Gene therapy prolongs survival, slows disease progression in ALS mice

Scientists at the Salk Institute in La Jolla, California, report in the August 8 issue of Science that a novel gene therapy approach prolonged survival and slowed disease progression in mice with ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig's disease. ALS is a fatal neuromuscular disease characterized by the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, head-and-neck, and respiratory muscles. Plans for the first ALS patient trial utilizing this approach are underway.

Fred H. Gage, Brian Kaspar, and colleagues, injected an adeno-associated viral vector (AAV) to express a protein that promotes the survival of neurons into the limb muscles of mice. When delivered to the cell bodies of the neurons, this gene, called insulin-like growth factor-1 (IGF1), delayed disease progression and prolonged survival. The authors report that their results demonstrate substantial behavioral, functional, and pathological improvements in a clinically relevant mouse model of a motor neuron disease.

Says Fred Gage, "Adeno-associated viruses are retrogradely transported from the synapses in the muscle to the nucleus of the motor neuron inside the spinal cord, emphasizing the importance of basic science in developing rational therapies for disease."

Dr. Gage approached Project A.L.S. with the idea of trying this gene therapy approach in ALS a year-and-half ago. When data from the initial ALS studies proved promising, Project A.L.S. put Gage and Brian Kaspar together with Jeffrey D. Rothstein, an ALS researcher and clinician at Johns Hopkins University. The teaming of Gage with Rothstein is a prime example of the Project A.L.S. strategy: uniting the best basic scientists with those who can help translate that science into clinical application.

Adds Gage, "Project ALS has been impressive in recognizing and supporting fundamental science in what is now called translational science."

"I am proud and excited that this investigation may result in the first ever effective therapy for ALS. We've done it in the mice, now let's forge ahead aggressively and responsibly, and try it on people," says Project A.L.S. CEO, Jenifer Estess, who has had ALS for six years. Project A.L.S. is collaborating with scientists, industry, and the Food and Drug Administration toward the first human trial of AAV-IGF1.

Founded in 1998, by Jenifer Estess, her friends and family, Project A.L.S. is a non-profit 501(c)3 dedicated to finding and funding treatments and a cure for ALS. Project A.L.S. is a leading contributor to research in the areas of genetics, stem cells, gene therapy, accelerated drug testing, and disease pathways. Project A.L.S. recruits the most talented scientists across academic and industry lines to work together on the complicated problem of ALS. Therapeutic approaches to ALS may help those with the closely related diseases Parkinson's, Alzheimer's, and Huntington's diseases, and spinal cord injury.

For more information or to schedule an interview, please contact:
Patricia Harrington
Harrington Communications
212-581-0407

Valerie Estess
Director of Research
Project A.L.S.
212-627-2568

Project A.L.S.
511 Avenue of the Americas, PMB #341, NYC 10011
tel: 212-969-0329 fax: 212-337-9915
email: info@projectals.org
website: www.projectals.org

---

---