GC01 is a peptide with 24 amino acids, whose sequence is highly conserved between species and corresponds to the binding site of tubulin on the light subunit of neurofilaments.
It presents itself in the form of a β sheet in the extra-cellular matrix, and in the form of a α helix in the cytoplasm.
Being hydrophilic, GC01 is soluble in water, and is easily produced by solid phase chemical synthesis, and has good physical stability.
GC01 is innovative due to its preferential entry into gliomal cells (stem cells and differentiated cells), and due to its specificity of action on these cells alone.
This double characteristic is linked on the one hand to the plasticity of its three-dimensional conformation (β sheet to enter gliomal cells and helix α to bind to tubulin) and on the other hand to its binding site on the C-terminal part of the βIII subunit of tubulin (a β tubulin isotype which is overexpressed in cancerous gliomal cells). This allows GC01 to specifically inhibit gliomal cell proliferation without toxic effects on healthy cells and tissues.
GC01 has strong anti-tumor activity.
At a concentration of 5 μM and after a single intra-tumor administration, GC01 inhibited tumor development by 60% and significantly increased the survival of tumor-bearing animals (mice and rats).
Recent studies have also shown that intracerebral and intravenous administration do not induce toxic effects in healthy animals.