The adoptive transfer of virus-specific T cells has produced remarkable clinical results in patients with viraldisease. However, broader implementation of this therapy has been limited by the (i) prohibitive productioncosts, (ii) complexity of manufacture, (iii) prolonged time for preparation and product release, and (iv) therequirement for individualized, patient-specific products. Over the past 6 years we have systematicallyaddressed these problems: we simplified and refined our manufacturing technology, removed biohazardouscomponents and employed a new cell expansion platform using a gas permeable culture device whichpromotes the proliferation and survival of large cell numbers in a GMP-compliant closed system with minimaltechnician intervention. Finally, we have established the clinical benefit associated with the infusion of partiallyHLA matched virus-specific T cells that are prospectively generated and banked, making them available forimmediate ?off the shelf? use. Thus, with the purpose of moving beyond highly specialized academic centers we established a BaylorCollege of Medicine-affiliated company called ViraCyte with the goal of commercializing ?off the shelf? virus-specific T cells. Our product - Viralym-A - is a bank of T cell lines with specificity for Adenovirus, which, inimmunocompromised individuals, is responsible for a wide range of severe clinical syndromes includingpneumonia, hemorrhagic cystitis, nephritis, colitis, hepatitis, and encephalitis, resulting in death in 18-26% ofpatients. Our therapy is intended for the treatment of drug-resistant infections/disease, a condition that afflictsless than 200,000 persons in the United States and for which there is no standard of care. Thus, in the currentapplication we will test the safety and potential for anti-viral activity of Viralym-A in allogeneic hematopoieticstem cell transplant recipients. Success of this application will lay the foundation for a Phase IIb study toconfirm the efficacy of ?off the shelf? Viralym-A cells and facilitate market approval, thereby moving T celltherapy for Adenovirus into the public domain as a standard of care.
The immune system is comprised of specialized cells call ?T cells? that are able to recognize and eliminateopportunistic infections. Therefore; in healthy individual; although viruses can cause mild flu-like symptomsthese resolve spontaneously without medical intervention. However; when the integrity of the immune systemis compromised [e.g. in babies with genetic immune defects or as a consequence of a medical treatment (eg.after bone marrow transplant)] viral infections may cause severe symptoms in multiple organs; underscoringthe need for new and safe approaches to treat infections.Over the past 20 years our scientific team has utilized T cells that were generated in the laboratory to treatdrug-resistant infections and we have validated the safety and clinical benefit of this therapy in immunecompromised patients. However; despite our success this therapy has not moved beyond academic centersdue to the complexity associated with the preparation of T cells. Therefore; for the past 6 years our team hasworked diligently to streamline and simplify our cell production process; while maintaining the product integrity.Thus; we are now in a position to transition from an academic center to making this therapy a standard of care.With this objective in mind we established a company called ViraCyte and in the current application wepropose testing the safety of our cell product ? Viralym-A ? in bone marrow transplant patients with drug-resistant adenovirus infections. If successful; this work will provide crucial enabling data that will allow us tomove towards product approval by testing the clinical efficacy of Viralym-A in a phase IIb clinical trial. We areconfident in achieving our goal as the ViraCyte co-founders ? a unique combination of scientists; clinicians andsuccessful entrepreneurs - have a long-standing collaborative relationship and history of success.
