Page 31 - Gates-AnnualReport-2018

2018 GATES GRUBSTAKE FUND UPDATE
2018 GATES GRUBSTAKE AWARDEES:
PREVENTION AND TREATMENT OF PULMONARY FIBROSIS, KEN LIECHTY, M.D.
Acute lung injury leading to Acute Respiratory Distress Syndrome (ARDS) causes 150,000 adult ICU admissions and 75,000 deaths annually in the US. Currently, treatment for ARDS is palliative, and does not address the underlying causes. Dr. Ken Liechty and his team have shown that their nanoparticle therapy is able to regenerate healthy lung tissue in a mouse model of acute lung injury. The Grubstake Award will allow them to develop an inhaled version of the nanoparticle therapy and do the additional safety studies required prior to a clinical trial in ARDS patients.
HEART REGENERATION BY CONVERSION OF NON-MYOCYTES INTO FUNCTIONAL CARDIOMYOCYTES, KUNHUA SONG, PH.D.
Eight million Americans suffer a heart attack annually; that is 3% of adults in the US. The annual cost for treating heart attacks is $12 billion, making it the most expensive condition treated in hospitals in the US. Even worse, 30-40% of heart attack patients develop heart failure, accounting for about 50% of all heart failure patients. The five-year survival rate for heart failure patients is only 50%. Despite the development of various therapies and medical devices such as the Left Ventricular Assist Device, the five-year survival rate has not changed in the past 20 years. Dr. Kunhua Song and his team have discovered a way to
regenerate healthy cardiac cells from the fibrotic tissue causing heart failure using a gene therapy approach. The Grubstake Award will allow them to complete their animal studies and begin preparing to file an IND to enter clinical trials.
GENERATION OF ENGRAFTABLE HEMATOPOIETIC STEM CELLS FROM INDUCED PLURIPOTENT STEM CELLS, MIKE VERNERIS, M.D.
Bone marrow transplants are a high risk, expensive procedure (>$13 billion) used to treat leukemia, lymphoma, and genetic diseases. Two thirds of patients receive transplants from unrelated donors, which causes delays to find matching donors, and increases the complications and cost associated with the procedure. Recently, scientists have discovered how to create induced pluripotent stem cells (iPS cells) from a patient’s own cells, expand them, and convert them to bone marrow cells. However, these cells have not been successful at engrafting into the bone marrow in mouse models, indicating they would not be effective
in a bone marrow transplant in patients. Dr. Verneris and his team have discovered that adding a small molecule during the conversion of the iPS cells allows engraftment of the cells in the mouse model. The Grubstake Award will allow them to confirm their initial finding in animal models, and to determine the most promising small molecule for clinical and commercial use.
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