WASHINGTON – The American Society of Gene & Cell Therapy this week honored four researchers as Outstanding New Investigators in the sciences of gene & cell therapy.
“These investigators are just establishing their independent careers, and each one has pioneered approaches to regulation of gene expression and treatment of disease by molecular therapeutic approaches,” said John J. Rossi, PhD, ASGCT Advisory Council chairman. “The Society wants to recognize these new investigators for their important contributions and encourage them to maintain their focus on gene and cell based therapies for disease.”
Alessandra Biffi, MD, San Raffaele Telethon Institute for Gene Therapy, Milan, recently began clinical testing of a stem cell therapy for Lysosomal Storage Disorders, a collection of inherited metabolic disorders affecting young children and usually resulting in death during infancy. Biffi’s research of hematopoietic stem cell transplants shows promise for improved, safe and effective treatment of these disorders.
Kevin V. Morris, PhD, Scripps Research Institute, La Jolla, CA, uses RNA for targeted control of gene expression, turning genes “on” or “off” as desired. These discoveries help explain how genes are controlled at the transcriptional level by RNA as well as offer insights into a new target approach to controlling gene expression for therapeutic gain.
“Such superlative control allows for minimized off-targeting with the ability to specifically regulate those genes involved in disease, such as HIV-1 and cancer,” Dr. Morris said. “The applications of our work go well beyond our model systems and can be applied to targeting any gene involved in virtually any malady.”
Bakhos A. Tannous, PhD, Massachusetts General Hospital, Charlestown, MA, developed a novel Gaussia luciferase (Gluc) enzyme that can be used to assess brain tumor responses to cell therapy, through a non-invasive blood test. He is currently developing other variations of the lucifarase for additional tumor monitoring.
“One of the limitations in cell therapy is that there is no current method to monitor survival of the cells upon implantation. The Gluc blood assay allows sensitive and real-time monitoring of cells’ survival and proliferation,” Dr. Tannous said. “This recognition gives me the encouragement and faith to continue my research hoping to make a difference in the field.”
Charles P. Venditti, MD, PhD, National Human Genome Research Institute, Bethesda, MD, studies methylmalonic acidemia (MMA), an inherited metabolic disorder that causes methylmalonic acid to accumulate in body fluids until they reach toxic levels. MMA is associated with severe metabolic instability, seizures and strokes, developmental delays, pancreatitis and kidney disease.
"In our first gene therapy experiments, we were able to demonstrate a high efficacy of a gene therapy to restore enzyme function in MMA cells in the laboratory," Dr. Venditti said. "…The award has served to highlight the potential that methylmalonic acidemia, and by extension, other organic acidemias, will emerge as fruitful candidate disorders for the application of gene and cellular therapies. I believe such therapies offer hope to the many families and patients who struggle to live a normal life, and that someday we will have better treatments for MMA."
“This group of recipients is impressive in their focus and determination to carry forward their important findings in gene and cell therapy,” added Rossi. “Each investigator has a different approach, but they are all focused and on the track to success in their respective disciplines.”
The Outstanding New Investigator program recognizes scientists new to gene and cell therapy – nominees must be within seven years of their first full-time position and must be conducting original research that advances the field. Award recipients receive a cash prize and complimentary registration to the ASGCT Annual Meeting. They each presented their work during a special plenary session at the meeting.
The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, www.asgct.org.
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