New stem-cell-derived cells hold promise for Alzheimer’s, other brain diseases
November 9, 2012
Choroid plexus epithelial cells generated in a culture medium using embryonic stem cells
(credit: Edwin S. Monuki and Momoko Watanabe/USPTO)
UC Irvine researchers have created a new stem cell-derived cell type with unique promise for treating neurodegenerative diseases such as Alzheimer’s.
Dr. Edwin Monuki of UCI’s Sue & Bill Gross Stem Cell Research Center and colleagues developed these cells — called choroid plexus epithelial cells (CPECs) — from existing mouse and human embryonic stem cell lines.
CPECs are critical for proper functioning of the choroid plexus, the tissue in the brain that produces cerebrospinal fluid (CSF ). CPECs make CSF and remove metabolic waste and foreign substances from the fluid and brain, among other tasks.
In neurodegenerative diseases, the choroid plexus and CPECs age prematurely, resulting in reduced CSF formation and decreased ability to flush out the plaque-forming proteins that are a hallmark of Alzheimer’s. Transplant studies have provided proof of concept for CPEC-based therapies. However, such therapies have been hindered by the inability to expand or generate CPECs in culture.
“Our method is promising, because for the first time we can use stem cells to create large amounts of these epithelial cells, which could be utilized in different ways to treat neurodegenerative diseases,” said Monuki, an associate professor of pathology & laboratory medicine and developmental & cell biology at UCI.
To create the new cells, Monuki and his colleagues coaxed embryonic stem cells to differentiate into immature neural stem cells. They then developed the immature cells into CPECs capable of being delivered to a patient’s choroid plexus.
These cells could be part of neurodegenerative disease treatments in at least three ways, Monuki said. First, they’re able to increase the production of CSF to help flush out plaque-causing proteins from brain tissue and limit disease progression. Second, CPEC “superpumps” could be designed to transport high levels of therapeutic compounds to the CSF, brain and spinal cord. Third, these cells can be used to screen and optimize drugs that improve choroid plexus function.
Monuki said the next steps are to develop an effective drug screening system and to conduct proof-of-concept studies to see how these CPECs affect the brain in mouse models of Huntington’s, Alzheimer’s and pediatric diseases.
The study as supported by the National Institutes of Health, the California Institute for Regenerative Medicine, UCI’s Institute for Clinical & Translational Science, and UCI’s Alzheimer’s Disease Research Center.
References:
- Momoko Watanabe et al., BMP4 Sufficiency to Induce Choroid Plexus Epithelial Fate from Embryonic Stem Cell-Derived Neuroepithelial Progenitors, The Journal of Neuroscience, 2012, DOI: 10.1523/JNEUROSCI.3227-12.2012
- GENERATION OF CHOROID PLEXUS EPITHELIAL CELLS FROM HUMAN EMBRYONIC STEM CELLS (patent application 20120201789, Aug. 9, 2012)
Comments (5)
by Bri
The problems outlined in reference to bringing drugs to market, are from older systems of regulation and governance. Whether we like it or not, we need regulations. Here in sandy’s aftermath we need inspectors to assess damaged houses and new repairs. Without these regulations people are literally burning down their houses, from salt water damage. New repairs need to be done properly to make sure a similiar siduation doesnt happen.The problem is that this takes time and is refered to as red tape. There are ways to streamline the system, but they also take time to design and implement. It would be nice if our local and federal governments were more focused on these issues, but unfortunately they are often influenced by lobbies. As we become more connected and AI is more capable of intervention, we may be able to address these issues. Till then the red tape serves the purpose of limiting the run away problems that might arise from errors of judgement. I think most of congresses time is wasted on bickering over measures that serve the few, not the general populace. Look at the problems in association with Sandy. There was so little foresight and yet catastrophes like this have been happening since the beginning of our country. What? They didn’t think the tunnels would flood? The power lines were vulnerable? The rules and regulations have been set to favor short term profits and not the likelihood of eventual disaster. If we had had a thirty foot storm surge, the damage would have been more than three times worse. Saying that this is a once in a lifetime disaster is saying it would happen in my life time. They begged for these problems. In a similiar way we could have runaway problems if we just forgo the FDA antiquated rules. Time must be taken to set up regulations that safely address the issues and yet streamline the rapid descoveries of our accelerating technology. It’s a tuff issue that needs much debate. I would prefer prudence over emotionalism. These afflictions have been causing misery for all of mankinds history. Let’s push for a safer and quicker system, though an open debate, in a democratic fashion.
by dk
why must we continue to read about these discoveries while our loved ones suffer. A civilized and rational FDA would allow stream lined testing for people with nothing to lose. My father has nothing to lose at this point and the politicians, lawyers, and pencil pushers drag thier feet while millions suffer
by Danny
Any chance of this working to help those of us affected by Parkinson’s disease?
by judge
This approach appears to go to an actual preventive and reversal of these diseases which in tandem with the amyloid protein inhibitor approach is very encouraging- onward and upward!
by Bennie Beaver
Hip hooray! These genetic are closer to a final solutions to disease, etc., are just what we need. End solutions that simply further empower health care manufacturing, doctors, clinics, hospital, and specialist. Otherwise, the America and the world will go broke. We need similar solutions reduce energy cost.