2017 Annual Report

It is our pleasure to inform the public about the research progress at John Paul II Medical Research Institute during 2017. 2017 was a very challenging year at the Institute due to a significant decline in donations. Consequently, the Institute had to prioritize its research in a way that would have the greatest impact for all patients suffering from diseases that are part of our four therapeutic priorities: 1) chronic neurodegenerative disorders; 2) rare diseases; 3) cancer; and 4) unmet or underperformed chronic diseases. Despite the financial challenges, the Institute was able to accomplish some very important cutting-edge research to create hope for treating incurable diseases, particularly those that involve the central nervous system.

As a reminder to our donors, the Institute has created one of the largest portfolios of adult stem cells in the world. These cells include adult stem cells from bone marrow, fat tissue and many types of stem cells from postnatal tissue like cord blood, umbilical cords and the placenta. The Institute has also contributed to the development of the next generation of induced pluripotent stem cells (iPSC). iPSC are stem cells that have the same capabilities as embryonic stem cells. iPSC are created by genetic manipulation of an adult cell without having to use and destroy a human embryo. However, the fundamental problem with iPSC has been that the process has required the use of viruses and oncogenes (cancer genes). In 2017, the Institute co-developed the next generation of iPSC without using viruses and oncogenes, which will provide a safer cell therapy than prior iPSC approaches. This new approach makes the use of embryonic stem cells unnecessary for therapeutic applications. This work was published and tracked at the 97th percentile among the 9 million articles published in 2017. As a follow up study, the Institute further modified its iPSC approach to work with cord blood and peripheral blood from children with Cystic Fibrosis (CF) and an adult with Alpha-1 Antitrypsin Deficiency (A1ATD), a genetic cause of COPD. It is now possible to develop more efficient means to carry out drug development for patients with genetic diseases. This latter scientific work has been submitted for publication consideration.

The Institute has also developed expertise in synthetic biology. Genes are composed of DNA sequences which are transcribed into messenger RNA, which are then translated into a protein. This process has historically been very laborious, taking months to years to artificially synthesize a gene. The Institute now uses synthetic biology to produce synthetic genes faster, better and at much lower cost. The Institute has made synthetic genes of Alpha-1 antitrypsin, CFTR (the gene mutated in Cystic Fibrosis) and other peptides and proteins that have therapeutic value. Ultimately, these genes have to be packaged into mammalian or human cells to produce a therapeutic protein. The Institute has also co-developed efficient methods

to produce these proteins in industrial mammalian cells at higher yields than conventional approaches. With these new technologies, it is now possible for the Institute to develop biologics for enzyme replacement therapy for rare diseases; vaccines, diagnostics and other biologic therapeutics.

Furthermore, the Institute has now combined its synthetic biology expertise with its stem cell expertise to enhance the potency of adult stem cells. The Institute is now codeveloping technologies that will allow adult stem cells to produce and deliver biologics that could effectively treat a variety of CNS diseases. These dual biologic-stem cells may enhance the potency of adult stem cells to treat a variety of conditions such as Alzheimer's disease, Parkinson's, stroke, traumatic brain injury, ALS and lysosomal storage diseases.

Thanks to our donor's financial contributions, the Institute was able to achieve these scientific milestones. We hope to now use these cells to advance drug discovery and hopefully find better therapies in the coming years. As you can imagine, this is a complicated research area that is going to take time and continued financial support to advance. However, we intend to continue the fight until there is a cure for these conditions.

Our research success depends directly on the active participation of our donors. Unfortunately, the financial support of this Institute is supported by

a very small group of committed donors. The Institute is one of the very few Catholic medical research organizations that conducts medical research consistent with pro-life values. Despite the Institute's unique and strict pro-life position, there remain many Catholics and Catholic healthcare institutions that are not aware of our existence. Furthermore, it is disconcerting that many of these individuals and organizations are unknowingly supporting medical research organizations that support the use of morally illicit tissues such as embryonic stem cells and aborted fetal tissue, which conflicts with Catholic teaching. Additionally, many of these secular organizations spend between 15-30 percent of their expenditure on research activity. In contrast, the Institute typically devotes over 50 percent to medical research expenditures. The Institute has had to adapt by finding ways to reduce the cost of medical research by 75 percent.

Since the Institute operates on a very small budget, it does not have resources to wage a national fundraising campaign. We must rely on our donors to inform others through word of mouth or by social media. We encourage our donors to notify their local schools, churches, civic groups and hospitals about our existence and the good work that we do. There are several ways in which individuals can donate to our mission. First, individuals can purchase apparel from our web site that support our research. Second, donors can donate online through our web site or mail donations to our administrative office. Third, churches, schools and civic groups can conduct joint venture fundraising events in which half of the raised money can be retained for local charities. To our knowledge, we are the only private medical organization that is willing to share its proceeds. Lastly, we request that individuals consider the Institute in their estate planning for charitable donations. To learn how you or your organization can help, please send an email to info@jp2mri.org.

In addition to our research mission, we also provide educational information on biotechnology for Catholics and on the ethics of biotechnology. For example, we published an article for the Catholic Medical Association on the issue of human-animal chimeras, which was published in the Linacre Quarterly. Further, the Institute continues to speak to Catholic lay and religious groups around the country on seminal biotechnology issues and medical ethics.

We cannot thank our donors enough for the generous donations they have already made and hope that you continue to support our ethical research through tax deductible financial contributions in the years to come.

Sincerely,

Alan Moy, MD

Founder and Scientific Director

Jay Kamath, JD

CEO

© 2019 John Paul II Medical Research Institute.