Patient Advocacy Summit 2014: Introducing Innovations in Science

July 17, 2014

One of the most exciting things about being a patient advocate is having the opportunity to see behind the scenes in science. All over the world, small labs with big ideas are breaking down barriers between patients and treatments. At this year’s RARE Patient Advocacy Summit, we’re bringing some of those explorations straight to you. You’ll have a front row seat to the presentations of four ground-breaking research platforms that are about to change the way we create solutions for rare disease on a global level.

These twenty-minute science pitches promise to show new approaches to research and drug development. In each segment, there will be five minutes of Q&A that will allow rare disease non-profits and patient advocates a chance to ask questions, learn more about each project and how its mission will help bring drugs to market more quickly for those in the fight against rare disease.


Introducing our Presenters

Project Violet

Screen Shot 2014-07-17 at 8.24.18 PMRun by an impressive team of scientists including Dr. Jim Olson, Roland Strong, Julian Simon, Patrick Paddison and Eduardo Medez, Project Violet is a research program created as a product of Fred Hutchinson Cancer Research Center. The group has already produced an exciting new molecule as part of their fight against all childhood cancers, called “Tumor Paint,” a peptide drug used for lighting up cancer cells so that surgeons can get a better view for were to surgically eliminate tissue. Now the research group is turning its attention to other rare diseases. Just as plants and animals make their own drugs to survive in the wild, Project Violet is taking an interest in these natural blueprints and their building blocks of protein that can help to create new drug candidates. To help further its research, you can adopt a drug candidate to be explored or join a team of benefactors who are already working on one.

Project Violet’s hope is that some of these new therapies that will come out of this will help major diseases, and revenue streams from them may help fund more research on rare diseases. In the meantime, thanks to the philanthropy and education-based structure of the program, the scientists are not restricted in their academic investigations.



Screen Shot 2014-07-17 at 8.22.41 PMImmusoft is a Bay Area biotech organization, and its Immune System Programming (ISP™) technology is making waves in the scientific community. Described by the team to be one of the most effective known ways to deliver biologic therapies, the system works by modifying a patient’s own immune cells to produce biologics, such as monoclonal antibodies and proteins. By using a patient’s own immune cells to produce these molecules, ISP™ eliminates the need for frequent injections and offers consistent and lower serum drug levels, resulting in increased safety, fewer side effects, higher patient compliance, and greater clinical efficacy.

Immusoft’s proof-of-concept studies were the first in the world to demonstrate a clinically viable method for directly programming a patient’s immune cells to produce broadly neutralizing antibodies against HIV, and the lysosomal enzyme, alpha-L-iduronidase to treat MPS I. Immusoft has been awarded federal and private grants to advance the ISP™ platform. Collaborators include scientists from the Fred Hutchinson Cancer Research Center, University of Minnesota, the Ragon Institute (Massachusetts General Hospital, MIT and Harvard), University of California San Francisco, and Discovery Genomics, Inc. Potential therapeutic applications of ISP™ technology include several metabolic diseases requiring enzyme replacement, autoimmune disorders, infectious diseases, maintenance therapy for cancers and heart disease.


American MedChem

Screen Shot 2014-07-17 at 8.23.23 PMSo if the equation is Pharmaceutical Companies + Science = Cures, what’s missing to make Cures = Reality? Dr. Robert Selliah sat down with Mark on the World to explain how American MedChem works with biologists in translational research to help fill a gap in the age-old journey to rare disease cures.

“[Translational Research] is the grunt work of creating medicine. Every tablet you take has come through the hands of someone like me—a drug designer, a molecular architect,” said Dr. Selliah.

“We design these drugs that affect your genes. So the work just has to be done. The biology is there. Prototype molecules are not drugs yet. But we can take them and convert them to drug candidates, which can be validated in the clinic, under the FDA’s supervision. So that middle ground is where there’s a huge gap. The traditional pharmaceutical companies, and the venture capitalists are calling it “The Valley of Death,” (I call it the “The Valley of Serendipity”—you do the work and serendipity helps you to find this medicine.) We just want to collaborate, do the work, and hand it over to the clinicians…who will do the studies. We look for philanthropic money to help us get started. There are also a lot of grants out there to move programs forward.”

Since each disease strikes only a small number of patients, systematic drug discovery R&D for these diseases generally fall outside the business scope of risk capital and commercial pharmaceutical R&D resources. As a result, treatments are not available for most of these diseases, and there is much suffering in this patient community. AMC challenges this status quo; kids with rare diseases also need effective medicines and personalized medicines.

AMC’s work, in turn, reduces the financial risk for pharmaceutical companies to take on further research and production of drugs. The nonprofit corporation is seeking benefactors who can help them take on more potential drug candidates for research.

RARE Science

Screen Shot 2014-07-17 at 8.24.57 PMRARE Science is a nonprofit research organization that’s constantly collecting and distributing rare disease data for the benefit of patients all over the globe.

With the mission to accelerate identification of therapeutic solutions, RARE Science is driving forward a new approach to research discovery, clinical translation and patient treatment. The organization aims to provide services to the rare disease research community for data storage, centralized access to disease information and data, analytics and avenues for new discovery through their extraordinary patient-tool, the RARE Hub.

RARE Science fosters and enables collaboration and sharing of data across organizations to leverage existing knowledge, create synergies and drive efficiencies closing the window of time between discovery and treatment.

It’s ready to help patient advocates connect and share the benefits of the RARE Hub at this year’s summit. The RARE Science Team will explain what kind of capabilities are provided by the network that can enable foundations and individuals the tools to drive research and accelerate discovery. We’ll see an example of one pilot study that will exhibit just how existing partnerships can bridge government, pharma, academia and non-profit organizations. 

Can’t attend in person? Our new Livestream component will allow up to 5,000 interested advocates from around the globe to attend virtually. The event will be broadcast live with opportunities for patients to participate from afar using social media, including twitter and Facebook. Follow using #2014GGSummit

To view the full schedule of events click here.

To register to attend the summit, in person, click here.



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