Stem cells for Retinitis Pigmentosa

Retinitis Pigmentosa (RP) is a group of genetic disorders, rare (1 in 4,000 people worldwide), that involve the loss and breakdown of rod and cone cells in the retina. RP is an inherited disorder due to harmful mutations in any of more than 50 different genes. There are three different effects of this mutations. The first one, the mutation is so severe that the body cannot make the protein that gene codes for. The second one is where the mutation changes the code in the gene and in result a toxic protein is made. The final one means the mutation altered the code leading to an abnormal protein that doesn’t work. All three lead to damage of the photoreceptors. At the beginning stages of the disease, people suffer from night blindness and later a more progressive loss of peripheral vision. These symptoms begin during childhood, although the progression varies depending on the gene affected and the mutation involved in the cause most patients with RP will lose most of their sight.

Currently, most people have to learn how to live with the loss of vision as there are only a few available experimental treatments to ameliorate the symptoms and slow the progression of the disease such as treatment with Vitamin A palmitate, cell based therapies, or now, just recently, the possibility of gene therapy.

Cell based therapies have demonstrated that either the stem cell injected replace the damaged cells or secrete factors that help cells with survival. We focus on utilizing autologous adipose derived stem cells (ADCs) with localized injection performed by a licensed ophthalmologist as well as a mannitol supplemented stem cell IV for better results.