Is the United States Effectively Advancing Adult Stem Cell Research? Part 2

1. Is the United States Effectively Advancing Adult Stem Cell Research?Part 2 Disclosures Alan Moy MD Director of John Paul II Medical Research Institute Founder and CEO of Cellular Engineering Technologies Inc. Pulmonary Associates of Iowa City Adjunct Faculty, College of Engineering, University of Iowa

2. Milestones of JP2MRI to Date

3. JP2MRI instrumental is coordinating patient recruitment and tissue procurement. CET, a private biotech company located at Oakdale, manufactures commercial adult stem cells for research market. In 2 years created the largest commercial supply of adult stem cells and progenitors (GLP). Developing normal and patient/disease-specific iPS cells. Do not conduct embryonic stem cell research or cloning. Products sold around the world to academia and pharmaceutical companies. Not approved for therapy or clinical research. Cellular Engineering Technologies, CET

4. Human Adipose Mesenchymal Stem Cells – Applications of Various Differentiation Media Hepatocyte like cells differentiated from HAMSC. Periodic acid--Schiff staining. Neuron like cell stained for early neuronal marker Beta III Tubulin HAMSC differentiated into adipose tissue, bone and cartilage. All cells differentiated using media developed by CET. Adipose tissue is stained with Oil O Red, bone is stained with Alizarin Red and cartilage is stained with toluidine blue.

5. Conversion to Neural Progenitor Cells • Human embryonic stem cell conversion into neural progenitors is inefficient and takes 3 weeks. • Adult stem cell conversion into neural progenitors more efficient and occurs within 24 hours. Tyrosine hydroxylase

6. Inducible Pluripotent Stem Cells (IPS) • Reprogramming adult human cells by genetic reprogramming via exogeneous expression of pluripotent transcriptional factors • Produce embryonic-like pluripotent stem cells without ethical controversy and yield patient and disease-specific stem cells.

7. Conversion to Cardiac Progenitors • Conversion of pluripotent stem cells into cardiac progenitors. • Exhibit automaticity and contractility.

8. Institute and CET Develop Patient-Specific Somatic Stem Cells • In December of 2008, the Institute helped Peyton Hadley, 11, and his sister, Kayla, 8, who both have Niemann-Pick Type C, a rare and fatal neurodegenerative disease due to cholesterol processing disturbance. • Harvested and grew tens of millions of their stem cells from a teaspoon of their fat within 2 weeks. • Used by scientists in Canada and NIH to screen drugs that may control Niemann-Pick Type C disease. • Cell model of dementia and atherosclerosis.

9. Capital Campaign to Build the John Paul II Medical Research Institute • Proposal to create a 16,000 square foot facility at the BioVenture in Coralville, IA. • Will house small number modular wet lab space, small scale GMP facility, small scale microfabrication laboratory, small animal laboratory facility, administrative offices and clinical treatment rooms. • Estimated startup cost is:~10 million • Estimated five year cost is:~ 30 million.

10. JP2MRI Poised to Address the Hurdles in Stem Cell Therapeutics • JP2MRI is singularly poised to develop the most comprehensive adult stem cell program. • 3 cord blood-derived stem cells. • 1 cord tissue stem cell. • 2 placenta-derived stem cell. • Bone marrow-derived stem cell. • Fat derived stem cell. • Progenitor cells (liver, nerve, muscle, bone, cartilage, fat). • Predict that JP2MRI will be one of the leaders in IPS technology. • Private, academic and industry relationships in place. • Animal safety and efficacy data - FDA requirement. • Academic collaborations. • Capital not science is the rate limiting problem.

11. BioVenture Center

12. Value of JP2MRI to Iowa • Research Center for • regenerative medicine • cancer treatments • orphan disease • Economic development • Advance status and expertise of hospitals.

13. Value of JP2MRI Globally • Expand consortium in adult stem cell research. • Lower the barriers of applied research. • Return high public investment to society.

14. Educational Mission • Objectives • Advance adult stem cell research in academia. • Increase the work force of adult stem cell scientists. • Advance pro-life bioethics. • Execution Plan • Train academic faculty, postdoctoral fellows and students in adult stem cell research and bioethics. • Provide sabbatical opportunities for academic faculty. • Provide out-reach opportunities for undergraduate and graduate students in adult stem cell research and bioethics (e.g. internships and fellowships).

15. Strategic Plan Principles • ASCR needs to be systematically well organized on a national level. • Pro-life individuals need to be active participant in science rather than be bystanders. • While we can still advocate our pro-life position, we’re going to have to find common ground between both political parties - looking to find cures and treatments for patients. • Science and economics, not political advocacy, will determine the end.

16. Catholic Challenge • Catholics/America are losing the battle on ASCR. • ASCR is woefully under funded-HHS report indicated US government needs to invest over 2 billion dollars. • Embryonic stem cell research community have successfully framed the debate as helping patients-pro-life. • Embryonic stem cell scientific community has a monopoly on influence in government and in the media. • Catholics are never going to change NIH agenda and secular academia. • Catholics have restricted effort to educational advocacy without a constructive alternative (e.g. missions, pregnancy crisis centers, churches, schools and hospitals). • Not a critical mass of adult stem cell researchers in US. • Catholics have to take ownership of this issue- financially support adult stem cell research and offer alternative to ESCR: examples

17. www.jp2mri.org