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​What Regenerative Medicine Research is the John Paul II Medical Research Institute Conducting for Alpha 1 Anti-Trypsin Deficiency? Part 4

A Cell Therapy Treatment to Replace Weekly IV Infusions

by Dr. Alan Moy, November 20, 2018

Since November is COPD Awareness month, it would be appropriate to discuss a genetic condition called Alpha 1 Anti-Trypsin Deficiency (A1ATD). A1ATD is a genetic cause of emphysema due to a lack of alpha 1 anti-trypsin (A1AT) protein. The protein is produced in the liver and secreted into the blood stream and travels to the lung where its therapeutic action takes place. A1AT inhibits inflammatory proteins that cause emphysema. A1ATD results is a deficient level of A1AT in the bloodstream because the liver is unable to produce and secrete the protein into the blood stream. Thus, A1ATD is caused by an imbalance between A1AT and pro-inflammatory molecules which cause emphysema. Every patient who is diagnosed with COPD should be tested for this genetic disorder.

In addition to standard treatment for COPD, patients with A1ATD are treated with replacement therapy of the A1AT protein. A1AT is currently manufactured by the pharmaceutical industry by purification of this protein from pooled plasma donors. Treatment is intravenously administered weekly because only a very small percent of the A1AT penetrates lung tissue where the drug activity takes place. The manufacturing process for producing A1AT is laborious and time consuming and the long-term cost to the healthcare system is quite expensive. For patients, receiving weekly injections is a hardship.

The requirement for weekly infusions raises the fundamental question: Is there a less expensive and more efficient way to deliver A1AT to the lung for an extended period? Further, could delivery of A1AT to the lung be coupled with an additional regenerative medicine therapy?

The Institute is conducting research to develop a stem cell therapy that has been genetically modified to synthesize and secrete A1AT. The Institute has identified a postnatal stem cells to serve as cell therapy. This stem cell has been genetically modified to synthesize and secrete a human A1AT. The goal would be to deliver this dual biologic-cell therapy to the lung. We hope that this would provide a continuous level of A1AT in the lung. There would be several advantages over the current approach. First, the treatment would eliminate weekly infusions. Second, the therapy would potentially last longer and perhaps could be permanent. Third, the long-term healthcare cost would be considerably lower. Lastly, a cell therapy could offer additional cell protection and cell repair benefits than the current approach.

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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