Airways for Children

The primary goal is to bring a safe and effective therapy to children with severe large airway disease. Our intent is to implement all of the necessary steps for a successful new stem/progenitor cell-derived airway transplant for clinical trials in children within 4 years. Our team builds on first-in-human surgical success with a stem cell-based tissue engineered airway implant in a compassionate care case in a young adult and in a child. To this end, we will perform the necessary preclinical studies to support a successful FDA application within 4 years. We propose to use stem/progenitor cells from the patient to treat an extraordinarily difficult-to-manage health problem in children, namely large airway disease. In children this leads to collapse of tracheal cartilage causing severe airway obstruction that is life-threatening. It occurs in approximately 200 California children each year and the morbidity and mortality associated with this disease is very high. Approximately 25% of these young patients die before their first birthday. Treatment costs for these children are very high, and the familial and societal investments are substantially higher, although outcomes are consistently poor. The endpoint desired is normal airway and lung function and a normal quality of life. Our team aims to eliminate the need for repeated surgical interventions which are not necessarily successful, presently the standard of care for children with large airway obstruction. Bioengineered airway transplants that use the cells of the patients could be used in humans of all age groups and would not require lifelong, harmful anti-rejection medications.

In 2008 and 2010, a stem cell based, tissue engineered tracheal implant was successfully used by our team to save a young woman's and a child's life, respectively. These first-in-human studies emphasize that our goal is realistic and paves the way for clinical trials in children after carefully designed safety studies are completed for preclinical testing. Stem/progenitor cell-derived airway transplants that use the patient's cells have the clinical advantage of not requiring anti-rejection medications long-term. Our experience, to date, indicates such medication is not needed and this finding represents a scientific and clinical breakthrough in organ transplantation. While clear medical benefit was demonstrated in these proof-of-principle, compassionate care human cases, there is substantial work that must be done before considering such transplants for pediatric patients, and on a large scale, for adults. We address this challenge with our team approach and emphasize the synergism resulting from linking team members with expertise in a variety of scientific and medical disciplines to address this critical need. This new therapeutic approach could offer a tremendous benefit to children and patients in other age groups in the State of California that are in desperate need of new treatment options.