By Olivia Baccellieri
The frontiers of medicine and research can reach far and wide in the quest for innovation. Journeys of discovery can be long-winded, or seemingly never-ending, for those seeking answers to scientific questions. One such issue that has long occupied researchers is Alzheimer’s Disease (AD). AD is the 6th leading cause of death in the United States, and takes more lives than prostate and breast cancer combined. The nature of AD is inherently devastating, as it the only disease within the United States’s top-10 causes of death that does not have streamlined prevention or treatments (The Alzheimer’s Association, 2017). For those afflicted with, or for loved ones ailing with AD, the disease can appear as an automatic death sentence. However, in April 2018, researchers at the University of California - San Francisco engaged in a ground-breaking trial that could give newfound hope to those affected by AD.
Wang et al. (2018) utilized human stem cells in the production of neurons for their research. Within their study of neurons, Wang et al. identified apolipoprotein E4, or ApoE4, as a significant genetic risk factor for people to develop AD. ApoE4 neurons have higher levels of GABA deterioration, which is a neurotransmitter responsible for communication between brain cells. When neurons have lower levels of GABA neurotransmitters, inter-cellular communication may slow down; ultimately producing difficulties for memory formation or muscle movement. ApoE4 is further depleted by neural tangles, which may produce plaque build-up in the brain (Wang et al., 2018). Throughout the course of their trials, Wang et al. identified ApoE3 as the most-similar protein to ApoE4, with only one amino acid difference between their structures. Wang et al. then used ApoE3 proteins from healthy human volunteers, and mixed them with the ApoE4 proteins from AD patients. They were able to produce neurons from this mixture of stem cells, which allowed the researchers to change the ApoE4 protein into the ApoE3 form, which lowers the risk associated with ApoE4 (Dovey 2018).
This particular study represents a tremendous breakthrough in neural medicine. Prior to this research, the majority of AD experiments have been conducted on mice brains. This creates a fundamental barrier when trying to replicate results within a human population, and may pose future challenges for researchers if successful treatments on mice are not transferable to humans. Such disparities have produced roadblocks for the development of successful AD treatments, and have stalled drug production (Gladstone Institutes, 2018). Further stem-cell testing should be utilized in order to streamline successful AD treatments, and potentially, even for a cure. Performing the first steps of a clinical trial on mice is understandable, as researchers need to adhere to ethical guidelines in their work. However, some use of human biological subjects is necessary for more accurate implementations of treatment in the human genome.
Studying the genetic and biological makeup of AD has been a long and arduous road for scientists and researchers. 2018 has proven to be a landmark year for medical research, especially in regards to Alzheimer’s Disease. The future of scientific exploration is a Pandora’s Box that the human population should pry open - with care, due diligence and fervor for saving lives.
Dovey, D. (2018, April 11). Alzheimer's disease brain damage was erased by changing the structure of this protein.
Gladstone Institutes. (2018, April 09). Scientists fix genetic risk factor for Alzheimer's disease in human brain cells.
The Alzheimer's Association. (2017, March). Women and Alzheimer's Disease: A Global Epidemic.
Wang, C. (2018). Gain of toxic apolipoprotein E4 effects in human iPSC-derived neurons is ameliorated by a small-molecule structure corrector. Nature Medicine, 24, 647-657.