Generation of T cells for therapeutic use

This technology is a method for generating large numbers of diverse, functional, naïve T cells using bone marrow cells from adult donors.

Unmet Need: Efficient and effective method of enhancing thymus function

Thymic dysfunction is a common condition that leads to immunodeficiency and a severe lack of functional T cells. Current treatment strategies for thymic insufficiency include administration of cytokines and growth factors to enhance thymus function and ex vivo production of T cells for infusion. However, these methods are cumbersome and inefficient. Improved technology in this field would have numerous therapeutic applications for patients with thymic insufficiency due to advanced age, chemo/radiotherapy treatment, immunosuppressive drug treatment, graft-vs-host disease, T cell-depleted hematopoietic stem cell transplant and HIV infection.

The Technology: Large-scale production of functional, naïve T cells

This technology proposes a method for producing large quantities of diverse, functional, naïve T cells for infusion into patients with thymic insufficiency. The in vivo generated T cells arise from bone marrow taken from the patient and become genetically compatible immune cells that are tolerant of the donor. In addition to being immunologically functional, the T cells are also rejuvenated – an increased percentage of the cells are “naïve”, which means that these cells are capable of mounting immune defenses against new targets once they are transferred into the donor patient. Overall, this technology has the potential to improve clinical outcomes for patients suffering from immunodeficiencies related to thymus hypofunction.

This technology has been validated with preclinical mouse models.

Applications:

• Treatment of patients with immunodeficiency arising from thymic insufficiency due to immunosuppressants, chemotherapy, or irradiation
• Treatment for complications of Type I Diabetes
• Production of T cells that are resistant to viruses such as HIV
• Production of T cells with desired specificities against tumors, viral antigens, or autoantigens
• Production of specific T cells, such as regulatory cells, that can treat autoimmune disease and treat/prevent graft rejection
• Production of mixed populations of T cells from two donor sources that are tolerant of one another, for use in rapid immune reconstitution in patients undergoing hematopoietic cell transplantation

Advantages:

• Efficient in vivo development of large numbers of functional T cells for clinical use
• Generation of diverse, naïve T cells
• Production of T cells with desired specificities, viral resistance, or of regulatory subtype

Lead Inventor:

Megan Sykes, M.D.

Patent Information:

Patent Status

Related Publications:

• Kalscheuer H, Danzl N, Onoe T, Faust T, Winchester R, Goland R, Greenberg E, Spitzer TR, Savage DG, Tahara H, Choi G, Yang YG, Sykes M. “A model for personalized in vivo analysis of human immune responsiveness” Sci Transl Med. 2012 Mar 14; 4(125): 125ra30.

Tech Ventures Reference:

• IR 2871, CU13146

• Licensing Contact: Jerry Kokoshka