Landmark 251 · Trial in people
PDGF-BB into the ventricles
A safety test in 12 people for 12 days. It was well tolerated and symptom scores did not change. It was too small to show any slowing.
In short
A second growth factor, PDGF-BB, was infused for 12 days into the ventricles of 12 people with Parkinson's; it was well tolerated, symptom scores did not change, and a dopamine-transporter scan hinted at a rise in one area.
- What it is
- Trial in people
- Outcome
- Mixed or early results
- When
- 2009–2015
- Size
- 12 people · Phase 1
- Approach
- Replace or repair what's lost
- Research lane
- Growth factors
- Trial registry
- NCT00866502
- Last checked
- 19 Oct 2026
The research record
This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".
Who
Gesine Paul, Olof Zachrisson, A. Varrone and colleagues; sponsor Newron Sweden AB (formerly NeuroNova), two Swedish centres, funded by Newron and the Swedish agency VINNOVA. Journal of Clinical Investigation 125(3):1339–1346, March 2015. Registry NCT00866502.
What happened
Platelet-derived growth factor BB (PDGF-BB) is a protein that, in animals, helped dopamine neurons survive and recover. Like GDNF (landmark 213), it follows the growth-factor strategy: give the brain a protein that keeps dopamine neurons alive.
- Design. Phase 1/2a, randomised, double-blind, placebo-controlled (March 2009 to March 2011). 12 people with moderate Parkinson's. A pump and catheter delivered the drug into the cerebral ventricles (fluid spaces), not into brain tissue. Doses 0.2, 1.5 or 5 µg a day for 12 days; 3:1 drug to placebo; 85 days of follow-up.
- Safety. All 12 finished. Three had serious adverse events, none judged related to the drug.
- Effect. Clinical rating scales showed no dose-dependent change. Dopamine-transporter binding rose in the right putamen of treated people, a secondary and unconfirmed finding.
- Follow-up. A long-term safety follow-up (NCT01807338) enrolled 11 people, August 2011 to August 2014. A higher-dose study in people who had previously had placebo or sNN0031 (NCT01829867, Phase 1, started April 2013) enrolled 5 and was terminated in October 2015. A repeat-dosing study (NCT02408562) was stopped in 2015 after one participant. The registry gives the reason for both: problems developing and supplying the infusion system, and a prolonged approval process.
Why it moved us toward a cure
It extended the growth-factor idea to a new protein and a new delivery route that avoids the brain tissue injections used for GDNF. It was a safety test only, 12 people for 12 days, so it could not show slowing.
Outcome
Open, but quiet. No later PDGF-BB Parkinson's trial was found in a ClinicalTrials.gov search (19 Oct 2026), and the repeat-dosing and dose-step studies ended early for device and approval reasons, not for lack of effect.
Sources
- Paul et al., J Clin Invest 2015, DOI 10.1172/JCI79635, PMID 25689258 (abstract read via Europe PMC)
- ClinicalTrials.gov NCT00866502, NCT01807338, NCT01829867, NCT02408562 (API, 19 Oct 2026)
- Not yet verified: whether the DAT binding was PET or SPECT; the full text (the abstract gives only a "positive effect" in the right putamen); Newron's later plans for sNN0031; the size of the DAT change.
How it connects
Built on
- builds on GDNF discovered · 1993
Led to
None recorded.