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21 Biotechnology L. Rep. 1 (2002)

handle is hein.journals/bothnl21 and id is 1 raw text is: 21 Biotechnology Law Report 1 Number 1 (February 2002) © Mary Ann Liebert, Inc. Proposed NIH Guidelines as Front-End Incentives for Orphan Drug Development Involving Bayh-Dole Licensing Agreements JAYSHREE AIYAR GERKEN, Ph.D.* INTRODUCTION BOB1 HAS POLYCYSTIC KIDNEY DISEASE (PKD).2 PKD is a life-threatening genetic disorder af- fecting about 600,000 Americans and characterized by the growth of numerous fluid-filled cysts in the kidneys that may gradually bring function to a halt, resulting in renal failure.3 To date, there is no cure for PKD; Bob's options for mitigating the harsh PKD symptoms include medication and surgery to ease the pain, antihypertensive drugs to ameliorate the high blood pressure, antibiotics to clear infec- tions that the kidneys are susceptible to, and, if he is one of more than 60% of patients in whom the disease progresses to renal failure, dialysis and transplantation to substitute for failed kidney func- tion.4 Aside from the lifelong pain and suffering that Bob might have to endure, there is the expense to the taxpayers of more than $1 billion in treatment costs for each patient like Bob, particularly in the event that the patient experiences kidney failure.5 A glimmer of hope for sufferers like Bob is the progress in PKD research during the past few years: progress that could result in a cure. In the years since 1985, several genes found to be defective in various forms of PKD have been mapped to specific chro- mosomal locations on human DNA, and, in some in- stances, the genes and their products have been iso- lated and identified.6 In addition, research to understand the function of these genes in the etiology of PKD has led to a genetically altered mouse that de- velops PKD in a manner that is similar to one form of the human disease.7 Other researchers have stud- ied the relatively simple organism, the roundworm C. *Dr. Gerken is a third-year student at California Western School of Law. elegans, and determined the function of C. elegans genes that have a high degree of homology to the hu- man PKD genes.8 1 Fictionalized account of the actual progression of polycystic kidney disease. 2 See generally Joel R. Cooper, Polycystic kidney disease: a challenging illness affecting hundreds of thousands. Medical Reporter June 1, 1995; available at http://medicalreporter. health.orgAmr0795/polycyst0795.html. 3 Polycystic Kidney Research Foundation (last visited Novem- ber 14, 2000) http://www.pkdcure.org. 4 Id. 5 Id. 6 See generally M. Koptides and C.C. Deltas: Autosomal dom- inant polycystic kidney disease: molecular genetics and mo- lecular pathogenesis. Hum Genet 2000;107:115. See also Stephen Reeders et al., Identification of Polycystic Kidney Dis- ease Gene, Diagnostics and Treatment, U.S. Patent No. 5,891,628 (April 6, 1999), E. Rondeau: Polycystic kidney: com- plete structure of the PKD1 gene and its protein. Nephrologie 1995;16:338; T. Mochizuki et al.: PKD2, a gene for polycys- tic kidney disease that encodes an integral membrane protein. Science 1996;272:1339;M.C. Daoust et al.: Evidence for a third genetic locus for autosomal dominant polycystic kidney dis- ease. Genomics 1995;25:733; S.T. Reeders et al.: A highly polymorphicDNA marker linked to adult polycystickidney dis- ease on chromosome 16. Nature 1985;317:542; W.J. Kimber- ling et al.: Autosomal dominant polycystic kidney disease: lo- calization of the second gene to chromosome 4q13-q23. Genomics 1993;18:467; K. Zerres et al.: Mapping of the gene for autosomal recessive polycystic kidney disease (ARPKD) to chromosome 6p21-cen. Nat. Genet. 1994;7:429. 7 G. Wu et al.: Somatic inactivation of Pkd2 results in poly- cystic kidney disease. Cell 1998;93:177. 8 Maureen M. Barr and Paul W. Sternberg: A polycystic kid- ney-disease gene homologue required for male mating behav- ior in C. elegans. Nature 1999;401:386. The term homology refers to the degree of identity between the DNA sequences of different genes from the same organism or genes from one more than one organism. In tis case, determining the function of genes in the roundworm that are very similar (homologous') to the human PKD genes would lead to an understanding of the role the PKD gene products play in the etiology of the disease, and the roundworm would also serve as a tool to screen for drugs that treat PKD by inhibiting the function or expression of the PKD gene products. 1

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