In his day job, UC Davis scientist Paul Knoepfler probes the inner workings of stem cells and cancer cells and what makes them behave the way they do.
On the side, the father of three daughters blogs about costly, unproven stem cell treatments and provides guidance for those seeking experimental therapies.
Knoepfler is a rare stem cell researcher who regularly explores the most problematic aspects of stem cell therapies on the Internet in full public gaze. He considers himself an advocate for patients as well as a scientist, having survived an aggressive form of prostate cancer at the age of 42.
Now 46, Knoepfler began his blog in 2010, shortly after his cancer was diagnosed. His blogging has encountered resistance from some colleagues, who are uncomfortable with such public endeavors. But he has polished and expanded the blog to the point that it has received international recognition. He will receive an award in December at the World Stem Cell Summit in San Diego for his advocacy efforts.
This month he moved beyond cyberspace and published “Stem Cells: An Insider’s Guide” (World Scientific Publishing). The book covers stem cells from A to Z and is aimed at the general reader, especially people considering stem cell therapy for themselves, a family member or friend.
The book comes during what has been described by Christopher Scott, a senior research scholar at Stanford University, as “an epidemic of transplant clinics offering so-called cures and therapies” both here and abroad. The international stem cell medical tourism business, widely promoted on the Internet, is taking in roughly $1 billion annually, according to an estimate in Stanford Medicine, a publication of the Stanford medical school.
Knoepfler is a believer in the potential and power of stem cells. But he says that some of the dubious stem cell treatments now being offered have resulted in deaths and injuries. Other unpleasant issues arise as well in stem cell research, including the tendency of some stem cells to generate cancer cells, and Knoepfler wants to talk about them.
“Too often in the academic section of the stem cell field, people pussyfoot around the most important issues or do not even dare talk about them at all,” says Knoepfler.
He is “not on some crusade to dissuade people from getting risky stem cell procedures,” but says safety and training need to be encouraged. Reckless behavior endangers the entire field, he says.
“There is no better illustration of the risks of unlicensed stem cell treatments administered by untrained doctors than the recently reported case of a woman who received a stem cell facelift, only to have bone grow in her eye,” he said, referring to a Scientific American report involving a Beverly Hills clinic.
To help patients, Knoepfler’s book begins with the basics: What are stem cells? He moves on to topics ranging from whether stem cells can treat baldness to whether they can help with afflictions such as Alzheimer’s, autism and arthritis. Along the way, he discusses the potential for regenerating limbs, made-to-order organ transplants and curing spinal paralysis, none of which have reached the stage where patients can be assured of using the techniques safely and effectively.
Stem cell treatments are not exactly new. They were first used in the 1950s in bone marrow transplants. More recently, what has excited researchers and the public are pluripotent stem cells, which have the ability to transform themselves into any part of the body. The full range of stem cells, however, includes adult, fetal, embryonic and induced pluripotent cells (iPS), which are also known as reprogrammed adult stem cells.
“Each type of stem cell has clinical promise for specific diseases, but also certain weaknesses. Some stem cells also stir controversy. For example, embryonic stem cells have been at the center of ethical debates for more than a dozen years,” Knoepfler writes.
Embryonic stem cells are derived from an embryo only a few days after fertilization and require destruction of the embryo. It was that type of process that triggered the federal restrictions – now rescinded by President Barack Obama – on federal funding of research using human embryonic stem cells. Some people believe that the process amounts to killing a human being, while others do not.
Knoepfler has come up with a battery of questions and “rights” that persons considering a stem cell treatment should address as they contemplate spending tens of thousands of dollars for treatments.
His “patient bill of rights” includes the right to treatment by a trained provider, the right to continuing follow-up and the right not to be charged to participate in a clinical trial.
Just this month, he added on his blog, ipscell.com, the top 10 questions that patients should ask. They include such areas as the details of the methods to be used and costs, the cell numbers and types, regulatory compliance by the provider, data supporting the cellular product’s effectiveness and data supporting its safety.
As for mainstream media reports and research papers that seem to promise miraculous cures, Knoepler says, “My advice to patients is to be cautious when reading papers on the clinical use of stem cells. Do not believe everything you read and avoid placing too much weight on any one paper. If something is real, it should be reproducible by multiple groups.”
One of the areas of Knoepfler’s research involves cancer stem cells, a topic of special interest to him because of his own encounter with the disease. “Even with something as wonderful as stem cells, there also can too much of a good thing. Stem cells can cause cancer and in certain conditions they can make cancer especially hard to cure,” he explains.
In the case of embryonic stem cells, he cites their ability to form an unusual tumor called a teratoma. Knoepfler, who was an English literature major as an undergraduate, says, “The name ‘teratoma‘ literally means ‘monster tumor,’ and there is good reason for that nomenclature. These tumors look monstrous when observed by eye, akin to an animal put into a developmental blender.”
Knoepfler did not use stem cell therapy to treat his cancer, but he says that as a cancer survivor he understands all too well why patients turn to it when they’re dealing with a life-threatening or life-changing medical condition.
“If you feel that your disease has put you in a place for which patience is not an option,” he says, “talk with your physician. Get second and maybe even third and fourth opinions before deciding whether or not to proceed.”
Knoepfler’s advice may not be appreciated by some of the firms he writes about, but others think well of him.
Jonathan Thomas, chairman of the $3 billion California stem cell agency, says the field needs more scientists like Knoepfler.
“It's hard to overstate the value of what Paul does,” Thomas said in an email.
“Paul is a powerful advocate for helping the public understand what research is being done, and why it is important. He is gifted at taking complex science and turning it into plain English so that anyone can understand what he's talking about.”
Knoepfler has received $2.2 million from the stem cell agency for his research, which is also funded by the federal government and other sources. He has been at UC Davis since 2006, part of the campus’ stem cell program, which has received $131 million in grants from the state stem cell agency since 2005, generating 333 research-related jobs.
Knoepfler, whose father was a physician and mother a counselor, sees a bright future for stem cells, indeed a “medical revolution.”
“Stem cells are today’s new frontier of medicine that will no doubt have an unimaginable impact on our lives, but even more so on the lives of many of our kids and grandchildren,” he said.
Jensen is a retired Sacramento Bee journalist and has produced the California Stem Cell Report (californiastemcellreport.blogspot.com) on the Internet since 2005, writing nearly 3,600 items.