GERN announced (5/19/10) positive data on its orally available small molecule telomerase activator, TAT153, in an animal model of idiopathic pulmonary fibrosis (NYSEARCA:IPF). The data show that administration of TAT153 increased telomerase activity in the lung tissue, reduced inflammation, preserved functional lung tissue, slowed disease progression and attenuated loss of pulmonary function. This strong correlation between the onset of pulmonary fibrosis, including IPF, and telomere shortening in aged patients provides rationale for telomerase activation as a potential therapeutic approach for the disease.
Telomerase activator can affect fibrotic disease progression in a model system and the data indicate that telomerase activation may be a useful therapeutic modality in IPF. Currently, there are no drugs that reduce the fibrotic process in lung disease or other organs. This is a very large unmet medical need (T Okarma, PhD, MD – CEO)
The study used a standard mouse model of IPF. The fibrotic process and accompanying deterioration of lung function were induced by bleomycin administration into the trachea. TAT153 was given twice daily for 3 weeks. Progression of pulmonary fibrosis in the animals was assessed by lung function tests, quantification of inflammatory cells and collagen deposits in the lungs. Inflammation precedes and is associated with fibrosis of the lung. Study animals showed a 40% decrease in inflammatory cells in the TAT153-treated group. Histology showed less extensive fibrosis preserving a greater proportion of functional lung tissue in treated animals. The study included measurements of lung function in TAT153-treated animals and controls. The TAT153-treated animals showed a 30% increase in lung compliance (elasticity) and a 30% decrease in airway resistance (mechanical factors which limit the access of inspired air to the alveoli, or air sacs) compared to controls, demonstrating a significant attenuation of functional deterioration. These positive effects of TAT153 in the mouse model of pulmonary fibrosis were associated with an approximately 2 fold increase in telomerase activity in lung tissue samples.
Idiopathic Pulmonary Fibrosis (IPF) is a chronic, progressive, and usually fatal lung disease, characterized by inflammation and scarring, or fibrosis, of the lung. Patients have an impaired ability to process oxygen and a reduced lung volume and experience coughing and shortness of breath. IPF affects 100,000 people in the US and there are between 30,000 and 40,000 new cases each year. The median survival time from diagnosis is 2 to 5 years. The age of onset is between 40 and 70, with 2/3’s of patients older than 60 at presentation. Incidence rates are expected to be driven higher by an aging population. There are currently no FDA approved treatments for IPF.
The data was presented at the American Thoracic Society 2010 International Conference in New Orleans, LA by Geron collaborator Dr. Claude Jourdan Le Saux from the University of Texas Health Science Center at San Antonio.