Medical treatments Use Patients’ Own Stem Cells

A girl born without a windpipe has received a new one grown from stem cells—not embryonic stem cells, but her own.

According to Lindsey Tanner’s April 30th Associated Press article “Canadian-Korean girl, 2, is youngest ever to get windpipe from her own stem cells “, Hannah Warren could not breathe, eat, drink or swallow on her own and had spent her life in a Seoul hospital.

When Hannah struggled to breathe at birth, doctors ran a breathing tube down her throat to her lungs. They discovered she lacked a windpipe, a birth defect that affects about 1 in 50 000 children worldwide. Doctors informed the Warrens that Hannah could not live long with the tube in her esophagus but that they could do nothing more.

The Warrens learned about Dr. Paolo Macchiarini’s work with stem cell-based tracheas at the Karolinska Institute in Stockholm, Sweden. Dr. Macchiarini had been involved in fourteen windpipe operations using patients’ own stem cells: five using manmade scaffolds and nine using scaffolds made from cadaver windpipes. Hannah’s parents could not afford the cost of the procedure at Dr. Macchiarini’s facility in Stockholm.

After much planning, Dr. Mark Holterman, a pediatric surgeon at Children’s Hospital of Illinois in Peoria who had met the Warrens during a business trip to South Korea, arranged to have the procedure performed at the Hospital at no cost to the family. Ms. Tanner tells us that “Part of OSF Saint Francis Medical Centre, the Roman Catholic hospital considers the operation part of their mission to provide charity care, but also views it as a way to champion a type of stem-cell therapy that doesn’t involve human embryos, the surgeons said. The Catholic Church opposes using stem cells derived from human embryos in research or treatment.”

Stem cells extracted from bone marrow in Hannah’s hip were seeded in a lab onto a plastic scaffold and quickly multiplied to form a new windpipe. On April 9th, a team led by Dr. Macchiarini implanted Hannah’s windpipe, making her the youngest patient ever to undergo this treatment.

Hannah remains on a ventilator to help her breathe and cannot eat normally yet, but she no longer has a tube in her mouth. Her doctors said that early signs suggested the windpipe is working and she should be able to live at home and lead a normal life. Since she has a windpipe and can breathe more normally now, doctors expect her larynx to grow and function properly, and speech therapists will help her learn to talk. As she grows, Hannah will probably need a new windpipe in about five years.
According to her doctors, the stem-cell technique has been used to make other body parts and has the potential to be used in treating other birth defects and childhood diseases. Bladders and urethras have been grown using similar methods, and in 2012 a girl in Sweden received a vein created using her own stem cells and a cadaver vein.

Dr. Anthony Atala, Director of the Wake Forest Institute for Regenerative Medicine, said scientists hope one day to create solid organs such as kidneys and livers using this method.

Stem-cell research has also led to a multiple sclerosis (MS) treatment that appears to have eliminated the disease in the 24 study participants, according to Derek Spalding and Charlie Fidelman’s April 2nd Ottawa Citizen article “Ottawa doctors behind breakthrough MS report.”
About 13 years ago, a team of doctors at the Ottawa Hospital began the study for patients with severe MS symptoms that didn’t improve with drug therapy.

Neurologist Dr. Mark Freedman and bone-marrow transplant expert Dr. Harold Atkins planned to wipe out the patients’ immune systems, restart them with a transplant of the patients’ own bone marrow, and wait for MS to redevelop. Their hope was that the team would be able to identify one of the signals that trigger MS and so find a way to treat the degenerative disease. But the disease did not reappear.

The procedure is not without risk. The pill form of the drug, Busulphan, led to the death of one patient in an early phase of the trial. A year later, a new intravenous version of the drug was used and found to be much safer for patients.

Study participants reported no relapses and all improved dramatically, according to a study on the treatment by a team of MS researchers at the Montreal Neurological Institute and the Université de Montréal. Moreover, findings published in the Annals of Neurology journal stated that magnetic resonance imaging (MRI) showed no new brain lesions.
Dr. Amit Bar-Or, a neurologist at the Montreal Neurological Institute and professor at McGill University, was the study’s main investigator. He noted that it’s unknown whether this treatment would stop other types of MS and that MS treatments involving bone-marrow stem-cell transplants have not been approved outside of clinical trials.

Results notwithstanding, the researchers are not calling this treatment  a cure.

Given that about 55 000 to 75 000 people in this country have MS, Canada has one of the world’s highest rates of the disease.

These amazing treatments have come through sources of stem cells that do not raise ethical problems. Embryonic stem  cell research on the other hand requires the destruction of a living human embryo, a human life at a very early stage.

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