Stem cell research may unlock secrets of incurable diseases

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With stem cells slowly making their way into common medical practice, the answer to hundreds of diseases and disabilities might be contained in one swift injection.

In his lab, David Schaffer, director of the Berkeley Stem Cell Center on campus, and others are currently trying to turn the cells from unused embryos into the specific neurons lost to Parkinson’s disease, a brain disorder that causes tremors and difficulty in movement and coordination. Current treatment options for Parkinson’s are limited to medication, brain surgery and physical therapy, but these only treat the symptoms of the disease.

“If you had the option of having to take prescription medicines for the rest of your life versus a single injection cure … You can see why the field’s so enthusiastic,” Schaffer said.

Others, citing moral and religious convictions, have had reservations about the development of such therapies.

Still, in 2004, California passed Proposition 71, officially deeming stem cell research a constitutional right. A Center of Excellence grant allocated $20 million to UC Berkeley to grow the field, with the money ultimately used for the construction of the Li Ka Shing Center, where the campus Stem Cell Center is housed.

The Scientific Method

The biggest question all researchers within the stem cell community strive to answer is how a stem cell can be controlled — or in Schaffer’s case, how embryonic stem cells can be converted, or “differentiated” into dopaminergic neurons, those chronically lacking in the brain of Parkinson’s patients.

The researchers, including postdoctoral fellows Badri Ananthanarayanan and Tandis Vazin, currently grapple with the challenge of transplanting the neurons from the petri dish, in which they are grown, to the site of injury, all without damaging the cells.

“There’s a 95 percent chance of those cells dying (once you inject them into the brain),” Ananthanarayanan, who works in Kumar Laboratory, headed by bioengineering professor Sanjay Kumar, said. “If you generate these cells in a dish, scrape the cells off the dish, collect them and inject them, a lot of them die in the process because they go through a lot of stress.”

Ananthanarayanan and his colleagues are specifically experimenting on rats, injecting dopaminergic neurons into their brains and subsequently observing whether the neurons function properly and survive. Working with a total of approximately 12 rats, the researchers hope to increase their sample size to about 30 rats once they garner positive results to iteratively test their methods on a larger scale.

Ananthanarayanan said preliminary data is hopeful, and the next step is work on bigger animals, such as monkeys. Vazin said she predicts the team’s work with the rats could be finalized within a year.

But Schaffer predicts it will still take five years before his lab can start clinical trials to test stem cell-based cures for Parkinson’s, as there is much room for error.

The cells must undergo a crucial maturation before they can fully function. This delicate process is achieved by adding genes to stem cells to artificially age them and make them behave as mature cells, explained Lorenz Studer, director of the Center for Stem Cell Biology of the Sloan-Kettering Institute in New York.

In addition to these steps, Schaffer and his team must satisfy FDA guidelines to minimize the risk of harm and test the product’s efficiency, a process that normally takes many years to complete.

“You get stuck in the day-to-day grind, like, ‘Is my experiment working?’” said Dawn Spelke, a bioengineering graduate student who works in Schaffer’s lab but is not working on the study of Parkinson’s. “It’s exciting because it’s starting to be commercialized. We’re seeing that stem cell therapies can work.”

According to Vazin, the team’s goal is working to further understand the intricacies of the differentiation process so they can predict uniformly positive results with every trial. Successful trials would entail that the differentiated neurons do not multiply into a brain tumor or morph into a different cell entirely.

“I think there can be a public lack of understanding of how long therapies take,” Spelke said. “There are complex treatments to complex diseases.”

A philosophical debate, a medical advancement

According to researchers, this lack of understanding on the subject has also manifested as a philosophical debate among people who believe stem cell research to be immoral or even illegal.

Though federal funding for stem cell research has been consistent, public opposition, which peaked in the mid 2000s, creates precarious circumstances for researchers working to push forward the medical benefits of stem cell research.

“At any point in the future, if the political winds shift in the other direction, the field could become very restricted again,” Schaffer said.

From Studer’s perspective, most of today’s opposition comes from “fundamentalists” who equate the life of an embryo to the life of the baby.

Many researchers reason that because surplus egg cells, harvested in large quantities from the woman for in vitro fertilization, are usually thrown away after the fertilization process, the remaining egg cells could be used to conduct embryonic stem cell research.

“It is an opinion of religion and morality,” Studer said. “Embryos have moral value, but it’s a stretch to give more moral value to a fertilized egg than to an actual baby — an actual human.”

According to Lily Mirels, the program manager of the Berkeley Stem Cell Center, new strides in medicine have introduced alternative options for stem cell research that have begun to assuage much of the opposition surrounding the technology. Using induced pluripotent cells, stem cells not deriving from embryos, the same methods of experimentation can be executed to garner the same results.

Since the discovery of these cells in 2006, coupled with the recent medical breakthroughs emerging from institutions like the Berkeley Stem Cell Center, some of the controversy has begun to fizzle out.

According to a  a 2012 report by the National Science Foundation, the stigma surrounding stem cell research has notably dwindled in recent years, with 62 percent of Americans favoring embryonic stem cell research in 2010 and even more favoring studies with stem cells derived from alternative areas such as bone marrow and the brain.

Schaffer and his team, who have yet to encounter hostility to their stem cell research, are now focused on moving forward with their research and pushing the boundaries of biomedical capabilities.

“We believe it is the future of all medicine,” Spelke said.

Lydia Tuan covers research and ideas. Contact her at [email protected] and follow her on Twitter @tuanlydia.

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  • HeraSentMe

    We have ample evidence of what autologous (our own), bone marrow derived stem cells can do, especially for orthopedic problems like mine. My current FDA approved options are to submit to invasive, risky, potentially deadly, traditional back and knee surgeries, which typically result in the need for additional surgeries within three to ten years and narcotic drug dependencies (or take NSAIDS that TRIPLE my risk of a serious
    cardiovascular event) or be forced off-shore at great cost and physical pain to
    use my own stem cells. Three of my physicians, including one educated at Oxford, fully
    support my choice of treatment and my doctor of choice, Centeno, in lieu of traditional back and knee surgeries at my age. Moreover, recent research reveals that fake knee surgery is just as efficacious as traditional knee surgery: not very efficacious. Similarly, the human spine is meant to be flexible. Fusion leads to additional problems in other areas of the spine. Nor does the idea of having a narcotic pain pump installed in my back appeal to me. It is noteworthy that almost everyone has back and/or knee problems by the time they are in their forties; this is not an insignificant population in the
    medical community.

    Although embryonic stem cells, induced pluripotent stem cells, and other “designer cells” may be important for future generations, I do not have decades to wait. I have been a
    disabled “shut in” for over two years now; this has been emotionally and financially devastating to my family and me. There is no justification for holding what should be a practice of medicine to the standards of the mass manufacture of pharmaceuticals. There is no public health threat that merits the FDA’s regulatory overreach into the practice of medicine, which is already regulated by state boards, hospital privileges, the tort system, etc. Only I will receive my cells; they will not be distributed to the masses like a pharmaceutical drug.

    Safety and ethics are red herrings in the debate over autologous stem cells. If heart transplants, gastric bypasses, or liposuctions had been held to these new regulatory standards, they would never have become standard practice of medicine. Autologous stem cells, administered properly, have been found safe in numerous studies, despite some media articles that portray autologous stem cells as risky.

    I have already spent a fortune over the last two decades on FDA approved, standard of care medications and treatments for my back and knees, to no avail. Moreover, many of my medications and approved treatments have dangerous side effects or simply do not work at all. I find those treatments to be unsafe and unethical. I find it unethical that, under the current FDA overreach, I will be forced to travel outside the US for autologous
    stem cell treatments for my back conditions. I find it unsafe and unethical that the currently approved treatment options for my back and knees are invasive, risky, and typically lead to additional surgeries and life-long drug dependencies. FDA approved drugs have to only be slightly more efficacious than placebos to get approval in many cases. How is that safe or ethical? FDA approved drugs are one of the leading causes of death in this country. How is that safe or ethical?

  • Pat A

    SammyJo – I concur. Voters need to be wary of the promises this time around. Some day should be TODAY. There is already plenty of safety data from adult stem cells. Why hasn’t CIRM got the ball rolling so patients instead of mice are being treated? CIRM has an obligation to taxpayers to get patients into the clinic. They could have concentrated on this early on, but instead it’s become a huge grant trough for researchers who as you say are tinkering around. Not impressed. Voters were promised so much more than has been delivered.

  • SammyJo Wilkinson

    taxpayers get ready, you’ll be seeing lots of “someday” articles like
    this during the CIRM campaign for the next $5 billion bond. But we don’t have
    to wait. Stem cells don’t need to be controlled, unless you are a researcher
    who needs to keep the grant $$ flowing for years while they tinker around
    trying to reinvent nature’s wheel. Your own Autologous Mesenchymal Stem Cells (AMSC) know exactly what
    tissue to repair or become. Over 2,000 humans have been safely treated with
    expanded AMSC Physicians were quickly adopting this (and saving lives) until
    the FDA declared AMSC stem cells are a drug, requiring years more testing, and
    of course drug approval fees to FDA. I know this story first hand, I got this
    life saving therapy for MS, and I know many patients who reversed Parkinson’s
    and other serious ailments. That was before FDA slammed the door shut. Unfortunately
    for them some of us got treated, and our recoveries compel us to expose the
    truth. and tell everyone our right to this therapy is being preempted. If you
    want to know what’s going on, we are explaining this at

  • Jarrod

    This is a good thing all the way around. Thee is so much good that can come from this.