2018 Annual Report
It is our pleasure to inform you about the research progress at John Paul II Medical Research Institute during 2018. While our year end fund raising goal was lower than we had hoped, the Institute still managed to make significant research advances which are highlighted below.
As a reminder to our donors, the Institute has four therapeutic priorities: 1) neurodegenerative disorders; 2) rare diseases; 3) cancer; and 4) unmet or underperformed chronic diseases. Since our establishment in 2006, JP2MRI 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. We hope that this new approach will make the use of embryonic stem cells unnecessary for therapeutic applications.
How This New Milestone in iPSC Technology Will Be Deployed to Advance the Institute's Mission
This year, JP2MRI's focus was to try and raise the necessary funding to transition our stem cell technology from the laboratory to the clinic. Our goal was to raise at least $200,000 to cover the costs associated with performing the required FDA testing to show that our stem cells are safe and efficacious when introduced into animal models. While the Institute has not yet met our financial objective, we did perform animal studies with our collaborators at the University of Iowa and evaluated whether our iPSC results in lower tumor formation than traditional iPSC approaches. Our research showed that mice injected with our iPSC formed a much lower tumor risk than traditional iPSC. However, greater financial support is essential to perform the necessary preclinical regulatory requirements before our iPSC can be used in clinical trials.
There has been a global interest to make drug development more efficient and cost effective by creating patient and disease-specific iPSC biobanks. Current biobanks have been limited in their abilities due to several shortcomings. First, the initial iPSC technology was based on viruses. Second, current iPSC biobanks use oncogenes or cancer genes during their creation. Viruses and oncogenes distort the predictability of drug safety and efficacy. Thus, there was a need to produce better patient-specific iPSC free of oncogenes and viruses. The Institute recently co-authored a paper that was published in the highly regarded stem cell journal, Regenerative Medicine, which demonstrated how virus-free and oncogene-free iPSC were produced from cord blood and from peripheral blood from patients with Cystic Fibrosis and alpha one anti-trypsin deficiency, a genetic cause of Chronic Obstructive Pulmonary Disease (COPD). This publication was cited as a top cord blood publication in Hematopoiesis News during 2018. The Institute is now poised to assist the scientific community with better iPSC technology in accelerating drug development. Towards that end, we urge our supporters to sign up on the Institute's patient registry to help us with future clinical trials. The Institute's registry, and the medical information shared on it, is secure, private and kept confidential.
Closing Statement of Gratitude and Future Support from Our Donors
Because of the financial support from our donors, JP2MRI was able to achieve these important scientific milestones. We intend to continue developing our technology and plan to use our novel iPSC 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. Our research success depends directly on the active participation of our donors. Unfortunately, the government has passed some new tax regulations this year that might impact our overall fundraising efforts as a result of how tax deductions are calculated. We have previously outlined these changes in our newsletter and ask you to review and discuss this matter with your accountant in planning out your year end and future tax deductible donations. Given our organization's size and budget, most our expenditures are applied towards actual research as opposed to marketing and administration. We rely on our donors to spread the word of the good work that the Institute is uniquely accomplishing through word of mouth or by social media. We encourage each of you to notify your local schools, churches, civic groups and hospitals about our research and need for support.
There are several ways to support our mission. First, individuals can purchase apparel from our web site to help us increase our visibility. Second, you can make a charitable donation online through our website 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 learn how you or your organization can help, please send an email to firstname.lastname@example.org. Lastly, individuals can consider donating to the Institute through their estate planning efforts.
We cannot thank our donors enough for the generous donations that you have made and hope that you continue to support our ethical research in the years to come.
Alan Moy, MD
Founder and Scientific Director
Jay Kamath, JD