2016 Annual Report
2016 JP2MRI Achievements
It is my pleasure to inform you about the research progress at John Paul II Medical Research Institute in 2016. Our team has had a very productive year and we are confident that with your continued support we will build on our research advances in 2017. Thanks to your generous financial contributions this year, our Institute was able to obtain blood samples from patients with a variety of diseases, isolate their stem cells to form induced pluripotent stem cells and then differentiate these stem cells into a variety of specialized cells of the body that are being effected by their particular disease. We hope to now use these cells to advance drug discovery and hopefully find better therapies in the coming years.
Many of you will recall that you first supported our Institute in the summer of 2014 when the ALS Ice Bucket Challenge brought attention to the need for more and better ALS research. When you discovered that the national ALS Association was participating in embryonic stem cell research, you looked for an ethical medical research organization that has gone on the record to speak out against such research and found JP2MRI. We are pleased to report that thanks to your support in 2014 and 2015, our researchers have been able to differentiate induced pluripotent stem cells into neurons that are damaged by ALS and other neurodegenerative diseases such as Parkinson's disease and Muscular Dystrophy. Our Institute is now working to differentiate these neurons into motor neurons so that our researchers can study how they behave in patients who are ill and look for possible drugs that can treat or halt the progression of the disease. As you can imagine, this is a complicated research area that is going to take time and continued financial support to advance but we intend to continue the fight until there is a cure for these conditions.
This year our Institute also isolated induced pluripotent stem cells from donors and differentiated these stem cells into beating cardiomyocytes or heart cells. These heart cells will assist the Institute in studying various forms of heart disease and heart failure and will play a role in better drug therapies in the years to come. We also initiated a Rare Disease Research Program in February 2016 to bring more attention to the suffering of children afflicted with these incurable diseases and the high mortality rates they carry. While there are thousands of diseases classified as "rare diseases" and which impact millions of people worldwide, our Institute has started to focus on a few genetically based rare diseases that affect patients in the United States. So far in 2016, our researchers have isolated stem cells and differentiated them into induced pluripotent stem cells from children suffering from Gaucher's disease, Aplha -1 and Niemann Pick Type C. Our Institute is currently working on creating IPS cells from children suffering from Cystic Fibrosis, Duchenne Muscular Dystrophy, Alpha 1 Anti-Trypsin Deficiency and Mucopolysaccharidosis type I (MPS I). If you know someone who has been diagnosed with any of these rare diseases and they are interested in helping us with our research efforts, please direct them to our patient registry (www.jp2mri.org).
Our research success depends directly on you and your active participation. We cannot thank you enough for the generous donations you have already made and hope that you will continue to support our ethical research through your tax deductible financial contributions. However, just as equally important, our research depends on those of you who have either been diagnosed or know someone who has been diagnosed with a neurodegenerative disease or a rare disease to sign up on our patient registry. It is available on our Institute’s website: jp2mri.org. Once you sign up, a representative from our Institute will contact you when appropriate to discuss your willingness to provide the necessary blood or tissue samples our Institute needs to further our research studies. In most instances, thanks to the technology that our Institute has developed, we will only need a small blood sample from you when you visit your local doctor. Once donated, your blood sample will be shipped by mail to a processing facility within our Institute where your personal information will be de-identified and our researchers will work on developing the cell lines needed for testing. It is important to note that all information you provide on our patient registry is kept secure and confidential as required by HIPPA regulations and patient names and conditions are not revealed to third parties. We strongly encourage participation from all our supporters. Our Institute prides ourselves in answering any and all questions you may have promptly and they may be directed to us at info@jp2mri.org.
In June 2016, members of our Institute attended the International Society for Stem Cell Research (ISSCR) meeting that was held in San Francisco to present on some of our research findings in a joint abstract that was accepted by the organizers. This annual meeting is the premier event to meet with stem cell scientists from across the globe and to learn about the new advances being sought in stem cell research worldwide. Additionally, in October 2016, the Institute’s Founder Dr. Alan Moy presented an abstract at the 6th Annual Conference on Rare and Orphan Disease demonstrating the ability to create "disease in a petri dish" stem cell models from patients with genetic diseases. The Institute now has the technology to accelerate drug development for these diseases. Our abstract explained how "disease in a dish" stem cell models can be created from a simple blood sample from a patient. These "disease in a dish" models can then be used to screen large drug libraries to identify the most effective drugs that should be tested in clinical trials.
What is Ahead in 2017
In 2017, JP2MRI will continue to further our adult stem cell research to look for better treatments and cures for the diseases identified above. Additionally, our researchers will start focusing on new diseases that need our attention and which are directly tied to the research platform we have already built to study the earlier diseases. Our expanded research focus will include: Duchenne Muscular Dystrophy, Cystic Fibrosis, Alpha 1 Anti-Trypsin Deficiency and Mucopolysaccharidosis type I (MPS I).
Second Annual Rare Disease Month
Rare Disease Day is recognized every year on the last day of February. There are over 7,000 rare diseases and over 30 million Americans, most of whom are children, suffer from a rare disease. About 98% of rare diseases do not have any treatments or cures and 30% of those diagnosed with a rare disease will sadly die before the age of 5. Given these sobering statistics, our Institute believes that rare disease awareness deserves more than simply one day of recognition and therefore have chosen to dedicate all of February to fight for rare disease advances.
JP2MRI will be running various promotions and fundraisers in February 2017 to raise awareness and funds to help advance our rare disease research program. To learn how you or your organization can help, please send an email to info@jp2mri.org and add the subject line "Rare Disease Month."