Custom Cocktail for Curing any Cancer: A Strategy for Destroying any Cancer without Harming the Patient

Special Article - Antisense Drug Research and Development

J Drug Discov Develop and Deliv. 2016; 3(1): 1020.

Custom Cocktail for Curing any Cancer: A Strategy for Destroying any Cancer without Harming the Patient

Summerton J*

Gene Tools, LLC, USA

*Corresponding author: Summerton J, Gene Tools, LLC, 1001 Summerton Way, Philomath, Oregon 97370, USA

Received: January 28, 2016; Accepted: April 27, 2016; Published: May 02, 2016

Abstract

Drugs for curing any cancer must completely destroy a patient’s cancer without harming the patient. To avoid harming the patient, precision-targeted drugs are used to inactivate RNA transcripts that are absent from the patient’s normal cells, but present in and essential to the viability of the patient’s cancer. Such “cancer-essential” transcripts are from embryo-active genes normally deactivated in adults, but reactivated in cancers, or from oncogenic viruses. For complete destruction of the patient’s cancer a “custom cocktail” of precisiontargeted drugs will target multiple cancer-essential transcripts found to be present in and essential to that patient’s cancer.

This paper describes: the basis for selecting targets in a patient’s cancer; selecting drugs for precisely inactivating those targets; and, assembly of a custom cocktail of such drugs designed to provide a safe and effective cure for that patient’s cancer. Preparation and use of a custom cocktail for a specific patient entails:

1. A biopsy of the patient’s cancer is sequenced to identify the canceressential transcripts present in that cancer.

2. With that information, assemble a cocktail of precision-targeted drugs effective against multiple cancer-essential transcripts found to be present in that patient’s cancer, and treat the patient with that custom cocktail.

Also described is a basis for possibly obtaining prompt and affordable regulatory approvals for such cocktails.

I believe this therapeutics strategy solves the central challenge in treating cancers: how to completely destroy any cancer without harming the patient. It also offers a direct and rapid route to a safe, effective, and affordable treatment for any cancer.

Keywords: Custom cocktails; Blocking cancer-essential transcripts

Introduction

Ideal cures for cancers should precisely attack and completely destroy any patient’s cancer - without damage to the patient. And such cures should be affordable. But two technical challenges have long foiled success in the quest for such ideal cures:

1. Cancer cells evolve from the patient’s normal cells and so both cell types share nearly all of the same genes, gene transcripts, and proteins. Thus it is very difficult to completely destroy the cancer cells without also damaging or killing the patient.

2. Each patient’s cancer differs significantly from all other cancers and so a safe and effective cure for each patient’s cancer will likely require a precisely-targeted custom treatment. But until recently the technologies required for crafting such custom treatments were not available, and it was widely assumed custom treatments could never be affordable.

However, since the year 2000 three new technical advances should now make it possible to provide patients with an affordable custom cancer treatment specific for their cancer.

a) One key advance is the huge reduction in the cost and great increase in the speed of sequencing the RNA transcripts present in a patient’s cancer (accessed by needle biopsy).

b) A second key advance is in computer informatics programs which now provide a fast inexpensive assessment of RNA sequence data which allows one to identify those RNA transcripts which are present in a patient’s cancer cells, but are absent from that patient’s normal cells. These “cancer-only” transcripts are primarily coded by genes which are active during embryogenesis, then deactivated before birth, but decades later reactivated due to mutation to form cancers. Precisely targeting such cancer-only transcripts is essential in order to avoid blocking transcripts present in and essential to the patient’s normal cells.

c) The third key advance is the availability of a family of drugs (Morpholinos) which can be targeted against any selected RNA transcript - designed just from knowledge of the sequence of the transcript to be targeted. For applicability against most or all cancers, such drugs: are easily and precisely targeted against any selected transcript; are completely stable in biological systems; are generally free of off-target effects; are deliverable to most or all tissues in the body; and, provide maximal versatility because they are effective in both the nucleus and the cytosol of cells.

The above technical advances open the door for development of personalized cancer treatments which: do not damage the patients; reliably destroy the cancers; and, allow precise treatment of any patient’s cancer. However, a final element is needed for development of actual drugs with these capabilities. That final element is a novel drug targeting strategy where the key elements are defined as follows and illustrated in (Figure 1).

Citation: Summerton J. Custom Cocktail for Curing any Cancer: A Strategy for Destroying any Cancer without Harming the Patient. J Drug Discov Develop and Deliv. 2016; 3(1): 1020. ISSN : 2471-0288