Alzheimer's Disease: A Case Study

lzheimer's is a chronic disease caused by a progressive loss of structure or function of neurons. The most common symptoms of Alzheimer's include memory loss and confusion. As of now, no cures exist but many strategies may help improve symptoms, thus creating a happier life. Alzheimer's is widespread with nearly 44 million people having it (“2015”). Alzheimer's disease is one of the most widespread disorders in the world. Current treatments and therapy can treat symptoms experienced, especially in the early stages, but none can delay or hinder the progression of Alzheimer's. With the need of new treatments to deter Alzheimer's advancement, gene therapy is seen as a great alternative to the current treatments (Nobre). All Alzheimer’s patients …show more content…
This can be seen when neurologists at UC San Diego began experimenting with a new treatment for Alzheimer's. With the use of skin samples from the backs of eight Alzheimer’s patients, the neurologists secluded an enduring type of connective tissue cells. Using a virus, the neurologists genetically modified the cells to attempt to produce and secrete a likely therapeutic protein known as NGF (Nerve growth factor), to try to turn the cells into small biological drug pumps. After this, the neurologists inserted these mutated skin cells straight into each of the eight patient’s basal fore brain. Cells that live in the basal fore brain are usually the first to die from Alzheimer's disorder and the neurologists wanted to test whether NGF could help rescue the withering of the fore brain. After ten years, they checked back on how the patients were doing. The gene therapy seemed to work. The autopsy results show that the mutant skill cells were able to reliably pump out NGF in the patients’ brains, which consequently enhanced the cells’ size and their ability to ramificate new neural fibers. The treatment rescued the susceptible cells from dying off; even the cells that showed …show more content…
Mark H. Tuszynski, MD, PhD, of the University of California, San Diego, and colleagues initiated a clinical trial of NGF (nerve growth factor) in patients with Alzheimer’s disease in 2001 with a goal of determining whether or not NGF could prevent or reduce neuronal degeneration in Alzheimer’s. The study, published in JAMA Neurology, documents the “postmortem findings in 10 patients with survival times from one to 10 years after treatment” (Nobre). During the clinical trials, which ran from March 2001 to October 2012, 10 patients with Alzheimer’s underwent NGF gene therapy using ex vivo (outside an organism) or in vivo (inside an organism) gene transfer. Degenerating neurons in all 10 patients were found to respond to NGF, with axonal sprouting; “a process where fine nerve processes – sprouts – grow out from the intact axons to reinnervate degenerated muscle fibers (“Axonal”).” In three of the patients that underwent the gene transfer, cholinergic neuronal hypertrophy, which was beneficial to the brain, occurred in the NGF-treated side of the brain (Nobre). Additionally, two patients were found to have activation of cellular signaling and functional markers. Neurons exhibited tau (type of protein) pathology and those lacking tau expressed NGF (Nerve growth factor), showing that cells that are dying can be regenerated with

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