Mutations Linked to Autosomal Dominant Nocturnal Frontal Lobe Epilepsy Affect Allosteric Ca 2+ Activation of the 42 Nicotinic Acetylcholine Receptor S Nivalda O. Rodrigues-Pinguet, Thierry J. Pinguet, Antonio Figl, Henry A. Lester, and Bruce N. Cohen Division of Biology, California Institute of Technology, Pasadena, California (N.O.R.-P., H.A.L., B.N.C.); Luxtera, Inc., Carlsbad, California (T.J.P.); and Molecular Devices Corp., Union City, California (A.F.) Received January 18, 2005; accepted May 17, 2005 ABSTRACT Extracellular Ca 2+ robustly potentiates the acetylcholine re- sponse of 42 nicotinic receptors. Rat orthologs of five mutations linked to autosomal dominant nocturnal frontal lobe epilepsy (ADNFLE)—4(S252F), 4(S256L), 4(+L264), 2(V262L), and 2(V262M)—reduced 2 mM Ca 2+ potentiation of the 42 1 mM acetylcholine response by 55 to 74%. To determine whether altered allosteric Ca 2+ activation or en- hanced Ca 2+ block caused this reduction, we coexpressed the rat ADNFLE mutations with an 4 N-terminal mutation, 4(E180Q), that abolished 42 allosteric Ca 2+ activation. In each case, Ca 2+ inhibition of the double mutants was less than that expected from a Ca 2+ blocking mechanism. In fact, the effects of Ca 2+ on the ADNFLE mutations near the intracellular end of the M2 region—4(S252F) and 4(S256L)—were con- sistent with a straightforward allosteric mechanism. In contrast, the effects of Ca 2+ on the ADNFLE mutations near the extra- cellular end of the M2 region—4(+L264)2, 2(V262L), and 2(V262M)—were consistent with a mixed mechanism involv- ing both altered allosteric activation and enhanced block. How- ever, the effects of 2 mM Ca 2+ on the 42, 4(+L264)2, and 42(V262L) single-channel conductances, the effects of membrane potential on the 2(V262L)-mediated reduction in Ca 2+ potentiation, and the effects of eliminating the negative charges in the extracellular ring on this reduction failed to provide any direct evidence of mutant-enhanced Ca 2+ block. Moreover, analyses of the 42, 4(S256L), and 4(+L264) Ca 2+ concentration-potentiation relations suggested that the ADNFLE mutations reduce Ca 2+ potentiation of the 42 ace- tylcholine response by altering allosteric activation rather than by enhancing block. ADNFLE is a monogenic partial epilepsy linked to four 4 and two 2 nicotinic subunit mutations (Steinlein et al., 1995, 1997; Hirose et al., 1999; De Fusco et al., 2000; Phillips et al., 2001; Leniger et al., 2003). ADNFLE seizures occur primarily during slow-wave sleep and seem to originate in the frontal lobe (Scheffer et al., 1995). However, the physio- logical mechanism that generates these seizures has not been established. At physiological concentrations (1.5–2 mM), Ca 2+ potenti- ates the neuronal nicotinic agonist response by binding to an N-terminal site in the receptor protein (Galzi et al., 1996; Le Novere et al., 2002; Rodrigues-Pinguet et al., 2003). Alloste- ric Ca 2+ activation of the neuronal nicotinic receptors is conserved across species (rat, human, chicken) and across nicotinic receptor subtypes (Mulle et al., 1992; Vernino et al., 1992; Galzi et al., 1996; Steinlein et al., 1997; Liu and Berg, 1999). Adding 2 to 2.5 mM Ca 2+ to the extracellular saline increases the wild-type 42 acetylcholine response by 300 to 500% (Steinlein et al., 1997; Figl et al., 1998; Rodrigues- Pinguet et al., 2003). A common feature of the ADNFLE mutations is that they reduce 2 to 2.5 mM Ca 2+ potentiation of the 42 acetylcholine response by 50 to 72%, at acetyl- choline concentrations 30 M (Steinlein et al., 1997; Figl et al., 1998; Rodrigues-Pinguet et al., 2003). Because the extra- cellular Ca 2+ concentration in the mammalian brain is nor- mally 1.5 to 2 mM (Egelman and Montague, 1999), a decrease in Ca 2+ potentiation of the 42 acetylcholine response could contribute to ADNFLE seizures either by (1) reducing 42- This work was supported by grants from the National Institute of Health (NS0438000 and NS011756, to B.N.C. and H.A.L., respectively and a minority supplement (NS0438000-04S1 to N.O.R.). Portions of this work were published previously by UMI publishers in a doctoral dissertation by N.O.R. Article, publication date, and citation information can be found at http://molpharm.aspetjournals.org. doi:10.1124/mol.105.011155. S The online version of this article (available at http://molpharm. aspetjournals.org) contains supplemental material. ABBREVIATIONS: ADNFLE, autosomal dominant nocturnal frontal lobe epilepsy; [Ca 2+ ] o , added extracellular Ca 2+ concentration; i s , single- channel current; g s , single-channel conductance; SSD, percentage of steady-state desensitization; D , time constant of desensitization. 0026-895X/05/6802-487–501$20.00 MOLECULAR PHARMACOLOGY Vol. 68, No. 2 Copyright © 2005 The American Society for Pharmacology and Experimental Therapeutics 11155/3044683 Mol Pharmacol 68:487–501, 2005 Printed in U.S.A. 487