What Regenerative Medicine Research is the John Paul II Medical Research Institute Conducting for Lung Disease? Part 2

Producing a Safer Pluripotent Stem Cell to Produce Lung Cells for COPD Patients

by Dr. Alan Moy, November 16, 2018

The pharmaceutical industry has failed to innovate and develop treatments for patients with emphysema, while spending billions of dollars in research and development over decades for nothing but drugs. As one of my colleagues used to say, "The pharmaceutical industry is shooting for mediocrity and failing miserably when it comes to developing treatments for lung disease." In fact, GalaxoSmithKline, the largest pharmaceutical company in the respiratory space, appears to have thrown up their hands in defeat by cutting back on their research and development for COPD (see post). This explains why their stock has remained flat for over two decades, while other pharmaceutical company's stock is rising. As mentioned in Part 1 of our prior post, stem cell therapies typically act by offering cell protection and cell repair. Yet, they likely will not be sufficient to replace the alveolar cells that are lost in diseases like COPD. Thus, we will also need a pluripotent stem cell that is devoid of ethical controversy; has much lower neoplastic (tumor forming) risk; and can differentiate into lung cells.

Notwithstanding that embryonic stem cells carry ethical controversy, they are difficult to source and pose a neoplastic risk if the cells are maintained in their pluripotent state. Induced pluripotent stem cells (iPSC) were introduced in 2006 by Shinya Yamanaka, who subsequently became a Nobel Laureate for his discovery, by genetically reprogramming skin cells into pluripotent stem cells using 4 genes (Sox2, Oct4, Klf4 and c-Myc) delivered by a retrovirus. Similarly, Dr. James Thompson's team developed iPSC by using a different set of genes (Sox2, Oct3/4, Nanog, Lin28). Both versions of iPSC will cause neoplasms. To make a long story short, the cause for the neoplasms are due to the oncogenes (Nanog, c-Myc and Lin28). Thus, to create a safer source of iPSC, a process is needed that is free of virus and free of these oncogenes or cancer-causing genes.

In 2017, a team of scientists at Cellular Engineering Technologies, the John Paul II Medical Research Institute, the University of Iowa, University of Pittsburgh and Western New England University published the first report of a technique to produce iPSC in neonatal skin cells without the need for viruses and oncogenes (Published in Future Science Open Access). More recently, Cellular Engineering Technologies, the John Paul II Medical Research Institute and the University of Iowa, received notice that a follow up paper was accepted for publication in the scientific journal Regenerative Medicine. The scientific work showed how this new iPSC technology could be universally applied to cord blood and to peripheral blood in patients with Cystic Fibrosis and Alpha 1 Antitrypsin Deficiency, the latter is a genetic cause of emphysema. The paper further demonstrates how iPSC can differentiate into neural cells, cardiac cells and lung cells. We now have the largest pipeline of virus-free and oncogene-free iPSC from postnatal stem cells. This now provides, for the first time, safer and universal iPSC for the treatment of many chronic diseases, including lung disease like COPD. More importantly, the formula for producing these iPSC can be easily transferred into a good manufacturing practice (GMP) operation, which is required for clinical applications by the Food and Drug Administration and other regulatory bodies around the world.

The next post will describe the challenges experienced and research the Institute accomplished for converting iPSC into lung cells.

Based on historic timelines and cost, the Institute estimates that it will take 5 years and 10 million dollars (2 million dollars per year) to complete the FDA-required preclinical research to launch the first regenerative medicine clinical trial in COPD. According to the World Health Organization, there are 65 million individuals around the world that live with moderate to severe COPD. If only 100,000 of these 65 million were to commit to donating $20 a year, this goal would be met. That translates to sacrificing 2 packs of cigarettes per year.

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