Deluca Hector F
Department of Biochemistry, University of Wisconsin-Madison , Madison, WI 53706-1544, USA.
Bonekey Rep. 2014 Mar 5;3:514. doi: 10.1038/bonekey.2014.9. eCollection 2014.
The goal of synthetic chemists in the vitamin D field has been to produce an analog(s) of 1α,25-dihydroxyvitamin D3 (1,25-(OH)2D3) that is selective for a specific function. The accumulation of structure/function information has led to the synthesis of two analogs that are both selective and more potent than 1,25-(OH)2D3, that is, 2-methylene-19-nor-(20S)-1α,25-dihydroxyvitamin D3 (2MD) and 2α-methyl-19-nor-(20S)-1α,25-dihydroxyvitamin D3 (2AMD). In vivo, the efficacy of 2MD is approximately equal to that of 1,25-(OH)2D3 in intestinal calcium transport but is 30- to 100-fold more active in bone mobilization. In vitro, 2MD supports new bone synthesis at 10(-12) M, whereas 1,25-(OH)2D3 is active at 10(-8) M. Similarly, 2MD is two orders of magnitude more potent than 1,25-(OH)2D3 in stimulating osteoclastogenesis and osteoclastic bone resorption. 2MD also markedly increases bone mass and bone strength of ovariectomized female rats. In postmenopausal women, 2MD significantly increases markers of both bone formation and resorption but has minimal effect on bone mass. Thus, in patients who are undergoing primarily remodeling rather than modeling (rat), the increased resorption largely counteracts the increased bone formation. So far, 2MD has not been tested for reduction of fractures in this population. However, its selectivity includes the parathyroid gland. Thus in the 5/6-nephrectomy model of chronic renal failure, 2MD is much more potent than currently available vitamin D compounds used to suppress secondary hyperparathyroidism of renal failure without causing hypercalcemia. It is currently in phase 2B trials in patients on dialysis.
维生素D领域的合成化学家们的目标是制备出对特定功能具有选择性的1α,25 - 二羟基维生素D3(1,25-(OH)2D3)类似物。结构/功能信息的积累促使合成了两种比1,25-(OH)2D3更具选择性且活性更强的类似物,即2 - 亚甲基 - 19 - 去甲 - (20S)-1α,25 - 二羟基维生素D3(2MD)和2α - 甲基 - 19 - 去甲 - (20S)-1α,25 - 二羟基维生素D3(2AMD)。在体内,2MD在肠道钙转运方面的功效与1,25-(OH)2D3大致相当,但在骨动员方面的活性要高30至100倍。在体外,2MD在10(-12) M时就能支持新骨合成,而1,25-(OH)2D3在10(-8) M时才有活性。同样,在刺激破骨细胞生成和破骨细胞性骨吸收方面,2MD的效力比1,25-(OH)2D3高两个数量级。2MD还能显著增加去卵巢雌性大鼠的骨量和骨强度。在绝经后女性中,2MD能显著增加骨形成和骨吸收的标志物,但对骨量的影响极小。因此,在主要经历重塑而非塑形的患者(大鼠)中,增加的骨吸收在很大程度上抵消了增加的骨形成。到目前为止,尚未对该人群中2MD减少骨折的效果进行测试。然而,其选择性包括甲状旁腺。因此,在慢性肾衰竭的5/6肾切除模型中,2MD比目前用于抑制肾衰竭继发性甲状旁腺功能亢进且不引起高钙血症的维生素D化合物效力要强得多。它目前正处于针对透析患者的2B期试验阶段。