Sloan-Kettering

CIRM interviews Lorenz Studer: 2017 recipient of the Ogawa-Yamanaka Stem Cell Prize [Video]

/ Todd Dubnicoff / 1 Comment

For eight long years, researchers who were trying to develop a stem cell-based therapy for Parkinson’s disease – an incurable movement disorder marked by uncontrollable shaking, body stiffness and difficulty walking – found themselves lost in the proverbial wilderness. In initial studies, rodent stem cells were successfully coaxed to specialize into dopamine-producing nerve cells, the type that are lost in Parkinson’s disease. And further animal studies showed these cells could treat Parkinson’s like symptoms when transplanted into the brain.

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Lorenz Studer, MD
Photo Credit: Sloan Kettering

But when identical recipes were used to make human stem cell-derived dopamine nerve cells the same animal experiments didn’t work. By examining the normal developmental biology of dopamine neurons much more closely, Lorenz Studer cracked the case in 2011. Now seven years later, Dr. Studer, director of the Center for Stem Cell Biology at the Memorial-Sloan Kettering Cancer Center, and his team are on the verge of beginning clinical trials to test their Parkinson’s cell therapy in patients

It’s for these bottleneck-busting contributions to the stem cell field that Dr. Studer was awarded the Gladstone Institutes’ 2017 Ogawa-Yamanaka Stem Cell Prize. Now in its third year, the prize was founded by philanthropists Hiro and Betty Ogawa along with  Shinya Yamanaka, Gladstone researcher and director of the Center for iPS Cell Research and Application at Kyoto University, and is meant to inspire and celebrate discoveries that build upon Yamanaka’s Nobel prize winning discovery of induced pluripotent stem cells (iPSCs).

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(L to R) Shinya Yamanaka, Andrew Ogawa, Deepak Srivastava present Lorenz Studer the 2017 Ogawa-Yamanaka Stem Cell Prize at Gladstone Institutes. Photo Credit: Todd Dubnicoff/CIRM

Studer was honored at the Gladstone in November and presented the Ogawa-Yamanka Stem Cell Prize Lecture. He was kind enough to sit down with me for a brief video interview (watch it below) a few minutes before he took the stage. He touched upon his Parkinson’s disease research as well as newer work related to hirschsprung disease, a dangerous intestinal disorder often diagnosed at birth that is caused by the loss of nerve cells in the gut. Using human embryonic stem cells and iPSCs derived from hirschsprung patients, Studer’s team has worked out the methods for making the gut nerve cells that are lost in the disease. This accomplishment has allowed his lab to better understand the disease and to make solid progress toward a stem cell-based therapy.

His groundbreaking work has also opened up the gates for other Parkinson’s researchers to make important insights in the field. In fact, CIRM is funding several interesting early stage projects aimed at moving therapy development forward:

We posted the 8-minute video with Dr. Studer today on our official YouTube channel, CIRM TV. You can watch the video here:

And for a more detailed description of Studer’s research, watch Gladstone’s webcast recording of his entire lecture:

The Fatal Flip: How Nerve Cells go from Healthy to Cancerous

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Every gene in the human genome has a job to do. One such gene, called Merlin, prevents cells from dividing out of control and forming into tumors. A so-called ‘tumor suppressor,’ Merlin has proven to be essential to maintaining healthy cell division. Scientists knew that without Merlin, nerve cells grew uncontrollably, often leading to tumors and a type of inherited cancer called neurofibromatosis type 2 (NF2).

Scientists have uncovered the mechanism whereby an absence of the gene called Merlin causes normal nerve cells to turn rogue.

Scientists have uncovered the mechanism whereby an absence of the gene called Merlin causes normal nerve cells to turn rogue.

Now, scientists have uncovered the mechanism that causes an absence of Merlin to transform nerve cells into rogue, tumor-producing cells—helping shed new light on how the smallest genetic shifts—even in just one gene—can have an impact on the normal pattern of growth and development of cells.

Reporting in the latest issue of Cancer Cell, a joint team from the Sloan-Kettering Institute for Cancer Research and Plymouth University Peninsula Schools of Medicine and Dentistry have found that without Merlin a chemical pathway, called the Hippo pathway, switches on. This, in turn, spurs tumor cell growth in nerve cells.

Professor Dr. Oliver Hanemann of Plymouth University and one of the study’s senior authors, explained in a July 22 news release:

“We have known for some time that the loss of the tumor suppressor Merlin resulted in the development of nervous system tumors, and we have come tantalizingly close to understanding how this occurs.”

This research advance is especially important in the case of NF2, a condition for which there are limited treatment options. Current treatments usually involve a combination of surgery and radiation, but rarely is the cancer fully eradicated.

“By understanding the mechanism [of Merlin], we can use this knowledge to develop effective drug therapies—in some cases adapting existing drugs—to treat patients for whom current therapies are limited and potentially devastating.”

Stem cell biology has also proven essential when shining a light on or explaining the complexities surrounding cancer’s underlying mechanisms, including the notion of cancer stem cells—a concept that has gained increasing support in recent years. To learn more about how CIRM-funded scientists are harnessing stem cells to understand—and develop treatments for—cancer, check out our 2009 Spotlight on Cancer Stem Cells as well as our Brain Tumor fact sheet.