| |
As Chief of Hematology/Oncology and Director of Translational Research at Children’s, David A. Williams, MD, works with children suffering from a rare condition called severe combined immunodeficiency disease, or “boy in the bubble syndrome.” Previous gene therapy trials, in which healthy genes are inserted into patients’ cells using a viral vector (a virus that has been altered to carry human DNA) boosted the immune system of these patients, but also caused leukemia in many.
Dr. Williams has run multiple trials to test human gene therapy for a range of diseases. Drawing on his experience, he partnered with researchers in Germany and London to create a new vector that would be safer for these patients.
Dr. Williams’ vector is safer in preclinical and animal studies for treating two genetic forms of the condition. His improved vectors are far enough along to test within a year, given sufficient funding— a significant hurdle, considering that designing, testing and producing each vector costs more than $350,000. Significant costs also go into implementing the necessary clinical trials. Going forward, his work will provide a foundation for developing ever safer gene therapy approaches to treat many life-threatening diseases.
|
|
| |
