Over the last few years, many researchers have been utilizing organoids as a pathway to learn more about diseases and spur drug development. Organoids may be helpful, moving forward, in enhancing the drug discovery and development process within the rare disease or rare cancer sphere. For example, organoids could help identify potential therapeutic options for difficult-to-treat illnesses, such as chordomas. A chordoma is a rare and malignant tumor that is found on the spine. Historically, chordomas have a high rate of recurrence and limited treatment options. More so, the incidence of chordoma is so rare that it can be hard to study as well.
According to a ScienceDaily news release from the University of California – Los Angeles Health Sciences, researchers have created personalized organoid models of chordoma for drug development. These organoids will, hopefully, help researchers to investigate the tumors’ underlying biological characteristics as well as test and identify potential therapeutics.
To learn more about this study, take a look at the information published within Science Advances.
The Process of Organoid Creation
To begin, what exactly are organoids? The Harvard Stem Cell Institute (HSCI) describes organoids as:
tiny, self-organized three-dimensional tissue cultures that are derived from stem cells. Such cultures can be crafted to replicate much of the complexity of an organ, or to express selected aspects of it like producing only certain types of cells.
Within this particular study, researchers sought to create chordoma organoid models. To begin, the researchers sourced seven tumor samples from five patients. Next, researchers developed organoids from the sourced tumor samples. Much like chordoma tumors, the organoids had characteristics like Ki-67 expression and brachyury. Once the organoids were created, the researchers then began performing high-throughput drug screening. To learn more about high-throughput drug screening and its efficacy, head to BMG Labtech.
Through this process, researchers were able to discover:
- Potential biological pathways to target for more effective treatments
- Various drug targets
What is a Chordoma?
TBXT gene mutations cause chordomas, a malignant tumor that forms on the spine, to develop. These mutations cause excess brachyury production. Normally, this protein plays a role in spinal development, though researchers are not sure why excess protein causes the tumors. Despite the fact that a chordoma typically grows slowly, it is possible for the tumor to spread into other bones and tissues. Chordomas are most often found where the spine meets the skull (clival) or near the tailbone (sacral). Chordoma tumors are more often found in males than females and manifest between the ages of 40 to 70. Symptoms vary depending on which part of the spinal cord is affected. Some common symptoms include:
- Pain, weakness, and numbness in the back and extremities
- Headache
- Double vision
- Lump at the base of the spine
- Runny nose or nosebleeds
- Bladder and bowel problems
Learn more about chordomas.
