羅倫佐的油

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本文內容為對於腎上腺腦白質失養症(adrenoleukodystrophy, ALD)治療上所採用的羅倫佐的油。同名電影請見羅倫佐的油 (電影)

羅倫佐的油(Lorenzo's oil)是三油酸甘油酯(glycerol trioleate)與三芥子酸甘油脂(glycerol trierucate)以4:1比例的混合物，兩種脂質分別是油酸(oleic acid)與芥子酸(erucic acid)的三酸甘油酯結合型。此混合物用在腎上腺腦白質失養症(adrenoleukodystrophy, ALD)的預防性治療上。此脂質藥劑是由義大利裔夫婦奧古斯都·歐東內與米凱拉·歐東內(Augusto and Michaela Odone)所合成，起因是他們的兒子羅倫佐於1984年被診斷患有ALD病症，時年五歲。奧古斯都·歐東內取得此油劑在美國的專利，編號5,331,009號。專利權所得用來支付髓鞘再生研究計劃(The Myelin Project)，此計劃目標在研究ALD與相似病症的治療方法。歐東內家族以及他們的發明因為1992年電影「羅倫佐的油」的拍攝而聲名大噪。

[编辑] 價格與保險給付

在美國，羅倫佐的油價格仍是相當高，一個月療程花費約美金$440。多數保險公司不給付此項花費，因為其被美國食品與藥物管理局(Food and Drug Administration, FDA)認為是實驗性治療。

[编辑] 作用機制

脂肪酸的混合劑可以降低導致ALD的極長鍊脂肪酸(very long chain fatty acids, VLCFA)在血中的濃度。其是透過對身體內合成VLCFA的酵素的競爭性抑制來達成這項目標。

[编辑] 效力

Lorenzo's oil has not been clinically proven to be effective against the progression of ALD. Of five published studies, four have failed to demonstrate benefit in symptomatic patients. It does, however, reduce the probability of developing symptoms if its administration is started before they begin. Its efficiency is lessened once symptoms start.

[编辑] 研究

In an early study, Duchesne studied eight males and reported improvements in follow-up magnetic resonance imaging of the brain.

In the only clinical study with positive findings, Moser tried Lorenzo's oil in 53 asymptomatic patients with ALD. Analysis of the effect of the oil indicated that there was only a slight but statistically significant slowing of clinical progression and delay of death.

However a controlled study by Moser concluded that Lorenzo's oil does not alter the course of the illness in symptomatic patients; however, dietary therapy started before the development of symptoms may reduce the frequency and severity of subsequent neurological disability.

A study by Poulos found that Lorenzo's oil is of limited value in correcting the accumulation of saturated VLCFAs in the brain of patients with ALD.

In the best study so far, Aubourg reported in the New England Journal of Medicine the results of an open trial of treatment with Lorenzo's oil in 14 men with adrenomyeloneuropathy, five symptomatic heterozygous women, and five boys (mean age, 13 years) with preclinical ALD. Over a mean follow-up of 33 months, none of the 14 men with ALD improved, one of the five asymptomatic boys developed signs of myelopathy, and there were no changes in the symptomatic heterozygous women. The authors concluded that this open trial found no evidence of a clinically relevant benefit from dietary treatment with Lorenzo's oil in patients with ALD.

[编辑] 現前狀況

In summary, although dietary manipulation using Lorenzo's oil has been shown to change blood test results, it appears to be ineffective for the symptomatic ALD although some parents of ALD patients feel otherwise. In addition, recent studies by Dr. Hugo Moser have found there is evidence that use of the oil by asymptomatic patients may delay the onset of symptoms significantly.

In the U.S., Lorenzo’s oil is currently only available to patients taking part in a clinical trial under the direction of Dr. Hugo Moser of the Kennedy Krieger Institute.