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

This patent describes a way to make an in-vitro diagnostic assay to test a patient's bone marrow cells to see if they're suitable for treating heart failure caused by reduced blood flow. The assay uses the characteristics of the cells to determine if they meet the necessary criteria for achieving a therapeutic effect. The patient's cells are also tested to see if they meet specific criteria to make sure the treatment will be effective. The therapy is delivered based on the number of specific cells, proteins, and genes in the patient's body that make them a good candidate for the treatment. The treatment is also delivered based on the efficiency of the delivery route and method, as well as the patient's likelihood of achieving the targeted therapeutic effects. The therapy can be made at the patient's bedside using a combination of various techniques to minimize contamination.

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 mesenchymal stem cells or time course of action preceding rejection for CSCs hold true, is that each lot of cells will come from a different universal donor or a combination of donors and they will not be identical.

Therefore, to a lesser extent, as in the first approach above, one should expect variations in response and in beneficial outcomes with each donor or batch lot and at present, one cannot presume these variations to be insignificant or predictable.

While this has value for advancing the status of our scientific knowledge and adds significant rigor to cell based therapies, it also adds enormously to the complexity of therapies when simpler approaches described herein are possible.

However, while there is consensus about the safety of these cells, efficacy results from similar clinical studies in the same patient population appear to be inconsistent.

However, it is noted that higher dosage does not necessarily correlate to greater efficacy.

Variations between patients in cell potency and in cell dosage delivered may also result in failed therapeutic efficacy trials.

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