SACRAMENTO, Calif., July 10, 2019 (GLOBE NEWSWIRE) -- Spina bifida, which refers to a condition where the spinal cord is improperly formed in utero leaving a section of the neural tube open, has been around since before the Dark Ages. In fact, skeletons found in Morocco that date back to 12,000 BC show evidence of the disease. Up until the past decade, this diagnosis often meant lifelong childhood paralysis for infants born with the disease and a series of cognitive, mobility, urinary and bowel disabilities depending on the severity of the malformation.
Between 1,500 and 2,000 babies born in the US each year are diagnosed with spina bifida. Due to the severity of complications caused by spina bifida, scientists have studied the disease for centuries in the hopes of finding a cure. Up until recent years, those efforts have been largely unsuccessful and treatment has consisted of surgery within the first few days after birth to limit the most negative effects of the disease.
Armed with the knowledge that the damage to the spinal cord worsens during the normal progression of pregnancy, medical researchers in the field of fetal medicine have worked diligently to find ways to repair the damage to the fetal spinal cord prior to birth. One of the pioneers in this field is Dr. Diana Farmer, who was the first female fetal surgeon in the world and is the chair of surgery and surgeon-in-chief of UC Davis Children’s Hospital.
Farmer was part of a project funded by the National Institutes of Health to study whether fetal surgery, performed in-utero prior to birth, would be beneficial to patients diagnosed with the most severe form of spina bifida. Over a seven-year period, patients were randomly assigned to either receive fetal surgery between 22 and 26 weeks gestation or to follow the traditional path in which surgery was completed shortly after birth. The results of the study, which were released in 2011, were overwhelmingly positive. Compared to the babies who had surgery after birth, less than half of those who underwent fetal surgery required a shunt to drain fluid build-up in the brain. In addition, the changes in the shape of the skull that are commonly found with spina bifida were much less severe among the babies who had fetal surgery. Unfortunately, mobility problems remained an issue among both test groups.
Buoyed by the success of the study, Farmer and a team at UC Davis then began researching whether stem cells could repair spina bifida before birth and improve neurologic functioning. The results they recorded after administering stem cell therapy to a group of lambs were amazingly successful: 67 percent of the lambs that received stem cell therapy were able to walk independently. In 2017, the team collaborated with the UC Davis William R. Pritchard Veterinary Medical Teaching Hospital to try to alleviate the symptoms of two 10-week-old bulldog puppies born with spina bifida who were unable to walk or even wag their tails. They treated the dogs with a combination of surgery and canine stems cells. Two and a half months later, the puppies were walking, running and playing.
The work done by Diana Farmer and the UC Davis research team got the attention of the California Institute for Regenerative Medicine, which awarded the team a $5.66 million grant in November 2018. The grant will allow the team to perform final preparations and testing needed for FDA approval so that they can begin a clinical trial to treat babies diagnosed with spina bifida with a combination of fetal surgery and stem cell therapy beginning in 2021.
Talitha McGuinness, executive director of the national nonprofit Fetal Health Foundation (FHF), is excited about the work being done at the UC Davis Fetal Care and Treatment Center. In 2006, McGuinness helped found FHF, which provides information about fetal syndromes, connects families diagnosed with a syndrome to specialist centers and to other families who have experienced a similar diagnosis, and awards research grants to study promising fetal treatments. Reflecting on the growth in the field of fetal medicine, McGuinness says, “When we began the Foundation, there were only a handful of maternal fetal treatment centers in the US and a small number of proven fetal syndrome treatments. Today, there are more than 100 centers, with expert maternal fetal specialists and researchers around the globe working on treatments like those being done at UC Davis.”
The explosive growth in fetal medicine has far-reaching implications. There are hundreds of known fetal syndromes that cause serious complications or death in 800,000 pregnancies each year. The knowledge that scientists gain while researching treatments for one type of fetal syndrome is often transferrable to many other syndromes. According to McGuiness, “It’s inspiring to think that the work being done at UC Davis to repair the spinal cord and limit the effects of spina bifida may ultimately help not just those babies but also babies suffering from lung, heart and other fetal syndromes.”
About UC Davis Fetal Care & Treatment Center
UC Davis Fetal Care and Treatment Center is home to a highly-specialized, comprehensive and multidisciplinary fetal diagnosis and therapy center, providing on-site fetal surgery for treating conditions before birth. For more information, visit fetalcare.ucdavis.edu.
About The Fetal Health Foundation
The Fetal Health Foundation (FHF) is a national nonprofit focused on empowering and supporting expectant parents by providing information, access to world class maternal fetal medical experts, financial support, connections and counseling. In addition, FHF funds research, increases awareness, and serves as an outlet for leading medical information pertaining to fetal conditions and syndromes. To learn more visit www.fetalhealthfoundation.org.
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