Identifying FDA Approved Drugs to Reverse Dry AMD
MentorJohn Hulleman, PhD The University of Texas Southwestern Medical Center
To employ readily available, FDA-approved small molecules and test their potential for reversing AMD-associated pathologies.
Specific Aim 1: Miniaturized “disease in a dish model” for AMD using porcine retinal cell cultures will be used to test a large drug library. Drugs will be evaluated for their capability to i) reverse AMD pathology and ii) promote retinal pigment epithelium health. Specific Aim 2: Drugs performing well in Aim 1 will be utilized in a mouse model of AMD pathology. After determining the proper dosage for these lead drugs, extensive evaluation by imaging, histology, and high-end microscopy for their disease reversal potential will be conducted.
Our “disease in a dish” culture system emulates advanced dry AMD pathology in humans. This miniaturized platform enables us to test drugs unbiasedly for their potential to reverse disease. Rescue intervention to reverse disease progression is a more probable real-life necessity, as most often, diagnoses are made when the disease is advanced and where preventative measures prove futile.
Successful completion of this study will identify drug(s) that are already deemed safe for human use and are capable of rescuing retinas from dry AMD-associated pathologies. As there are no known treatment so far for dry AMD, this discovery can be a breakthrough for future AMD treatments and prognoses.