Monthly Archives: May 2013

Public release date: 2-May-2013 [ | E-mail | Share ]

Contact: John Ascenzi Ascenzi@email.chop.edu 267-426-6055 Children's Hospital of Philadelphia

Medical researchers have manipulated human stem cells into producing types of brain cells known to play important roles in neurodevelopmental disorders such as epilepsy, schizophrenia and autism. The new model cell system allows neuroscientists to investigate normal brain development, as well as to identify specific disruptions in biological signals that may contribute to neuropsychiatric diseases.

Scientists from The Children's Hospital of Philadelphia and the Sloan-Kettering Institute for Cancer Research led a study team that described their research in the journal Cell Stem Cell, published online today.

The research harnesses human embryonic stem cells (hESCs), which differentiate into a broad range of different cell types. In the current study, the scientists directed the stem cells into becoming cortical interneuronsa class of brain cells that, by releasing the neurotransmitter GABA, controls electrical firing in brain circuits.

"Interneurons act like an orchestra conductor, directing other excitatory brain cells to fire in synchrony," said study co-leader Stewart A. Anderson, M.D., a research psychiatrist at The Children's Hospital of Philadelphia. "However, when interneurons malfunction, the synchrony is disrupted, and seizures or mental disorders can result."

Anderson and study co-leader Lorenz Studer, M.D., of the Center for Stem Cell Biology at Sloan-Kettering, derived interneurons in a laboratory model that simulates how neurons normally develop in the human forebrain.

"Unlike, say, liver diseases, in which researchers can biopsy a section of a patient's liver, neuroscientists cannot biopsy a living patient's brain tissue," said Anderson. Hence it is important to produce a cell culture model of brain tissue for studying neurological diseases. Significantly, the human-derived cells in the current study also "wire up" in circuits with other types of brain cells taken from mice, when cultured together. Those interactions, Anderson added, allowed the study team to observe cell-to-cell signaling that occurs during forebrain development.

In ongoing studies, Anderson explained, he and colleagues are using their cell model to better define molecular events that occur during brain development. By selectively manipulating genes in the interneurons, the researchers seek to better understand how gene abnormalities may disrupt brain circuitry and give rise to particular diseases. Ultimately, those studies could help inform drug development by identifying molecules that could offer therapeutic targets for more effective treatments of neuropsychiatric diseases.

In addition, Anderson's laboratory is studying interneurons derived from stem cells made from skin samples of patients with chromosome 22q.11.2 deletion syndrome, a genetic disease which has long been studied at The Children's Hospital of Philadelphia. In this multisystem disorder, about one third of patients have autistic spectrum disorders, and a partially overlapping third of patients develop schizophrenia. Investigating the roles of genes and signaling pathways in their model cells may reveal specific genes that are crucial in those patients with this syndrome who have neurodevelopmental problems.

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Turning human stem cells into brain cells sheds light on neural development

CHICAGOA 2-year-old girl born without a windpipe now has a new one grown from her own stem cells, the youngest patient in the world to benefit from the experimental treatment.

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Hannah Warren has been unable to breathe, eat, drink or swallow on her own since she was born in South Korea in 2010. Until the operation at a central Illinois hospital, she had spent her entire life in a hospital in Seoul. Doctors there told her parents there was no hope and they expected her to die.

The stem cells came from Hannah's bone marrow, extracted with a special needle inserted into her hip bone. They were seeded in a lab onto a plastic scaffold, where it took less than a week for them to multiply and create a new windpipe.

About the size of a 3-inch tube of penne pasta, it was implanted April 9 in a nine-hour procedure.

Early signs indicate the windpipe is working, Hannah's doctors announced Tuesday, although she is still on a ventilator. They believe she will eventually be able to live at home and lead a normal life.

"We feel like she's reborn," said Hannah's father, Darryl Warren.

"They hope that she can do everything that a normal child can do but it's going to take time. This is a brand new road that all of us are on," he said in a telephone interview. "This is her only chance but she's got a fantastic one and an unbelievable one."

Warren choked up and his wife, Lee Young-mi, was teary-eyed at a hospital news conference Tuesday. Hannah did not attend because she is still recovering from the surgery. She developed an infection after the operation but now is acting like a healthy 2-year-old, her doctors said.

Warren said he hopes the family can bring Hannah home for the first time in a month or so. Hannah turns 3 in August.

Read more here:
Windpipe made from stem cells implanted in 2-year-old girl

By Alexandra Sifferlin, TIME.com

updated 9:06 AM EDT, Wed May 1, 2013

Hannah Warren, 2, recovers in a post-op room at the Children's Hospital of Illinois in Peoria.

STORY HIGHLIGHTS

(TIME.com) -- Hannah Warren was born without a trachea but now has one made from plastic fibers and a stew of her own stem cells.

The 2-year-old Korean Canadian has spent every day of her life in intensive care, kept alive by a tube that substituted for the windpipe that was supposed to connect her mouth to her lungs. But nearly a month after her transplant, the toddler is mostly breathing on her own and is responding to doctors and nurses.

The surgery, pioneered by Dr. Paolo Macchiarini, director of the Advanced Center for Translational Regenerative Medicine at the Karolinska Institute in Stockholm, was only the sixth performed in the world, and Hannah was the youngest patient and first to receive the transplant in the United States.

The procedure was approved by the FDA as an experimental operation for patients with very little hope of survival; being born without a trachea is fatal in 99% of cases.

TIME.com: Stem cell therapies may cure chronic conditions

Macchiarini performed the nine-hour operation on April 9 at the Children's Hospital of Illinois after carefully creating the windpipe using stem cells from Hannah's bone marrow that were saturated over a matrix of plastic fibers shaped into a tube.

Originally posted here:
Toddler gets new windpipe from her own stem cells

By Alexandra Sifferlin, TIME.com

updated 9:06 AM EDT, Wed May 1, 2013

Hannah Warren, 2, recovers in a post-op room at the Children's Hospital of Illinois in Peoria.

STORY HIGHLIGHTS

(TIME.com) -- Hannah Warren was born without a trachea but now has one made from plastic fibers and a stew of her own stem cells.

The 2-year-old Korean Canadian has spent every day of her life in intensive care, kept alive by a tube that substituted for the windpipe that was supposed to connect her mouth to her lungs. But nearly a month after her transplant, the toddler is mostly breathing on her own and is responding to doctors and nurses.

The surgery, pioneered by Dr. Paolo Macchiarini, director of the Advanced Center for Translational Regenerative Medicine at the Karolinska Institute in Stockholm, was only the sixth performed in the world, and Hannah was the youngest patient and first to receive the transplant in the United States.

The procedure was approved by the FDA as an experimental operation for patients with very little hope of survival; being born without a trachea is fatal in 99% of cases.

TIME.com: Stem cell therapies may cure chronic conditions

Macchiarini performed the nine-hour operation on April 9 at the Children's Hospital of Illinois after carefully creating the windpipe using stem cells from Hannah's bone marrow that were saturated over a matrix of plastic fibers shaped into a tube.

Excerpt from:
Girl gets windpipe from her stem cells

By Lindsey Tanner

A two-year-old girl born without a windpipe now has a new one grown from her own stem cells, the youngest patient in the world to benefit from the experimental treatment.

Hannah Warren has been unable to breathe, eat, drink or swallow on her own since she was born in South Korea in 2010. Until the operation at a central Illinois hospital, she had spent her entire life in a hospital in Seoul. Doctors there told her parents there was no hope and they expected her to die.

The stem cells came from Hannah's bone marrow, extracted with a special needle inserted into her hip bone. They were seeded in a lab onto a plastic scaffold, where it took less than a week for them to multiply and create a new windpipe.

About the size of a three-inch tube of penne pasta, it was implanted April 9 in a nine-hour procedure.

Early signs indicate the windpipe is working, Hannah's doctors announced Tuesday, although she is still on a ventilator. They believe she will eventually be able to live at home and lead a normal life.

"We feel like she's reborn," said Hannah's father, Darryl Warren.

"They hope that she can do everything that a normal child can do but it's going to take time. This is a brand new road that all of us are on," he said in a telephone interview. "This is her only chance but she's got a fantastic one and an unbelievable one."

Warren choked up and his wife, Lee Young-mi, was teary-eyed at a hospital news conference Tuesday. Hannah did not attend because she is still recovering from the surgery. She developed an infection after the operation but now is acting like a healthy two-year-old, her doctors said.

Warren said he hopes the family can bring Hannah home for the first time in a month or so. Hannah turns three in August.

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Stem cells grow toddler a new windpipe