Recruiting for Phase 2 - (RESTORE-1 Clinical Study)

[Farooqji]

Based on the positive results from both the PD-1101 and PD-1102 trials, researchers have launched the RESTORE-1 Phase 2 trial (NCT03562494). The trial, currently recruiting participants, will randomize patients with advanced Parkinson’s disease who have failed to respond properly to oral therapy to either optimized medical management plus VY-AADC01 or continued optimized medical management — including levodopa — plus placebo-surgery.

clinicaltrials.gov/ct2/show...

restore1study.com/Index.aspx

[park_bear]

"Dopamine is made in the brain when the enzyme AADC (Aromatic l-amino acid decarboxylase) converts the chemical levodopa to dopamine. Levodopa, AADC, and dopamine are each present at normal levels in healthy people....levodopa medication, which is converted into dopamine by the enzyme AADC in the same way that naturally occurring levodopa is converted to dopamine.

As Parkinson’s disease worsens, there is less AADC enzyme in parts of the brain where it is needed to convert levodopa to dopamine. Therefore, the amount of dopamine that is produced from each dose of levodopa medicine may be reduced. When this happens, patients’ motor function may worsen with a less predictable response to medications.

Voyager Therapeutics’ investigational gene therapy is designed to put the AADC enzyme into brain cells where it can convert levodopa to dopamine. To do this, the AADC gene is delivered inside a transporter called “adeno-associated viral vector” (AAV), much like a letter that carries the instructions the brain needs to make the AADC enzyme with the AAV as the envelope that carries the letter."

Requires brain surgery and half the patients will get placebo. Might be worth it if you have advanced PD.

[glenandgerry in reply to park_bear]

I thought Voyager had ditched this project?

[park_bear in reply to glenandgerry]

No they have not. The trial webpage is copyright 2019 and I doubt they would leave it up if the trial were canceled. Also this is the lead drug in the current pipeline:

voyagertherapeutics.com/our...

[Farooqji in reply to glenandgerry]

parkinsonsnewstoday.com/201...