GRF6021 plasma fraction

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

Alkahest, Inc. (a subsidiary of Grifols), with funding support from The Michael J. Fox Foundation. Sites were in the US, Australia and France (29 in all, per Alzforum).

What happened

GRF6021 is a plasma fraction: proteins pulled from donated plasma, with clotting factors and immunoglobulins (antibodies) removed. It rests on the "young blood" idea that factors in the blood of young animals can offset ageing and inflammation in the brain; Alkahest says it improved memory and reduced brain inflammation in animal models.

The trial (NCT03713957) enrolled people with Parkinson's and mild cognitive impairment or dementia (MoCA score 13 to 25). Of 79 randomised, 53 got GRF6021 and 26 got placebo, as five daily intravenous infusions (250 mg a day, per Alzforum) at week 1 and again at week 13. The main measure was safety. Registry results show at least one adverse event in 45 of 53 on the drug and 20 of 26 on placebo. Five on GRF6021 had a serious adverse event, against none on placebo; Alzforum says none was attributed to the drug. Headache and falls were more common on the drug (Alzforum). Fewer on the drug finished (35 of 53 versus 16 of 26).

Cognitive measures were secondary. The MoCA (a 30-point screening test) rose by 1.3 points on the drug and 0.8 on placebo at week 16. The motor-and-daily-living score (MDS-UPDRS) improved by 8.9 and 9.5 points. Some other tests moved either way. The registry gives no significance tests. A March 2021 company release called the effects on cognition "positive" but gave no numbers; Alzforum, summarising it, reports small improvements in MoCA and PDQ-39 from baseline on the drug and none on placebo.

Why it moved us toward a cure

It is the first human test of the plasma idea in Parkinson's and a test of the inflammation branch (landmark 52 found activated immune cells in the nigra in 1988). The trial was too small and too short to say anything about slowing the disease, and the outcome measures were symptoms.

Outcome

Open. The trial finished in July 2020. I found no peer-reviewed paper and no announced Phase 3.

Sources

• ClinicalTrials.gov NCT03713957 (API, results section, 19 Oct 2026; 79 enrolled, 35 of 53 and 16 of 26 completed)
• Alkahest/Grifols release, 8 Mar 2021
• Alzforum: GRF6021 · MJFF, Alkahest begins Phase II trial (funding; search summary)
• Villeda et al., Nature 2011, DOI 10.1038/nature10357 (the young-blood mouse work behind the idea)
• Not yet verified: any peer-reviewed publication; whether Alkahest or Grifols still develops GRF6021 for Parkinson's; the figure for significance of the cognitive changes; the exact composition of the product.