Stem Cells to Cure Disease

By Stephanie Smith

BU News Service


Despite the great debate over bioethical issues, stem cell research continues. Now, Boston-based researchers think they have hit upon a major development in stem cell studies, one which could benefit a large populations of patients suffering from inflammatory diseases.

A group of researchers from Brigham and Women’s Hospital, the Harvard Stem Cell Institute, MIT, and Massachusetts General Hospital have uncovered a way for adult stem cells to act as a “drug factory” at the site of inflammation in the body.

Speaking to the Harvard Stem Cell Institute, researcher Jeffrey Karp said, “If you think of a cell as a drug factory, what we’re doing is targeting cell-based, drug factories to damaged or diseased tissues, where the cells can produce drugs at high enough levels to have a therapeutic effect.”


A graphic depiction of the cells acting as a drug factory to suppress the inflammation.
A graphic depiction of the cells acting as a drug factory to suppress the inflammation.

Lead author Oren Levy, PhD was cautiously optimistic about the discovery’s potential to treat certain diseases associated with inflammation, such as heart attack, multiple sclerosis, and even certain types of cancer.

“It’s important to not create false hope, but we do see a lot of progress to be made,” Levy said.

Levy, started the research venture to test his hypothesis that stem cells could be used to target a localized region of the body. The study, published on Aug. 21 in the scientific journal Blood, also included study authors Jeffrey Karp, Weian Zhao, Mehmet Fatih Yanik, and Charles Lin.

Stem cells are undifferentiated cells – essentially cells that haven’t decided yet if they will become a blood cell, or a skin cell or any other kind of specialized cell. At the “stem cell” phase, these cells still have the potential to turn into any specialized cell. This study used adult stem cells, which are less controversial than embryonic stem cells only found in embryos that have not fully developed.

Researchers tweaked these adult stem cells using modified strands of mRNA, a type of single stranded genetic material similar to DNA that is naturally found in human cells. Upon mRNA insertion, the cells produced an anti-inflammatory protein, known as interleukin-10.

These modified human stem cells were injected into the bloodstream of a mouse with inflammation in the ear. The cells targeted the site of inflammation and released the interleukin-10 to reduce the swelling.

“We basically used the cells as a vehicle to deliver, and they did,” Levy said in prepared remarks.

Ultimately, the group was able to develop a type of cell that has the capability of reaching the site of inflammation and suppressing  it.

“If you think about biological therapeutic drugs, they’re a huge part of medicine, but there [are] still challenges,” he adds. “It’s hard to target them where you want them to act.”

Though this study looked at a simple local inflammation model in the ear of a mouse, the group hopes to target a clinically relevant model in the future for common conditions like inflammation in the wrists caused by arthritis. But, Levy warns, “This has a lot of potential happening in the next years to come, but it’s still early.”

This research garnered from the stem cell studies could potentially lead to treatments, or even cures, for diseases caused by chronic inflammation like atherosclerosis, arthritis, and even some cancers that progress due to inflammation.

Sarah Anderson, a student studying Economics at the University of North Carolina, Chapel Hill, was diagnosed with juvenile rheumatoid arthritis (JRA) at age three. JRA affects more than 250,000 children. Since then, she has lived with the burden that inflammatory disease has on a patient and loved ones.

“I’ve been through it all—oral medications, steroid injections, which are super painful because they have to get directly to the bone, physical therapy, and now I’m on a weekly injection,” she said. “When initially being tested, and doctors telling my mom that it could be leukemia, she said that it was the worst year of her life.”

Although the steps toward treating larger scale diseases with this type of stem cell therapy will take time and additional progress in the lab, Levy and colleagues are optimistic about the future.

“We’re harvesting the knowledge that we already have to hopefully modify cells to go to any relevant organ that needs treating,” Levy said. “We see a lot of progress for autoimmune diseases and other inflammatory diseases.”

While researchers remain optimistic, patients, like Anderson, remain somewhat skeptical. “Since joint damage is a side effect of arthritis, when not treated, I would want to see how this medication works long term,” she said. “I would love a cure one day or to stop these inconvenient injections, but when it comes to my health, I need to know with certainty that the benefits outweigh any costs.”

Now, Anderson is on a weekly regime of injections and says that after suffering for 17 years she has finally controlled her symptoms. “I have only been off medication for one year over the last 17 and that was when I was in remission in second grade,” she said. “Luckily today, even though I am not in remission, I do have it under control, but the future remains a mystery.”

Researchers say that though they are making progress with the development of the drug, there will not be an opportunity for public use for several years.

“We’ll keep on trying and keep on optimizing the process until we get a maximal response,” Levy said. “We’ll keep an open mind until we solve it.”