FDA Approves Phase I Stem Cells for Babies with Hearing Loss

There's a new FDA approved cord blood stem cell safety study to be done on deaf children age 6 weeks to 18 months who acquired sensorineural hearing loss (nonsyndromic deafness). This is a year long study.

Children’s Memorial Hermann Hospital and Cord Blood Registry® (CBR) are launching the first FDA-approved, Phase I safety study on the use of cord blood stem cells to treat children with sensorineural hearing loss.

The study, which will use patients’ stem cells from their own stored umbilical cord blood, is the first-of-its-kind, and has the potential to restore hearing. This follows evidence from published laboratory studies that cord blood helps repair damaged organs in the inner ear.

The year-long study will follow 10 children, ages 6 weeks to 18 months, who have sustained post-birth hearing loss. Children who are deaf as a result of a genetic anomaly or syndrome are not eligible. To ensure consistency in cord blood stem cell processing, storage, and release for infusion, CBR is the only stem cell bank providing clients for the study.

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Researchers will obtain and process the patients’ stored cord blood for treatment. The cells then will be given to the patients via IV infusion, and patients will be observed for several hours in the hospital.

Patients will return to the hospital to repeat all tests except the MRI at one month and one year, and all tests with an MRI at six months.

“This study is exciting because it might offer a non-surgical option for some children with profound loss,” Linda Baumgartner said. “More importantly, this is the first treatment with the potential to restore normal hearing.”

The new Phase 1 study already began in April 2011 and will end April 2015 although the collection on final study results will end in April 2014. This study will use stem cells from cord blood saved at a cord bank after a baby is born. Stem cells from the deaf baby's cord blood will be done via IV transfusion back into the child. Parents whose deaf children qualify for the trial study will decide whether to try the new procedure or not.

Since this news came out it didn't take long for an outcry in the form of a petition for culturally deaf people and others to decry the IV transfusion stem cell study as:

....inhumane and unethical and condemn this hospital, corporation, other collaborators and the FDA for treating our Deaf infants in this experiment as their guinea pigs.

The original petitioner went so far as to claim such procedure as dangerous and that it carries "long lasting risks" but never elaborated on exactly what those "long lasting risks" are. However, there is a similar but previous safety study using cord blood stem cells on children with cerebral palsy age 1 to 12 years old that began in January 2010. Final data collection on outcome ends in February 2012 which is next month.  Ironically, there appears to be no objection from any particular protest group for this IV transfusion safety study for babies and older on reversing their cerebral palsy condition.

The MCG team follows on the heels of a pilot study at Duke University which is exploring if cord blood can be readily applied to infant’s with palsy like symptoms in the first two weeks after birth. Both teams are looking to verify the anecdotal evidence that cord stem cells can be used to ‘cure’ CP children. If successful, the work at MCG and Duke University may provide parents with hope that storing a child’s cord blood could be an effective shield against cerebral palsy.

Cord blood IV transfusions have been done in children and adults.

To be safe, most cord blood transplants done so far have been in children and smaller adults. Researchers are now looking for ways to use cord blood for transplants in larger adults.

This first ever FDA approved study on hearing restoration (sensorineural hearing loss) for deaf children describes the study:

Acquired sensorineural hearing loss is characterized by a loss of functioning hair cells in the Organ of Corti, with greater hair cell loss correlating with more severe hearing impairment. Children with sensorineural hearing loss experience difficulty developing normal language which usually leads to poor academic and social development. Currently, there are no reparative therapeutic options available, and treatments are designed to augment the diminished function of the injured Organ of Corti.


Pre-clinical data suggest progenitor cell infusions may enhance intrinsic repair mechanisms in the Organ of Corti which may restore hair cells. This treatment could ultimately lead to hearing improvement. Human umbilical cord blood (hUCB) is an available, autologous, stored progenitor cell population available for potential therapeutic use. The primary objective of this study is to determine the safety of autologous hUCB infusion in children with acquired hearing loss. The secondary objective is to determine if functional, physiologic and anatomic outcomes are improved following hUCB treatment in this patient population.

This may turn out to be a promising result since it says that pre-clinical data suggest that such a procedure may restore hair cells thus lead to hearing improvement. This is obviously a much more natural process, if proved successful, involving the baby's own stem cells from the baby's umbilical cord (cord blood) could ultimately turn out to be much better option than cochlear implants. In fact, if successful would shorten the prediction time scale to seveal years on a reversing sensorineural hearing loss instead of a few decades away. Over a year and half a ago in my blog I commented that in 2004 a prediction was made that an application for hearing loss rather than a cure could be at least twenty years away (or by 2024) by Dr. Rubel. In another article in 2004 Dr. Rivolta said a cure could be possible in 10 to 15 years away (by 2014 to 2019). In 2009 in the Department of Otolaryngology University of Miami Ear Institute's newsletter quoted believing they could be 10 years away (or 2019) from a cure at the earliest possible timeline. But most scientists seem to concur that a cure or a very applicable treatment on reversing hearing loss would be seen within their (the scientists') lifetime...and that's not very far off folks.