Alzheimer’s disease is classified as one of the top five challenging and enigmatic neurological disorders, and, up to present, there are only a handful of treatment interventions that have marginal effectiveness; it is incurable. Nonetheless, the advances that have been made in scientific studies provide a fresh understanding of the processes causing the disease and potential new therapeutic targets. Perhaps the most encouraging discovery is that made in the course of defining new goals for drug therapy for Alzheimer’s targeting any part of this disease, such as the cerebrospinal fluid—a liquid that bathes the brain and spinal cord. This development will not only enhance knowledge on Alzheimer disease at the molecular level but also create opportunity for possible treatment in the future.
Cerebrospinal Fluid: The Future Role in Alzheimer’s Research
Over the years, the principal object of study of Alzheimer’s has been the cerebrospinal fluid. This fluid serves as a medium of communication between the brain and the other parts of the body; it also carries so many other biomarkers that the scientist uses in describing the progression of the disease. Historically, knowledge regarding Alzheimer’s illness has centered on the genetic approach, amyloid plaques, and tau tangle formations within the human brain. While these remain key hallmarks of the disease, recent studies indicate that alterations in the protein composition of CSF might also provide important new insights into disease onset and progression.
A team of researchers affiliated with Washington University School of Medicine in St. Louis conducted a landmark study in pursuit of the cerebrospinal fluid molecular changes in subjects diagnosed with Alzheimer’s. This research project analyzed the CSF protein levels against other tissues like blood plasma and brain tissue and identified several proteins that could be treated with new Alzheimer’s drugs.
Unvealing New Drug Targets
The new study applies the most recent technologies to map protein profiles within the CSF of patients with and without Alzheimer’s disease. The research team, studying protein levels from almost 1,600 individuals, compared proteins from people with Alzheimer’s to healthy controls. This large-scale analysis discovered 274 proteins within the CSF that were associated with Alzheimer’s disease. These proteins include some that have never been related to the disease, thus giving further insight into the biological pathways involved.
These proteins serve as potential drug targets that could influence the slowing or possibly even reversal of cognitive decline in patients with Alzheimer’s disease. This is quite far from previous approaches, which involve targeting genes that have been predominantly linked to Alzheimer’s. The research team has focused on the actual proteins involved, since these are the molecules directly implicated in both brain function and disease progression.
Repurposing of Already Available Drugs: A Potential Capability
One of the more intriguing aspects of the study was the identification of already-approved drugs that could be repurposed for the treatment of Alzheimer’s. The research team has cross-referenced the proteins in CSF with the existing database on drugs and found 15 FDA-approved drugs that could target newly identified protein pathways in Alzheimer’s. Because these drugs have already been extensively tested for their safety in other conditions, they could be fast-tracked into clinical trials for Alzheimer’s disease.
There are a number of advantages to repurposing existing drugs. First, it enables big reductions in the normal time and cost associated with developing new drugs. The new Alzheimer’s targets are already well understood at a molecular level, which means these repurposed drugs could be tested in clinical trials sooner than completely new drug candidates. These drugs also have established safety profiles, making them prime candidates for Alzheimer’s trials that could potentially shorten the path to treatment for patients.
Drugs identified in this study are currently used to treat various diseases. For example, an antibody drug that has already been approved for the treatment of leukemia works by targeting a protein called CD33; it could be reused in treating Alzheimer’s patients. On the other hand, one of the drugs identified previously was administered to people suffering from osteoarthritis and targets a protein implicated in Parkinson’s disease, although it is also a potential Alzheimer’s treatment. The possibility of leveraging existing drugs for Alzheimer’s may accelerate treatment options and offer hope to patients who have struggled for a long time with limited choices.
The Way Forward: The Bright Future of Medication for Alzheimer’s Disease
These new drug targets and the possibility of repurposing existing drugs in the treatment of the disease highlight a major leap forward in the fight against Alzheimer’s disease. But much work remains to be done. These would have to be followed by clinical trials aimed at assessing safety and efficacy in the setting of Alzheimer’s disease. Given such promising findings, a research team at Washington University School of Medicine remains optimistic about possible rapid translation from the laboratory to the clinic.
Moreover, the research team, while mostly focusing on already existing drugs, is using advanced techniques to refine their search for other promising drug targets, even applying machine learning. By applying these techniques, they hope to further understand how various proteins in the brain contribute to Alzheimer’s progression and identify even more precise and effective therapeutic targets.
Further, this research points to the importance of investigation into CSF and other body fluids that may give an even more vivid picture of Alzheimer’s disease at the molecular-genetic level. Elaboration of a detailed understanding of protein interactions with each other and with brain cells will be required for future treatments aimed at the halting or reversal, rather than simple amelioration, of symptoms.
Why This Research Matters
Because identifying new drug targets from the CSF allows for a treatment that will be more focused and hopefully effective in treating Alzheimer’s disease, it is truly exciting. For many years, Alzheimer’s disease has held secrets about its underlying biological processes, but focusing on proteins and their role in the disease has allowed scientists to start cracking that code. While this work brings hope to the patients and family members suffering from this disease, it serves as a stepping stone toward new frontiers in personalized medicine related to neurodegenerative diseases.
In all, the journey to find a cure for Alzheimer’s is arduous and circuitous; the identification of new drug targets from cerebrospinal fluid provides a milestone. Equipped with such knowledge, scientists are now better poised to develop drugs that may slow down or even reverse the effects brought on by Alzheimer’s disease. As research unfolds, the hope is that the next generation of treatments will finally bring relief to millions of people coping with this devastating condition.
