ALSAM Impact Report 2017

ALSAM T H E R A P E U T I C I N N O VAT I O N S G R A N T

2017 IMPACT REPORT With this inaugural round of ALSAM Therapeutic Innovations Grant funding, four top faculty in the CU Skaggs School of Pharmacy and Pharmaceutical Sciences and their collaborators have the resources they need to move promising early-stage research efforts forward. By fueling investigation and discovery, The ALSAM Foundation is fostering bold ideas with big potential for transforming medicine and health care.

OUR APPRECIATION FOR THE ALSAM FOUNDATION

For nearly three decades, The ALSAM Foundation and the Skaggs family have helped the University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences pursue pioneering breakthroughs and new approaches to investigation and discovery. What we have achieved together is a testament to your vision and generosity, and we are grateful for your partnership.

With major funding for the four research projects highlighted on these pages, The ALSAM Foundation is investing in the future of health care. Your support keeps us at the forefront of innovation – and that is where the greatest returns on philanthropic investment often lie. By accelerating efforts to find novel treatments for infections like Zika and HIV, uncover a cure for muscular dystrophy, develop a deeper understanding of the microbiome and more effectively target cancer proteins, you are helping some of the bright minds in pharmacy today transform the future of medicine.

With these investments in our faculty, you are further advancing our vision of improving health care in our communities and revolutionizing patient care. Thank you.

Ralph J. Altiere, PhD Dean and Professor

David Ross, PhD Associate Dean for Research and Professor

We recently discovered something that we believe is quite significant and has the potential for effectively treating many infectious diseases. The advanced therapeutics innovation grant from The ALSAM Foundation allows us to immediately start pursuing this promising research direction. The funding also solidifies a significant collaborative effort with a new clinical colleague.

-Tom Anchordoquy, PhD

T O M A N C H O R D O Q U Y, P h D Professor, Pharmaceutical Sciences

Nucleic acid-based therapies for blood-borne viruses In collaboration with Dr. Maria Nagel in the Department of Neurology, we have developed a nanoparticle that is taken up by the same cells that are responsible for spreading viral infections. Although our initial work focuses on the chicken pox virus, the general strategy of using this nanoparticle-based approach to arrest viral replication offers the potential for treating a wide range of viral infections including Zika, West Nile and HIV.

Your grant will enable us to pursue never explored ideas on how to develop therapies to treat the most common muscle disease in young kids. With help from collaborator Dr. Bradley Olwin of the CU BioFrontiers Institute, we will translate the fundamental knowledge on protein structure and function acquired in our lab into more applied research that will directly impact patients.

-Krishna Mallela, PhD

KRISHNA MALLELA, PhD Associate Professor, Biophysical Chemistry

Protein structure-function based engineering of mini-dystrophins and mini-utrophins to treat muscular dystrophy This proposal aims to find a cure for Duchenne muscular dystrophy, the most common genetic lethal disorder in children. This disease literally tears skeletal muscle apart since dystrophic muscle lacks critical forcedissipating capacity. Symptoms beginning at age 2 that include constant falling, waddling and outturned knees, progress rapidly with patients’ life spans rarely exceeding early to mid-twenties. We plan to develop a new combinatorial therapy using gene constructs, viral vectors and stem cells. We will test these approaches in human muscle cells derived from patients.

Your generous grant will allow my laboratory to advance into a more therapeutically relevant and impactful research area much earlier in my career than I originally anticipated. We now have a unique opportunity to perform high-risk/ high-reward research that will lead to improved treatment options, especially for children suffering from pulmonary infections associated with cystic fibrosis. This will also facilitate collaboration with highly regarded clinical and research faculty on the CU Anschutz Medical Campus, expansion of laboratory personnel, and training opportunities for graduate students and postdocs.

-Vanessa Phelan, PhD

VANESSA PHELAN, PhD L.S. Skaggs Assistant Professor, Pharmaceutical Sciences Multiplex interactions leading to the development of microbiome dysbiosis and response of a microbiome community to clinical therapy Many diseases have been linked to changes in the composition of our microbial inhabitants. This project aims to understand how and why those changes occur. In particular, we are interested in how clinical treatments affect these microbial communities.

I am very grateful to be one of the first ALSAM Therapeutic Innovations Grant awardees. Your grant will allow my lab to move forward with initial research in a new area, and will help strengthen my collaboration with Dr. Craig Jordan and support our efforts to find novel treatments for blood cancers.

-Philip Reigan, PhD

PHILIP REIGAN, PhD

Assistant Professor, Medicinal Chemistry Targeting the MUC1-C Oncoprotein MUC1-C is a protein that is highly expressed in a number of cancer types and acts to promote tumor growth and survival. We propose to target MUC1-C directly with small molecules to disrupt these pro-tumor signaling cascades, and indirectly by targeting other components in the signaling network.

In collaboration with Dr. Craig Jordan in the Division of Hematology, we will evaluate these MUC1-C inhibitors in model systems of acute myeloid leukemia and determine their effect in the bulk tumor and in drug-resistant cancer stem cells.

C O N TA C T I N F O R M AT I O N Ralph J. Altiere, PhD Dean and Professor 303-724-2887 | ralph.altiere@ucdenver.edu