Therapeutics

GRF6019

Overview

Name: GRF6019
Therapy Type: Other
Target Type: Inflammation (timeline), Other (timeline), Unknown
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Phase 2)
Company: Alkahest, Inc., Grifols Biologicals Inc.

Background

GRF6019 is a proprietary plasma fraction of about 400 proteins, developed by Alkahest and manufactured by Grifols. It represents a second-generation product of earlier clinical research infusing young adult plasma to treat age-related neurodegenerative disease (see Young Plasma). This treatment approach grows out of mouse parabiosis studies indicating a rejuvenating effect of young blood on the aging brain (e.g. Nov 2009 newsMay 2014 conference news). The rationale behind GRF6019 is that the components of young blood that counteract inflammatory and other age-related degenerative processes in the brain can be enriched and delivered as a plasma-derived product that does not require matching of donor and recipient blood types.

In February 2019, responding to reports of commercial delivery of young plasma as a purported treatment for various conditions including memory loss, the FDA issued a statement warning the public of the potential risks of this practice. GRF6019 is a plasma fraction from which clotting factors and immunoglobulins have been removed to reduce those risks. 

Findings

In April 2018, Alkahest started a Phase 2 trial in 40 people with clinical diagnoses of mild to moderate AD; biomarkers to confirm underlying molecular pathology were not required. The study compared two doses of 100 ml or 250 ml GRF6019, given as five once-daily intravenous infusions in week one, and again in week 13, primarily on safety measures. Secondary outcomes include six-month change on some widely used cognitive and clinical batteries—MMSE, ADASCog11, CDR-SB, ADCS-ADL, ADCS Clinical Global Impression of Change, NPI Questionnaire—as well as on some individual tests, e.g., the Grooved Pegboard and Category Fluency tests. The trial ran at 10 U.S. sites, and finished in May 2019. In August, top-line data were announced. According to the company press release, GRF6019 was safe and well-tolerated, participants showed no decline on the ADAS-Cog11 or MMSE, and showed "negligible decline" on the CDR-SB and ADCS-ADL over six months. There was no placebo group for comparison.

In January 2019, Alkahest began a placebo-controlled Phase 2 trial in 20 people with clinically diagnosed AD and advanced dementia, i.e., an MMSE score below 10. Participants receive infusions of 250 ml GRF6019 or placebo daily for five days, followed by a month of observation. Primary outcomes will assess safety and tolerability; secondary outcomes include change in MMSE, ADCS-ADL, and other measures of cognitive impairment and neuropsychiatric symptoms. The study, at six U.S. locations, is set to end in March, 2020.

In February 2018, Alkahest received a grant from the Michael J Fox Foundation to support development of a second plasma fraction, GRF6021, for Parkinson's disease with cognitive impairment.

For all clinical trials of GRF6016, see clinicaltrials.gov.

Clinical Trial Timeline

  • Phase 2
  • Study completed / Planned end date
  • Planned end date unavailable
  • Study aborted
Sponsor Clinical Trial 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029
Alkahest, Inc. NCT03520998
N=40

Last Updated: 28 Nov 2019

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References

Therapeutics Citations

  1. Young Plasma

News Citations

  1. Chicago: The Vampire Principle—Young Blood Rejuvenates Aging Brain?
  2. In Revival of Parabiosis, Young Blood Rejuvenates Aging Microglia, Cognition

External Citations

  1. company press release
  2. clinicaltrials.gov
  3. statement

Further Reading