APDA overview of some of the PD disease-modifying therapies currently being investigated in clinical trials

[jeffreyn]

Categories covered:

- Alpha-synuclein strategies – decreasing aggregation of alpha-synuclein;

--- Alpha-synuclein antibodies (passive immunity);

--- Alpha-synuclein vaccine (active immunity);

--- Molecules that block alpha-synuclein;

- Glucagon-like peptide-1 (GLP-1) agonist strategies – decreasing neuro-inflammation;

- Glucocerebrosidase (GBA) – enhancing the cell’s lysosomal system;

- LRRK2 strategies;

- c-Abl kinase strategies – decreasing oxidative stress.

apdaparkinson.org/article/f...

NB: The APDA article is based on a recent review published in the Journal of Parkinson’s Disease.

content.iospress.com/downlo...

[MBAnderson]

do you have a favorite?

[jeffreyn in reply to MBAnderson]

Yes. It's in the LRRK2 category:

- BIIB094 is a LRRK2 antisense oligonucleotide (ASO), a molecule that binds the messenger ribonucleic acid (mRNA) of LRRK2 and inhibits its translation into a protein – NCT03976349.

The reason it's my favourite has nothing to do with LRRK2. If the clinical trial is successful, it should clear the way for ASOs directly targeting a-syn, as described in a recent paper [1].

[1] Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson's disease-like mouse model and in monkeys, Diana Alarcon-Aris et al., EBioMedicine 59 (2020).

thelancet.com/journals/ebio...

[MBAnderson in reply to jeffreyn]

Are you in that trial and if not are you outside the qualification criteria or did you choose not to get in?

[jeffreyn in reply to MBAnderson]

If Australia was included in the list for the LRRK2 trial, I'd be happy to sign up.

clinicaltrials.gov/ct2/show...

[MBAnderson in reply to jeffreyn]

Too bad. Good article.

(I'm probably wrong, but I thought W.T. Poo is in the BIIB054 trial.)

[jeffreyn in reply to MBAnderson]

Correct! But that's BIIB054, not BIIB094 (and he's in France).