Improving Stem Cell Treatment with Novel Drug Compound

One of the primary limitations of stem cell treatment and the subject of ongoing research is the challenge of directing stem cells to their necessary destination in the body which has been a subject of ongoing research. Prior studies have discovered that stem cells are drawn to inflammation in the body, however, using this as a therapeutic lure still poses risks. Thus, researchers continue their search for tools that would aid stem cells in their migration and conversion into specific types of cells necessary for optimal treatment. The ability to do so would have a wide range of implications for regenerative medicine as well as the treatment of disorders in which inflammatory signals fade over time, such as chronic spinal cord injury, stroke, or conditions in which the role of inflammation remains unknown. 

Scientific findings published in the Proceedings of the National Academy of Sciences (PNAS) reported the development of a drug with the potential to lure stem cells to damaged tissue and improve overall treatment efficacy. A scientific first and major advancement in anti-aging medicine, this discovery could improve current stem cell therapies designed to treat such neurological disorders as spinal cord injury, stroke, amyotrophic lateral sclerosis (ALS), and other neurodegenerative disorders; and expand their use to new conditions, such as heart disease or arthritis.

Evaluating Effects of the Novel Drug  

A group of scientists at Sanford Burnham Prebys Medical Discovery Institute modified CXCL12, an inflammatory molecule which could guide healing stem cells to needed sites of repair, to create a drug called SDV1a. The novel drug works by enhancing stem cells binding and minimizing inflammatory signaling; it can be injected anywhere to lure stem cells to a specified location without causing inflammation.

According to the study’s authors, the modified CXCL12 compound can be used to create a drug that draws stem cells to the region of pathology without unwanted adverse effects while also improving the overall efficacy of stem cell treatments. To prove this hypothesis, the researchers implanted SDV1a and human neural stem cells into the brains of mice with the neurodegenerative disease called Sandhoff disease.

SDV1a and Cell Direction

Per the study’s findings, SDV1a helped the human neural stem cells migrate and perform healing functions, which included extending lifespan, delaying symptom onset, and preserving motor function for much longer than evidenced in the mice that did not receive the drug. Importantly, inflammation was not activated, and the stem cells were able to suppress any pre-existing inflammation. In addition, toxic cells disappeared when mice with neurodegenerative conditions received therapeutic stem cells and SDV1-a which corresponded with longer lives and delayed symptom onset.

The study’s results suggest SDV1a can be used to improve efficacy in stem cell treatment. “The ability to instruct a stem cell where to go in the body or to a particular region of a given organ is the Holy Grail for regenerative medicine,” Evan Y. Snyder, M.D. Ph.D., professor and director of the Center for Stem Cells & Regenerative Medicine at Sanford Burnham Prebys and senior author of the study told Eureka Alert. “Now, for the first time ever, we can direct a stem cell to a desired location and focus its therapeutic impact.”

“Thanks to decades of investment in stem cell science, we are making tremendous progress in our understanding of how these cells work and how they can be harnessed to help reverse injury or disease,” Maria T. Millan, M.D., Maria T. Millan, MD, president and CEO of the California Institute for Regenerative Medicine (CIRM), which contributed to research funding, told Eureka Alert. “Dr. Snyder’s group has identified a drug that could boost the ability of neural stem cells to home to sites of injury and initiate repair. This candidate could help speed the development of stem cell treatments for conditions such as spinal cord injury and Alzheimer’s disease.”

The research team is already continuing its study by testing SDV1a’s ability to improve stem cell treatment in mice models of amyotrophic lateral sclerosis (ALS). They are optimistic about the drug’s mechanism of action and potential to improve the treatment of a variety of neurodegenerative diseases as well as non-neurological health conditions. However, as it is the first of its kind, the novel compound will require extensive further study before it can be used more broadly in a clinical setting.

Key Takeaways

The inability to guarantee the proper migration and targeting of cells as part of stem cell therapies has proven to be a significant limitation. Novel compounds aim to assist stem cell techniques by promoting proper migration and function leading them to be more effective and widely-used.