A 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.
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.
"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.
"It's going to be amazing for us to finally be together as a family of four," he said. The couple has an older daughter.
Only about one in 50,000 children worldwide are born with the windpipe defect. The stem-cell technique has been used to make other body parts besides windpipes and holds promise for treating other birth defects and childhood diseases, her doctors said.
Hannah's parents had read about Dr. Paolo Macchiarini's success using stem-cell based tracheas but couldn't afford to pay for the operation at his centre, the Karolinska Institute in Stockholm. So Dr. Mark Holterman helped the family arrange to have the procedure at his Peoria hospital, bringing in Macchiarini to lead the operation. Children's Hospital waived the cost, likely hundreds of thousands of dollars, Holterman said.
Macchiarini has been involved in 14 previous windpipe operations using patients' own stem cells -- five using man-made scaffolds like Hannah's but in adults; and nine using scaffolds made from cadaver windpipes, including one in a 10-year-old British boy.
Similar methods have been used to grow bladders, urethras and last year a girl in Sweden got a lab-made vein using her own stem cells and a cadaver vein.
Scientists hope to eventually use the method to create solid organs, including kidneys and livers, said Dr. Anthony Atala, director of Wake Forest University's Institute for Regenerative Medicine. He said the operation on Hannah Warren "is really showing that the technique is workable."
Holterman said Hannah will likely need a new windpipe in about five years, as she grows.
"I asked her, 'Is it good?"' he said, "and she immediately nodded her head.
Some words from our team here at Provia Labs:
"This is an exciting example of how regenerative medicine is real today. We are only going to see more and more cases like this, where the use of stem cells will provide life-altering and life-saving benefits to patients. It is also an excellent illustration of the value of autologous stem cells - that is, the patient's own stem cells. Hannah will not need to take immunosuppressant drugs for the rest of her life, along with the many side effects associated with their use."
-Peter C. Verlander, PhD
Chief Scientific Officer, Provia Laboratories, LLC
"This type of therapy, where a scaffold is populated by your own cells, is becoming more of a reality. The potential for transformative medical procedures is present in our own stem cells from a variety of sources and her surgery is the beginning of more widespread treatments."
-Joseph C. Laning, PhD
Scientific Advisor, Provia Laboratories, LLC
"Having lost someone important in my life due to the critical, but severely dangerous regimen of anti-rejection drugs required after a donor transplant, I am deeply inspired and excited to see the emergence of therapies that benefit from one’s own stem cells."
Chief Executive Officer, Provia Laboratories, LLC
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