by Don C. Reed
There are some people so valuable I wish the world could put a magic shield around them, to provide for their needs and protect them from harm.
One such person is Paul Knoepfler, a professor at UC Davis School of Medicine. His lab focuses on the roles of stem cells in brain tumors, normal brain development, and neurodevelopmental disorders. He also does educational outreach to help the local and global communities including through his site “the Niche”, Ipscell.com.
To my knowledge he was the first stem cell scientist-blogger in the world.
The particular help Paul needs is research funding to tackle the chores — and chores they certainly are!
I saw a science film once of a person with a brain tumor, brutally discouraging because it kept coming back. One second the tumor seemed gone, the next it was there again. What determination it must have taken for the scientist to keep returning to the battle. (The film was not Paul in action, but it was the sort of thing he does — to fight for the health of the brain.)
Paul says: “The two areas we most need research funding is our pediatric brain tumor work and cerebral palsy. We are working to develop new therapies for the 100% fatal brain tumors ( diffuse midline glioma) in kids. We are defining the vulnerabilities of those cancers — to know how to defeat them — and are testing drugs…for a possible clinical trial.
“…we used gene editing to change a cancer-causing mutation (in the deadly childhood brain cancer)… back to the normal version of the gene.
“We also did the reverse (using) gene editing to give brain cells the cancer-causing mutation. Together, this allowed us to (study) …how the mutation causes the tumors and how to target them.
The other major thrust of Paul and his team’s effort is Cerebral Palsy (CP). Paul’s team is studying two main contributors to CP: low oxygen (hypoxia) and genetic factors. They are using brain organoids (tiny living models of the developing human brain made from stem cells) and CRISPR gene editing for these studies. The core idea here is that certain mutations may make some future kids much more susceptible to hypoxia in the womb. Figuring out how this happens could lead to new prevention and treatment strategies.
According to the Cerebral Palsy Foundation:
“Cerebral Palsy (CP) is the most common lifelong motor impairment impacting more than 17,000,000 people around the world.” Seventeen million? What a staggering number!
How are they doing? Quoting Paul:
“On the Cerebral Palsy (CP) research front we have defined some potential genomic mechanisms that contribute to CP in part using human stem cell based brain “organoids”. (Like dots in a dish, small collections of cells).
These genomic elements may cause CP directly .
Why would anyone want to make cerebral palsy-like conditions in an organoid model?
It reminds me of something Abraham Lincoln once said:
“Before we can know where we are going, we must first know where we are.”
We must study the enemy, and learn its weakness, to know how to defeat it.
One more thing about Paul.
Most scientists are (understandably!) too busy to answer questions. They are always swamped with work, and I hesitate before disturbing them. Also, some have difficulty with “people talk”. Their vocabularies are huge, and they seem to be talking in Latin! Paul will break down what he is saying into smaller words, until you and he are both on the same page — a huge favor for non-scientists like myself.
What does this all boil down to?
If you can find a way to help Paul and his crew in their struggle for funding, please do so. If you hear that he is up for a grant, raise your voice on his behalf.
You will be doing a favor for science, and for children who suffer a terrible condition.
I recommend Dr. Paul Knoepfler in the strongest possible terms.