Personalized Therapeutic Testing for the Ultra-Rare is Coming

In breaking news for the progeria community, biomedical engineers have just succeeded in growing miniature human blood vessels that mimic progeria, the ultra-rare genetic condition characterized by premature aging in children.
Progeria is fatal by the early teenage years in the children it affects, so scientists are truly racing against the clock to come up with treatment options.

The only treatment available only extends a patient’s life by three months, on average, which was considered a major feat. This only proves how devastating progeria is. To learn more about the disease, click here.

That’s why the development of these miniature human blood vessels is such a breakthrough. They have been engineered to exemplify some of the common symptoms and drug reactions that associated with progeria in just four weeks, so this technology will give researchers and doctors the opportunity to screen potential therapies for the condition much more rapidly.
In addition, progeria is so rare that there simply aren’t enough patients to run the numerous clinical trials that are needed to develop a treatment. These blood vessels would provide an additional outlet to test clinical compounds, so more therapies can be tested.

All of these characteristics have not been recreated in any other simple cell cultures thus far, much less in such a quick, high-throughput, and detailed manner.

The ultimate goal is to create a platform for more personalized screening, which could have implications for other ultra-rare diseases, as well as developed heart disease in the elderly.
While there are limitations to this new technology (since the artificial blood vessels are not connected to any outside organs), it is clear that a new personalized testing platform for ultra-rare patients is on the horizon, and with it, hopefully more effective and personalized medicine.

To read more about the development of this new technology in Science Daily, click on the article here!

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