Abstract
L-Proline
is an important amino acid for the pathogenic protists belonging to Trypanosoma
and Leishmania genera. In Trypanosoma cruzi, the etiological
agent of Chagas disease, this amino acid is involved in fundamental biological
processes such as ATP production, differentiation of the insect and
intracellular stages, the host cell infection and the resistance to a variety
of stresses, including nutritional and osmotic as well as oxidative imbalance.
In this study, we explore the L-Proline uptake as a chemotherapeutic target for
T. cruzi. For this, we propose a novel rational to design inhibitors
containing this amino acid as a recognizable motif. This rational consists of
conjugating the amino acid (proline in this case) to a linker and a variable
region able to block the transporter. We obtained a series of sixteen 1,2,3-triazolyl-proline
derivatives through alkylation and copper(I)-catalyzed azide-alkyne cycloaddition
(click chemistry) for in vitro screening against T. cruzi epimastigotes,
trypanocidal activity and proline uptake. We successfully obtained inhibitors
that are able to interfere with the amino acid uptake, which validated the
first example of a rationally designed chemotherapeutic agent targeting a
metabolite's transport. Additionally, we designed and prepared fluorescent
analogues of the inhibitors that were successfully taken up by T. cruzi, allowing following up their intracellular
fate. In conclusion, we successfully designed and produced a series of
metabolite uptake inhibitors. This is one of few examples of rationally designed
amino acid transporter inhibitor, being the first case where the strategy is
applied on the development of chemotherapy against Chagas disease. This
unprecedented development is remarkable having in mind that only a small
percent of the metabolite transporters has been studied at the structural
and/or molecular level.