By Don C. Reed
Author, “STEM CELL BATTLES”, other books
Dear Friend of Stem Cell Research:
For the next month, I will make available a daily summary of one aspect of stem cell research—my layman’s understanding of it—done by scientists connected to the California Institute for Regenerative Medicine (CIRM). The first was spina bifida; the rest follow in no particular order. You can read the whole series at my website: www.stemcellbattles.net.
Most scientific writing was done by Kevin McCormack; friendly edits by Melissa King. Mistakes are my fault.
In most cases I have left out the scientists’ names. A few I have written about in my books, and those I felt free to credit directly. My main reference is always CIRM itself.
All I ask is that when you step into the voting booth, please consider which political party is likely to fund such research, and vote accordingly.
Remember in November!
HUNTINGTON’S DISEASE: Total Awards 15: Total Value: $34,496,262
Imagine a beloved parent, someone you had loved and trusted all your life, suddenly becoming mean, as if he or she might hate you? Huntington’s Disease not only brings mental/emotional/psychiatric changes, all bad, but physical damage as well, leading to paralysis, and a slow (10-20 years) spiral down to death. And there is a 50% chance the condition may be passed on to the next generation… (1)
We who support the California stem cell program know families that have lost entire generations to this disease.
But across our state, this horrific condition is being fought.
“Huntington’s (is caused by) a mutation to one gene. This gene produces a protein that is toxic to nerve cells and eventually kills them. CIRM has funded several projects that probe into both the nature of the mutation, and ways to prevent or repair the damage from the (toxic) protein.
“One obstacle to…potential therapies…is the lack of a good lab model to capture the complexity of the disease in people. Accordingly, researchers are using stem cells to create a “disease in a dish” model, which allows them to create cells with different forms of Huntington’s, and then screen different drugs to fight HD.
(Researchers are…creating these) “stem cells from patients with HD in hopes of genetically modifying the cells (to) produce the correct HD protein. This could be used as a personalized therapy: custom-designed for each person with HD to reduce the chance their own immune system would attack the new cells. Two other CIRM-funded teams seek to identify therapies that could be delivered to the damaged nerves…
“(A team at) UC Irvine proposes to use embryonic stem cells, matured into early forms of nerve cells… modified to deliver a compound that could protect patients’ other nerves from the toxic protein. (2)
“At UC Davis, a team plans to use mesenchymal stem cells, (from bone marrow), to deliver a genetic fragment to the nerves– and shut down the faulty gene. (3)
For decades, the Huntington’s community has been fighting the good fight: supporting the research, CIRM, and the people they love: patients living with the disease for far too long: for some, decades. The Huntington’s families worked hard to help pass the renewal of the program; they are exemplary.
1. https://www.cirm.ca.gov/our-progress/disease-information/huntingtons-disease-fact-sheet
2. https://innovation.uci.edu/2021/03/leslie-thompson-tackles-huntingtons-disease-one-gene-at-a-time/