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Engineered Immune Cells May Yield Novel Disease Therapies

Director of the UCSF Center for Systems and Synthetic Biology Wendell Lim (UCSF).Director of the UCSF Center for Systems and Synthetic Biology Wendell Lim (UCSF).
Director of the UCSF Center for Systems and Synthetic Biology Wendell Lim (UCSF).
Director of the UCSF Center for Systems and Synthetic Biology Wendell Lim (UCSF).
Rick Pantaleo
Researchers in California say that someday, doctors will be able to treat serious illnesses with modified cells, adding that the technique could become as common as it is now to treat the sick with drugs.

Researchers at the University of California, San Francisco say novel cell therapies have the potential to address critical needs in the treatment of some of the deadliest illnesses, including diabetes, cancer and inflammatory bowel diseases.

These possibilities are described in an article published in the online journal Science Translational Medicine, co-authored by Professor Wendell Lim, who is also director of the UCSF Center for Systems and Synthetic Biology. Lim says our body’s natural disease-fighting systems could be harnessed to do much more.

“Our bodies are made of cells and we have in our bodies cells, like immune cells, that go around and protect us," said Lim. "So, they actually carry out complex therapeutic functions.  What we just haven’t really found a lot about is the idea that we can actually use these cells, these living sort of entities, as the actual medicine.”

Lim says researchers have been developing complex new cell therapy strategies that build on our growing knowledge of how genes program the development and inner workings of cells.

For example, because the body’s natural immune response to spreading cancer cells is often weak, scientists are engineering and growing populations of immune cells that target specific molecules found on cancer cells.   Lim said that there have already been some remarkable cancer recoveries that can be credited to these experimental cell therapy treatments.

“In the last year or two, there have been some other really exciting findings that have shown that the idea of using cells as therapies maybe have some real legs [can exist and be successful]," he said. "One of them is that people have started taking out immune cells from patients who have cancer and actually engineering them to now attack and kill that cancer.  And, that’s turned out to be remarkably effective for a handful of patients with leukemia and lymphoma that have been treated with this kind of engineered immune cell.”

As with any proposed new medical treatment, the cell therapies that are currently being developed will face lengthy and rigorous testing by independent laboratories and regulatory agencies before they can be put to regular use.
But Lim says the testing will not only protect any of those who may use the therapies, but may also play an important role in further developing and refining the therapies themselves.

“You know a lot of drugs that we use as therapeutics started out as some natural product within the bark of some tree," he said. "And really that’s not a very controlled way of treating a disease. You have to know how to purify that compound, how to make variants of it that optimize the efficiency, but also minimize toxicity.  These are the type of things that we need to be able to do to cells to make this viable.”
Lim and his colleagues are conducting a daylong symposium on April 12 to discuss the future of cell therapy.  The meeting will feature talks by some of the nation's leading researchers and biomedical scientists to see if cell-based therapies can someday become a viable pillar of medicine.

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