ALD Case Study
There are three main treatments for ALD all of which are most effective in the early stages of the disease. One of the treatments is directed at the involvement of the adrenal glands and their subsequent destruction and the resultant Addison’s disease. The treatment involves replacement of the hormones Cortisol and Aldosterone. This helps mitigate the primary adrenal insufficiency caused by the lack of these two hormones. Another treatment, which gained recognition via. the 1992 movie Lorenzo’s Oil, is the use of a 4:1 mix of oleic acid and erucic acid. This, in combination with a low fat diet results in a lowering of VLCFA with few side effects, mainly bruising and bleeding (Web MD http://www.webmd.com/vitamins-supplements/ingredientmono-255-lorenzo's%20oil.aspx?activeingredientid=255&activeingredientname=lorenzo%27s%20oil
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(Moser et al. 1987; Rizzo et al. 1989) ADL Database http://www.x-ald.nl/treatment-options/lorenzos-oil/). The final and most effective treatment is allogenic blood and marrow transplant (BMT). BMT is often taken from a family member or from cord blood. This therapy is most effective in the early stages of childhood ALD prior to any cerebral involvement. The mechanism involved with BMT itself is not well understood (University of Minnesota https://bmt.umn.edu/new-advancements-ald). A 2011 study done at the University of Minnesota estimated the 5 year survival rate after BMT to be 75% ,a much better prognosis than other two treatments (Miller et al. 2011) ALD Database http://www.x-ald.nl/treatment-options/hsct/). After over four decades of targeted research the treatments available only remain palliative at best. The newest and most promising treatment on the horizon is gene therapy. The 2016 Starbeam Study delivered the gene therapy drug Lenti-D originally to 17 boys, the study was later expanded to 25. This study is designated as a two-year study and then these same patients will be enrolled in a 13 year study to determine the long term affects. Preliminary finding are encouraging and this is an exciting area of hopeful development ( Gene Therapy for
(Moser et al. 1987; Rizzo et al. 1989) ADL Database http://www.x-ald.nl/treatment-options/lorenzos-oil/). The final and most effective treatment is allogenic blood and marrow transplant (BMT). BMT is often taken from a family member or from cord blood. This therapy is most effective in the early stages of childhood ALD prior to any cerebral involvement. The mechanism involved with BMT itself is not well understood (University of Minnesota https://bmt.umn.edu/new-advancements-ald). A 2011 study done at the University of Minnesota estimated the 5 year survival rate after BMT to be 75% ,a much better prognosis than other two treatments (Miller et al. 2011) ALD Database http://www.x-ald.nl/treatment-options/hsct/). After over four decades of targeted research the treatments available only remain palliative at best. The newest and most promising treatment on the horizon is gene therapy. The 2016 Starbeam Study delivered the gene therapy drug Lenti-D originally to 17 boys, the study was later expanded to 25. This study is designated as a two-year study and then these same patients will be enrolled in a 13 year study to determine the long term affects. Preliminary finding are encouraging and this is an exciting area of hopeful development ( Gene Therapy for