Neuroprotection

Cerebrolysin (FPF-1070) is not a single peptide but a porcine-brain-derived preparation of low-molecular-weight peptides and free amino acids, produced by standardised enzymatic proteolysis. It is approved in several countries (including Austria, Russia and parts of Asia) for stroke, dementia and traumatic brain injury, but is not FDA-approved in the United States and not centrally approved by the EMA. Its efficacy is contested: Cochrane systematic reviews found no convincing benefit and flagged possible harm signals.

Elamipretide (SS-31, MTP-131, formerly Bendavia) is a synthetic, mitochondria-targeting tetrapeptide (sequence D-Arg-Dmt-Lys-Phe-NH2) that binds cardiolipin on the inner mitochondrial membrane and is proposed to stabilise cristae structure and support mitochondrial bioenergetics. It was investigated clinically by Stealth BioTherapeutics across several indications, including primary mitochondrial myopathy, Barth syndrome, heart failure, and dry age-related macular degeneration (geographic atrophy). The trial record is mixed, with several pivotal studies missing their primary endpoints. In September 2025 elamipretide (brand name Forzinity) received accelerated FDA approval in the United States solely for the ultra-rare Barth syndrome; for all other investigated indications it remains investigational and it is not approved as a medicine outside the United States.

Humanin is a 24-amino-acid mitochondrial-encoded peptide (mitochondrial-derived peptide, MDP) whose open reading frame lies within the 16S rRNA region (gene MT-RNR2) of mitochondrial DNA. It is considered the founding member of the MDP family and was discovered in 2001 by the Hashimoto/Nishimoto group while searching for neuroprotective factors in the brain of an Alzheimer's patient. In basic research (including the laboratory of Pinchas Cohen) humanin is described as a cytoprotective, anti-apoptotic peptide and is studied in the contexts of Alzheimer's/neuroprotection, metabolism/insulin action and aging. The evidence comes almost entirely from cell and animal models and from observations of endogenous levels in humans; controlled human trials of exogenous humanin as a therapeutic are lacking. It is not approved as a medicine anywhere and is traded on the grey market as a research chemical.

Synthetic heptapeptide derived from the N-terminal fragment of adrenocorticotropic hormone (ACTH 4-10). Approved in Russia for ischaemic stroke, cognitive function and ADHD in children. Western phase-3 trials absent.