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Quarterly Updates

These are the kinds of quarterly updates we secure for our funding partners so that they feel connected to their projects and know what impact their funding hs creating.

Type I Diabetes, Dr. Denise Faustman, Harvard Medical School, Funded by Friends United for Juvenile Diabetes Research

As you will see from the reports, The research team is now done with enrollment into Phase I with the long term follow-up blood sampling of enrolled patients. The researchers, led by Dr. Denise Faustman at Harvard, hope to have the data submitted to the biostatistics center by October/November and then a paper submitted sometime thereafter. They have had no calls from the data safety monitoring board which means they have the go ahead for Phase II human clinical trial from the FDA once they have raised sufficient money for Phase II. The primary goal of Phase I was to validate in a double blinded study that researchers can accurately detect auto reactive immune cells in people with long standing diabetes because finding these cells will be the key to conducting the Phase II human clinical trial that will be the first human clinical type 1 diabetes trial since insulin and glucose. It is all very exciting and philanthropic support has been are only avenue for the development of this clinical protocol since repurposing these generic drugs will not make a profit for any company, but it is a very inexpensive way to rapidly get drugs to people with disease.

Lung Cancer, Dr. Jeff Borgia, Rush Medical Center, Funded by LUNGevity Foundation and Judy Hirsch Foundation

Two separate projects are being funded, both to create tests for detection of lung cancer. The first project is for staging disease when a patient is diagnosed. I the second quarter of this study, the research team discovered several new targets that have improved the accuracy of the blood test to identify nodal status in NSCLC patients that are surgical candidates and may help guide treatment strategies once fully validated. In the second project, designed to provide early detection screening of patients at high risk for lung cancer, the tests can distinguish patients with lung cancer from non-cancer "high risk" individuals with other lung conditions. The research team used the tests on samples from 83 patients with lung cancer, 40 patients with Chronic Obstructive Pulmonary Disorder (COPD) category, and 40 patients with a smoking history without cancer or a lung disorder. A manuscript will be submitted on this work early in July, 2009. Statistical analyses are begin done that could lead to a human clinical trial very soon.

Lung Cancer, Dr. Sreeneth Sharma, Mass General Hospital, Funded by LUNGevity Foundation and Judy Hirsch Foundation

The Mass General research project is combining several generic FDA approved drugs with a new treatment drug to make the new drug work for more patients for a longer period of time. The research has already created a clinical trial that is enrolling lung cancer patients in less than 1 year since we started-THAT IS IMPACT! Because of our ability to locate and validate high patient impact projects, LUNGevity has approved another $100,000 of research funding for 2009, and we are reviewing the final projects right now.

Multiple Sclerosis, Dr. Douglas Feinstein, University of Illinois Medical Center, Funded by Grant Healthcare Foundation

Initial studies were done to provide a proof of concept that increasing brain levels of Noradrenaline (NA) could provide benefit in the mouse model of multiple sclerosis . To ensure translational potential, the research team treated the mice after they became ill, rather than before they became ill, to better model what would occur in human treatment. The combined results of the initial studies showed that treatment of ill mice with the NA precursor L-DOPS (which works similarly to the compound L-DOPA well known for its ability to treat Parkinson's patients) stabilized the clinical symptoms (e.g. the control mice continued to get worse but the test mice did not get worse). In the 2nd qtr, the researchers tested an alternative method of increasing brain NA levels, by using an a second drug that prevents the cells from reducing NA levels. In contrast to DOPS, we found that this treatment did not effect clinical signs. The researchers then did a combination study using a moderate dose of DOPS plus a high dose of the NA inhibitor, and found a significant reduction in clinical symptoms. In view of that finding, in the 3rd and 4th quarters the researchers plan to confirm that DOPS together with an NA inhibitor can be of benefit in EAE, and carry out molecular studies to begin to understand how this may be occurring. It is apparent that the road to discovery is not direct; however the researchers now have clear means to reduce the clinical symptoms in the mouse model, the major goal of this project; and the further studies will hopefully confirm this initial finding and provide the basis for a full grant to NIH.

Multiple Sclerosis, TCNL Staff (Kaczmarek, Danilov, Tyler), University of Wisconsin, Funded by the Rockford Chapter of PFC

Our 9 patients human clinical trial was completed at this world renown TCNL research center creating treatments that help individuals regain lost brain and body function. TCNL scientists developed a device that sends mild electric impulses through the tongue to stimulate critical areas of the brain. They combine this brain stimulation with physical activity to help patients regain walking, balance, vision, hearing, sleep and other body functions. ALL PATIENTS IN THIS STUDY RECOVERED SIGNIFICANT FUNCTION. The first prototype portable devices arrived in the test lab this week. This research could also help patients Parkinson's Disease or trauma such as whiplash injury.

Blood Cancer, Dr. Luis Porrata, Mayo Clinic, Funded by the Brinson Foundation, Plunkett Foundation, Rockford Chapter of PFC

The Brinson Foundation, the Plunkett Family Foundation and our Rockford Group have supported this Mayo Clinic blood cancer transplant human clinical trail. After 17 months over 80 patients have been enrolled. THIS PROJECT HAS THE POTENTIAL TO INCREASE THE SUCCESS RATE OF THESE BONE MARROW TRANSPLANTS FROM 20% TO 80%! By the fall of this year we should have the first data that show this effect, less that two years after the research started!

Myelodysplastic Syndrome Initiative, Funded by the Sherman-Pincus Funding Group

Three new projects were initiated in June 2009.

1)Dr. Azra Raza, St. Vincents Comprehensive Cancer Center-Dr. Raza's research is looking at the genetics of MDS patient samples to determine what repurposed drugs might help

2)Dr. Amit Verma, Albert Einstein Medical Center-Dr. Verma's project is testing a repurposed drug for MDS patients

3)Drs. Seth Corey and Olga Frankfurt, Northwestern University Medical Center-. This project is adding an existing non-MDS drug to patient treatment to see if it helps, as well as starting the first MDS Clinic here in Chicago.

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