Winners of the 2009 Outstanding Achievement and Young Investigators Awards
The Outstanding Achievement Award
Marina Cavazzana-Calvo
Prof. Cavazzana-Calvo has made outstanding and seminal contributions
to the field of gene therapy that had a tremendous impact on the
field. The first successful gene therapy trial ever was realised
thanks to the efforts of Prof. Cavazzana-Calvo (Cavazzana-Calvo,
Science. 2000 Apr 28;288(5466):669-72). She targeted primary immune
deficiencies (particularly SCID-X1) by gene therapy using genetically
modified stem cells, which served as a trailblazer for the entire
field. She continued to be extremely productive in the field of gene
therapy and published many papers in the highest impact factor
journals (Science, NEJM, Cell, Nature Genetics, JCI, Immunity, Blood
etc.).
She and her team and collaborators were also playing a key role
in understanding the mechanisms that contributed to the
leukemiogenesis following insertional mutagenesis (Hacein-Bay-Abina et
al., J Clin Invest. 2008 Sep;118(9):3132-42.; Deichmann et al., J Clin
Invest. 2007 Aug;117(8):2225-32, Hacein-Bay-Abina et al Science. 2003
Oct 17;302(5644):415-9., Hacein-Bay-Abina et al N Engl J Med. 2003 Jan
16;348(3):255-6.) Her research contributed to a better understanding
of the side-effect of retroviral gene transfer and prompted a new area
of research to reduce the risk of insertional oncogenesis using
improved vectors. She is also taking the lead in moving forward with
lentiviral vectors in clinical trials (e.g. ALD) that is yielding
promising new results. Her studies provided novel mechanistic
insights into the molecular etiology of cancer. Prof. Cavazzana-Calvo
also made seminal contributions to the understanding of the genetic
causes of primary immune deficiencies and hematopoietic defects
leading to numerous high-impact factor publications (e.g.
Lagresle-Peyrou, Nat Genet. 2009 Jan;41(1):106-11). Her papers are
among the most highly cited in the field of gene and cell therapy.
The Young Investigator Award
Dr Alessandra Biffi
Alessandra's career as a young scientist clearly
demonstrates an outstanding capacity for productive research. Her
contribution to the development of gene therapy approaches based on
hematopoietic stem cells (HSC) and lentiviral vectors has opened the
way to novel therapeutic opportunities for treating lysosomal storage
disorders (LSD) with CNS involvement. Indeed, Alessandra and
colleagues have provided the first experimental evidence that
lysosomal enzyme over-expression mediated by lentiviral gene transfer
confers a unique therapeutic advantage to HSC transplantation for
treating the CNS manifestations of LSD (Biffi et al., J. Clin. Inv.
2004; Biffi et al., J. Clin. Inv. 2006). This seminal work conducted
in preclinical models has provided the scientific rationale for a
forthcoming trial of HSC gene therapy in patients affected by
metachromatic leukodystrophy (MLD), expected to launch at our
Institute in the first trimester of 2010.
Dr Kerry Fisher
