Experimental Treatments That Target Tau Protein

Alzheimer’s disease is characterized by the protein plaques and tangles that are thought to be responsible for cell death and tissue loss in the brain. Tangles are twisted fibers of a protein called tau, which normally plays a role transporting nutrients through nerve cells. The tangled tau proteins cause the transport to be disrupted, leading to cells not getting enough nutrients and eventually dying.

There are several experimental compounds being developed that target the tau protein tangles in Alzheimer’s disease. Some of these are listed below. More information is available about each compound by clicking on its name.


AADvac1 is an active vaccine being developed by Axon Neuroscience. It is a synthetic peptide derived from tau protein. It is designed to elicit an immune response against pathologically modified forms of tau protein. It is being investigated in a Phase 1 pilot study (NCT03174886) in Germany.


ACI-35 is a liposome-based vaccine licensed to Janssen. It is designed to elicit an immune response against certain pathological forms of phosphorylated tau protein without also mounting autoimmune B-cell or T-cell responses.


LMTM, a stable reduced form of methylthioninium, is an investigational treatment being developed by TauRx Pharmaceuticals. The molecule is designed to inhibit tau protein aggregation and dissolve existing aggregates.


LY3303560 is a humanized antibody being developed by Eli Lilly and Company. It binds and neutralizes tau aggregates.


Nicotinamide, or vitamin B3, found in food and also used as a dietary supplement, is being investigated in a Phase 2 clinical trial (NCT03061474) in California as an early Alzheimer’s disease treatment.  Preclinical studies demonstrated that nicotinamide can reduce tau protein phosphorylation, an event associated with the development of Alzheimer’s disease.

RO 7105705

RO 7105705 is a monoclonal antibody, being developed by Genentech in collaboration with AC Immune. It specifically targets pathological tau protein and is designed to prevent its cell-to-cell spread in the extracellular space of the brain. It is being investigated in a Phase 2 clinical trial across the U.S., Australia, and Canada.

TPI 287

TPI 287 is a treatment being developed by Cortice Biosciences. It stabilizes the assembly of microtubules, structures that form the skeleton of the cell. The treatment can readily penetrate the blood-brain barrier and has been shown to improve nerve cell survival in laboratory studies by stabilizing disrupted microtubules and the associated tau protein.


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