TY - JOUR
T1 - Discovery and development of clofarabine
T2 - A nucleoside analogue for treating cancer
AU - Bonate, Peter L.
AU - Arthaud, Larry
AU - Cantrell, William R.
AU - Stephenson, Katherine
AU - Secrist, John A.
AU - Weitman, Steve
N1 - Funding Information:
Competing Interests Statement P.L.B., L.A., W.J.C. and K.S. are employees of Genzyme, the manufacturer of clofarabine. S.W. is a previous employee of Genzyme and current consultant to Genzyme. J.A.S. is an employee of Southern Research Institute, an organization that has received funding from Genzyme.
Copyright:
Copyright 2011 Elsevier B.V., All rights reserved.
PY - 2006/10
Y1 - 2006/10
N2 - The treatment of acute leukaemias, which are the most common paediatric cancers, has improved considerably in recent decades, with complete response rates approaching ∼90% in some cases. However, there remains a major need for treatments for patients who do not achieve or maintain complete remission, for whom the prognosis is very poor. In this article, we describe the challenges involved in the discovery and development of clofarabine, a second-generation nucleoside analogue that received accelerated approval from the US FDA at the end of 2004 for the treatment of paediatric patients 1-21 years old with relapsed or refractory acute lymphoblastic leukaemia after at least two prior regimens. It is the first such drug to be approved for paediatric leukaemia in more than a decade, and the first to receive approval for paediatric use before adult use.
AB - The treatment of acute leukaemias, which are the most common paediatric cancers, has improved considerably in recent decades, with complete response rates approaching ∼90% in some cases. However, there remains a major need for treatments for patients who do not achieve or maintain complete remission, for whom the prognosis is very poor. In this article, we describe the challenges involved in the discovery and development of clofarabine, a second-generation nucleoside analogue that received accelerated approval from the US FDA at the end of 2004 for the treatment of paediatric patients 1-21 years old with relapsed or refractory acute lymphoblastic leukaemia after at least two prior regimens. It is the first such drug to be approved for paediatric leukaemia in more than a decade, and the first to receive approval for paediatric use before adult use.
UR - http://www.scopus.com/inward/record.url?scp=33749263031&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=33749263031&partnerID=8YFLogxK
U2 - 10.1038/nrd2055
DO - 10.1038/nrd2055
M3 - Review article
C2 - 17016426
AN - SCOPUS:33749263031
VL - 5
SP - 855
EP - 863
JO - Nature Reviews Drug Discovery
JF - Nature Reviews Drug Discovery
SN - 1474-1776
IS - 10
ER -