Methods of measuring potential for therapeutic potency and defining dosages for autologous cell therapies

Abstract

Autologous bone marrow cells (BMC) are transplanted to a heterologous site in a patient after a sample of the patient's BMC has been tested and found to have a phenotypic profile which meets minimum criteria for transplantation. The phenotypic profile may be obtained by screening a sample of bone marrow cells (BMC) from the patient for the phenotypic profile, such as a CD profile, the phenotype profile may be assessed to determine the likelihood that the BMC will be suitable for transplantation to the heterologous tissue site without enriching particular phenotypic population(s) of the BMC.

Description

CROSS-REFERENCE TO RELATED APPLICATIONS[0001]This application claims the benefit of Provisional Application No. 61 / 896,450 (Attorney Docket No. 29181-707.101), filed Oct. 28, 2013, the entire content of which is incorporated herein by reference.BACKGROUND OF THE INVENTION[0002]Introduction to Pharmacogenomics and Personalized Medicine.[0003]Pharmacogenomics is the field of selecting the right drug for the right patient at the right time. Its genesis was the belief that the pharmaceutical industry would identify and match patients with specific genetic phenotypes and gene expression profiles who would respond to specific therapeutic medicines, providing a new era of personalized medicine. This would inherently enhance the drugs therapeutic profile, resulting in more successful clinical trial results as the selected patients would have already been identified as potential responders to the therapy. An example of a successful drug that has been developed using the concept of pharmacoge...

AI Technical Summary

Benefits of technology

[0018]For example, the in vitro diagnostic assay for autologous bone marrow derived mononuclear cells for the treatment of heart failure of ischemic etiology may be specified based on the CD profile of the constituent cells; (3) the development of in vitro diagnostic assays in which a small amount of tissue can be assessed to see if the source tissue meets defined thresholds of cellular, protein and/or genetic characteristics that increase a patient's likelihood of achieving a targeted therapeutic dosage when these same cells are harvested, concentrated, and re-administered at a later time point; (4) the delivery of an effective dosage based on the number of cells from a specific population, proteins and/or genes that is present in a patient that would qualify the patient as a responder to the autologous therapy; (5) the delivery of an effective therapeutic dosage based on the efficiency of the delivery route and the delivery method inclusive of the delivery system used to infuse the effective therapeutic dosage into a patient; (6) the delivery of an effecti...

Problems solved by technology

This may and oftentimes does include significant and expensive clinical care decisions, and these tests therefore have value in both mitigating the risks of unnecessary and potentially dangerous procedures and reducing medical costs.

This is a critical problem in the field.

These types of extensive manipulation, cell culture handling and analysis significantly raise the costs of these proposed therapeutic candidates.

Not only do the manipulations themselves have significant labor and complex reagents and materials costs, but they also have shipping and quality control costs on each patient's tissues, resulting in a separate manufacturing lot for each and every dosage form for one particular patient.

Each assay on a potential autologous therapeutic dosage formulation and each step in its handling add significantly to the costs of the therapy—which in turn means that the developer and manufacturer will ultimately have to charge a higher price for their product.

The problem with this therapeutic approach even if the theoretical assertions on the immune privilege status of...

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