REVIEWS DDT • Volume 10, Number 1 • January 2005
Reviews • KEYNOTE REVIEW
23 www.drugdiscoverytoday.com
Lipocalins are a widespread family of small, robust
proteins that typically transport or store biological
compounds which are either of low solubility or are
chemically sensitive, including vitamins, steroid
hormones, odorants and various secondary
metabolites. There are approximately ten different
lipocalins in the human body, with the plasma retinol-
binding protein being the most well known. Some
lipocalins have a pathophysiological role, which opens
possibilities for their use in medical applications.
Furthermore, lipocalins from blood-sucking insects
have evolved as scavengers for mediators of
inflammation. As well as using the natural ligand-
binding function, lipocalins have also been recruited
as scaffolds for the design of artificial binding proteins
termed ‘anticalins’
®
. These novel proteins have
potential applications as antidotes, antagonistic
protein therapeutics or as target-recognition modules
in a new generation of immunotoxins.
The lipocalins represent a family of functionally diverse, small pro-
teins that comprise 160–180 amino acid residues and have weak se-
quence homology but high similarity at the tertiary structural level
[1,2]. Members of this family have important biological functions in
a variety of organisms, from bacteria to humans. The majority of
lipocalins are responsible for the storage and transport of compounds
that have low solubility or are chemically sensitive, such as vitamins,
steroids and metabolic products [3]. The human plasma retinol-binding
protein (RBP) was the first lipocalin for which a 3D structure was
elucidated [4,5]; RBP transports the poorly soluble and oxidation-prone
vitamin A from the liver, where it is stored as a fatty acid ester, to
several target tissues. Some lipocalins appear to have a more specialized
role in vertebrates [6], participating, for example, in olfaction and
Steffen Schlehuber
PIERIS Proteolab AG,
Freising-Weihenstephan,
Germany
Arne Skerra*
Lehrstuhl für Biologische
Chemie,
Technische Universität
München,
Freising-Weihenstephan,
Germany
*e-mail: skerra@wzw.tum.de
1359-6446/04/$ – see front matter ©2005 Elsevier Ltd. All rights reserved. PII: S1359-6446(04)03294-5
STEFFEN SCHLEHUBER
Steffen Schlehuber was
born in Fulda, Germany,
and studied chemistry at
the Technical University of
Darmstadt, where he
specialized in biochemistry.
He went on to complete a
doctoral thesis in the laboratory of Arne Skerra at
the Technical University of Munich,Germany,
obtaining his PhD in 2001. During his doctoral
study,Schlehuber was involved in the development
of anticalins, which are engineered ligand-binding
proteins derived from natural lipocalin proteins.
Steffen Schlehuber is cofounder and CSO of PIERIS
Proteolab AG, a biotechnology company situated
in Freising-Weihenstephan, Germany.Founded in
2001, PIERIS focuses on the development and
commercialization of anticalins for therapeutic
and diagnostic uses, predominantly in the area of
oncology and cardiovascular diseases.
ARNE SKERRA
Arne Skerra was born in
Wiesbaden, Germany, and
studied chemistry at the
Technical University of
Darmstadt. In 1989, he
received his PhD at the
Ludwig-Maximilians
University, Munich, where he had performed,
under the supervision of Andres Plückthun and
Ernst-Ludwig Winnacker, important research on
the bacterial expression of functional antibody
fragments. After spending one year as a postdoctoral
research fellow with Greg Winter and Cesar Milstein
at the MRC Laboratory of Molecular Biology in
Cambridge, UK, he joined the department of Hartmut
Michel at the Max-Planck-Institute of Biophysics in
Frankfurt am Main. In 1994,Skerra became Professor
of Protein Chemistry at the Technical University of
Darmstadt.Four years later he moved to the Technical
University of Munich, where he was appointed a
Full Professor to the Chair of Biological Chemistry
at the Life Science Campus,Weihenstephan. Skerra
is Chairman of the study group on protein engineer-
ing and design at the Society for Biochemistry and
Molecular Biology and a Board Member of the
biochemistry section of the Society of German
Chemists. In 2001,he cofounded the biotechnology
start-up company PIERIS Proteolab AG.
Lipocalins are promising drug candidates, either based on
their natural ligand-binding functions or as engineered
‘anticalins’ with novel specificities.
Keynote review:
Lipocalins in drug discovery:
from natural ligand-binding
proteins to ‘anticalins’
Steffen Schlehuber and Arne Skerra