Q: What prompted you to start the Alpha-1 Foundation?
The Alpha-1 community evolved from our participation in a Phase IV requirement for longitudinal disease progression study conducted by the NHLBI. Those of us that were enrolled in the study had an opportunity to meet and appreciate the value of supporting each other as we all tried to deal with our diagnosis and related challenges, and as a result, individuals participating at 21 sites across the country created a virtual network by telephone, letters, newsletters, and a scattered number of support groups, which led to the creation of the Alpha-1 Association in 1991. When the NIH advised us (in 1994) that they had collected enough data to complete a natural history of Alpha-1 and would no longer continue intramural research on Alpha-1, we organized the Alpha-1 Foundation with the mission to provide the leadership and resources to increase research, improve health, promote worldwide detection, and cure Alpha-1 Antitrypsin Deficiency, with a very specific focus on research. We learned the true value of participating in clinical research through the NIH study experience and developed a unique partnership with the scientific and clinical community, which enhanced our patient perspective on leadership as we developed the Foundation.
Q: What do you consider your greatest achievements to date?
- As a result of our entrepreneurial approach to revenue generation for a rare disease, we have succeeded in funding over $43 million of research at 70 institutions in North America and Europe since launching our research grants program in 1998.
- With the guidance of our scientific and clinical leadership, we have developed a broad portfolio of research grants from basic science to translational research, to the social sciences (Ethical, Legal and Social Issues working group). We have managed to achieve the ultimate measure of success for a research program by facilitating the identification of therapeutic strategies and therapeutic targets through our investment in our investigator-initiated peer review program. These discoveries have led to significant interest from biotech and pharmaceutical companies and resulted in on-going clinical evaluation that may lead to therapeutic solutions for Alpha-1.
- The ability to develop a recurring source of revenue has allowed us to create a resource-sharing platform that has optimized our investment in research and minimize use of funds for administrative and overhead costs.
- In 2010, we launched and capitalized our venture philanthropy program, The Alpha-1 Project (TAP), with the mission to focus on patients, academia, pharmaceutical, and biotech companies in the relentless pursuit of cures and therapies for COPD and liver disease caused by Alpha-1. This platform allows us to make targeted investments to accelerate the development of therapies for Alpha-1.
Q: Tell us about the resources you have been able to create for the research community.
- We spent the first three years of the Foundation developing the infrastructure to support the international research community and keep the investigators that participated in the NHLBI study engaged in Alpha-1. The creation of the Alpha-1 Research Registry, the DNA & Tissue Bank reference lab, translational and diagnostic lab, and a network of Clinical Resource Centers have proved to be critically important to the progress that we have made.
- The creation of this research infrastructure has been used as a resource for clinical trial design, recruitment, management and ultimate licensure of three additional augmentation therapy products since 2003 thus eliminating critical shortage with the original orphan drug exclusivity.
- We created the Gordon L. Snider Critical Workshop Series to address the gaps in knowledge and the impediments to research on Alpha-1. This series of workshops has provided the opportunity to bring experts from around the world to cross-fertilize the expertise of investigators with a focus on therapeutic solutions and our international scientific conferences provided an additional opportunity to share information amongst investigators, keep them connected with each other, and encourage scientific collaboration.
Q: What is Alpha-Net, and how does it relate to your research agenda?
Consistent with the mission of the Alpha-1 Foundation, we created AlphaNet, Inc. as a not-for-profit health management entity to develop and implement a comprehensive health management program for all individuals with Alpha-1 and create a source of recurring revenue to support our research mission. AlphaNet currently provides services for a majority of the individuals on augmentation therapy and has published outcomes data demonstrating the validity of health management, reducing exacerbations and hospitalizations, and ultimately improving the quality of life of individuals with Alpha-1. AlphaNet is a unique business model that trains and employs individuals with Alpha-1 as peer health coaches (AlphaNet Coordinators) who coordinate between 120-160 “Alphas” including training them with our Big, Fat Reference Guide (BFRG-a health management guide). AlphaNet contributes to the Alpha-1 Foundation, the Alpha-1 Association, and other activities supporting our community. As of December 2010, AlphaNet contributed in excess of $30 million to the Alpha-1 Foundation to support our mission. AlphaNet's contribution has provided the necessary funding to support our research program infrastructure, a large percentage of our research grants, and volunteer scientific leadership structure.
Q: What are your top research goals for 2011 and what will it take to reach those goals?
- We are committed to increase our funding levels for our investigator-initiated peer review program
- Due to an alarming increase in the number of adults being diagnosed with end-stage cirrhosis related to Alpha-1, fund a liver biopsy study and also increase our investment in other investigator-initiated studies of liver disease
- Focus additional resources to support a study of the natural history of liver disease related to Alpha-1
- With the creation of TAP, we are committed to increase our capital investment in TAP to support therapeutic development activities under the guidance of our Scientific and Business Advisory Committee