested through the MJFF in conjunction with the University of Indiana as well as through my movement disorders clinic. Alternatively 23andme shows that variant

[Missy0202]

Very encouraging. Thanks for posting!

[jeffmayer]

Should we get excited or not

[jeffreyn in reply to jeffmayer]

The Phase 2 trial of DNL151 is a pretty big one (n=640). It is not due to finish until 2025.

I would call it interesting, rather than exciting.

[jeffmayer in reply to jeffreyn]

Thanks looks like I will have to do dbs

[HekateMoon]

Thank you Abdiqani. I wonder how long till it will become available...takes them ages

[goldengrove]

The molecule specifically targets the enzyme overactivity caused by the LRRK2 mutation but it turns out that LRRK2 is overactive as well in idiopathic PD patients who don't have the mutation so it's hoped that the molecule will be broadly useful. I'm excited.

[jeffreyn]

A new study record has been posted on the Clinical Trials website.

Title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Efficacy and Safety of BIIB122/DNL151 in Participants With Parkinson's Disease and Pathogenic LRRK2 Variants

Estimated Study Start Date: July 2022

Estimated Study Completion Date: January 2031

clinicaltrials.gov/ct2/show...