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Forward-thinking philanthropic funders of disease research can play an absolutely critical role in stimulating medical progress, particularly in under-resourced areas, and helping to bridge the Valley of Death. Free of external pressures, nonprofit foundations are ideally positioned to make relatively high-risk investments that could significantly move a field of research forward and increase the likelihood that other parties also will invest.

Today FasterCures launched a first-of-its-kind resource designed to help potential collaborators better understand the landscape of nonprofit disease research foundations and engage in meaningful partnerships with them. The Research Acceleration and Innovation Network (TRAIN) Inventory is a free, Web-based index that catalogues the operational and partnering practices of over 50 leading nonprofits that find and fund cutting-edge medical research.

As the medical research community continues to explore new business models, we can all learn from the approaches these organizations have taken to speed innovation. Our goal is to amplify what they’ve done, share what they’ve learned, and help those interested in engaging them determine how best to connect.  The Inventory  succinctly provides information crucial to understanding and working with over 50 of the most impactful foundations in this space.

With downloadable profiles for every organization that participates in FasterCures’ TRAIN initiative, the Inventory uses a common set of metrics to outline and compare each organization’s research portfolio, collaboration efforts, financials, and more. It also highlights the great medical advances these organizations have fostered over the years, often in collaboration with partners from other sectors.

For example, did you know . . .

• Multiple Myeloma Research Foundation (MMRF), through a partnership with Onyx Pharmaceuticals, provided the clinical trial support and resources necessary to develop the first new treatment for multiple myeloma in five years, Kyprolis. Trials conducted through MMRF’s Research Consortium are opened an average of 60 percent faster than the industry average.
  
• The Alzheimer’s Drug Discovery Foundation (ADDF) has granted more than $51 million to fund more than 370 Alzheimer’s drug discovery programs and clinical trials in academic centers and biotechs in 18 countries.
  
• A collaboration between the Cystic Fibrosis Foundation (CFF) and Vertex Pharmaceuticals brought about the cystic fibrosis drug Kalydeco, which received FDA approval in January 2012. CFF provided funding, information about the patient population, and helped recruit patients for clinical trials.
  
• Through a partnership between the Translational Genomics Research Institute and the Virginia G. Piper Cancer Center, the Center’s clinical trials program is currently running more than 50 active clinical trials for advanced and rare cancers.
  
• The Michael J. Fox Foundation (MJFF) for Parkinson’s Research engaged 3,000 volunteers across 20 countries in studies to speed development of LRRK-2 related therapies for Parkinson’s disease. 
  
• Thanks to Autism Speaks’ advocacy efforts, 29 states have enacted autism insurance reform laws.
  

These are just a few examples of model practices among our TRAIN groups. To learn more about them and the other organizations in the network, check out our inventory – and pass it on!

New cross-sector deal-making tools and resources on TRAIN Central Station

by Kristin Schneeman, Program Director, FasterCures

Nonprofit foundations in our TRAIN network are perhaps more concerned than most folks about not “reinventing the wheel,” not spending time and resources learning things and making mistakes that others have already made. Financial and human capital is especially precious at these organizations, and inefficiency has significant consequences for the patients they serve. We have always featured on TRAIN Central Station resources that groups have shared with us that they feel others can benefit from having as a starting point for their own work.

We’re pleased to announce that we now have available on the site a treasure trove of new tools and resources, shared by participants in a workshop we held in June on “The Nuts and Bolts of Cross-Sector Dealmaking.” This cache includes template agreements of all sorts; due diligence guides; publications on collaborative models, venture philanthropy royalties, and charitable investing; and much more.

Among the documents and resources available are:

• The master agreement between the Addario Lung Cancer Medical Institute and the 15 academic institutions that are part of its consortium;

• The Chordoma Foundation’s guidelines and agreement for $10,000 prizes for the creation, validation, and deposit of new chordoma cell lines;

• “Everything You Ever Wanted to Know about Royalties and Their Monetization but Were Afraid to Ask,” a new publication by the law firm Schaner & Lubitz;

• A set of model legal documents for venture capital investments put together by the National Venture Capital Association, a window into the priorities of companies and investors with whom foundations may be interested in partnering.

 This is just a sampling of the more than 50 new tools and resources available. We hope you’ll find these and the others available on TRAIN Central Station useful models for your own efforts. If you have a resource, internal or external, that you would like to contribute or recommend to the community, we hope you will send it to us at train@fastercures.org!

TRAIN Workshop Gets ‘in the Weeds’ on Making Deals

Kristin Schneeman, Program Director, FasterCures

Earlier this month, FasterCures hosted a workshop on “The Nuts and Bolts of Cross-Sector Dealmaking,” bringing together a number of the venture philanthropies in TRAIN (The Research Acceleration and Innovation Network) to learn from and share with their peers the actual strategies and tactics nonprofits and biopharmaceutical companies are using to partner, as well as how foundations can better find and fund the most promising, commercializable research within universities. It was a valuable boots-on-the-ground exchange of specific resources and ideas for further exploration among a cross-section of all the stakeholders involved in medical research – foundations, industry, academia, government, and finance.

The day began with a discussion of a number of nonprofit collaboration models, intended to demonstrate the range of relationships and activities that currently exists among foundations and their academic and industry partners, from building and managing academic consortia for precompetitive and clinical research, to providing “concierge” services for academic researchers interested in commercialization, to investing in small companies through a variety of mechanisms and co-funding with large companies, to nonprofit and virtual biotechs.

Subsequent sessions focused on sharing strategies for finding partners (“the dating game”); the nitty-gritty of negotiating relationships, from licensing and royalties to milestones and data-sharing; and finally, what’s needed to sustain and replicate these relationships.

There were far too many valuable bits of information to relay in a brief blog post, and we will be working on a longer summary of the discussion, but I’ll highlight a few key takeaways:

• Lawyers and tech transfer officers shouldn’t run the show, but should absolutely be part of the team to craft terms that support the goals of a collaboration and help trouble-shoot any problems that might arise. It is critical to understand the legitimate needs of all partners.
• Communication is key, and there are so many ways we can communicate informally that we don’t – e.g., sharing non-confidential information with potential partners, holding non-binding legal roundtables to talk through issues such as the distinction between commercial and non-commercial research, etc.
• Industry-quality project management is fundamental to keeping programs on track and developing an asset that will be attractive to industry partners.
• Collaborations need to develop legs of their own and not depend on individuals within companies and foundations to sustain them. Building in surrogate markers of success that show progress over time (not just that “research is happening”) can help expand the circle of champions.

There were also many ideas for actions that would be helpful in smoothing the creation and sustenance of these collaborations, including:

• More transparent information about points of contact within companies, nonprofits, and academia;
• Education of tech transfer offices about what criteria potential partners are looking for (other than the Association of University Technology Managers data points they have historically been judged against);
• An effort to require more sharing of data about “failed” research efforts (and perhaps rebranding them as something other than “failures”); and
• Working with the IRS to better define “program-related investments” in the medical research space (outside of global health) to increase foundations’ comfort level with making such investments.

One of the most important outcomes of the day was a collection of resources that participants brought to share with one another and with the broader research community. These included template agreements of all sorts; due diligence guides; publications on collaborative models, venture philanthropy royalties, and charitable investing; and much more. This treasure trove of new tools will be available very soon on TRAIN Central Station.

‘Translation is a Team Sport’

The Learning Collaborative, a partnership between the Leukemia & Lymphoma Society (LLS), University of Kansas Cancer Center, and the National Center for Advancing Translational Sciences (NCATS) at NIH, is attempting to break the traditional drug discovery and development paradigm by bringing together blood cancer, drug discovery, and development expertise across several organizations. The Collaborative was formed with the goal of targeting repurposed drugs as well as novel, new drugs for the treatment of rare blood cancers.

Last week at a TRAIN (The Research Acceleration and Innovation Network) Webinar, the leaders of this unique effort shared what they’ve learned since they signed the Cooperative Research and Development Agreement (CRADA) in 2010. CRADA defined the resources and expertise each collaborator brought to the effort.

The three partner organizations bring complementary strengths to this collaborative. LLS has ample experience working with industry partners, an established network of experts, about 400 active research projects to contribute, and the sense of urgency that patient-driven foundations uniquely bring to the table. The University of Kansas Cancer Center excels in “bench to bedside” translation in drug repurposing, leadership in chemistry, and pharmaceutical experience. NIH’s NCATS brings a focus on rare diseases, industrial scale capabilities, and pharmaceutical experience.

“While some progress has been made in combating these cancers – half of the FDA-approved drugs of the last decade were approved for use in blood cancers – 50 percent of those newly diagnosed will die from their disease within five years,” said Louis DeGennaro, PhD, chief mission officer of the Leukemia & Lymphoma Society.

Scott J. Weir, PharmD, PhD, director, Institute for Advancing Medical Innovation, University of Kansas Cancer Center, gave a snapshot of the first Learning Collaborative effort, the auranofin project, and shared key insights:

• Keeping the patient in mind will accelerate the process and ensure their needs are front and center in the research agenda.
• Defining the collaboration, setting collective objectives, and managing expectations are fundamental.
• Project management is critical, both to manage across organizations and to maximize appeal to industry.
• Tech transfer must be integrated into teams to optimize the leverage of the data.
• Regulatory science issues that will impact their chances of success need to be addressed in the course of the research.
• Defining exclusivity paths and reimbursement strategies from the earliest stages is central to the effort. The group is working on what they call a “ValueMaP” (Value Maximization Path) to help define the value proposition for drug repurposing.

Currently, there are four active projects, two of them involving repurposing rheumatoid arthritis drugs for use in rare blood cancers (the other two are in earlier stages). The goal is to advance projects to clinical proof of concept within 14 months and to engage industry partners along the way.

This model, said Christopher P. Austin, MD, director, Division of Preclinical Innovation, National Center for Advancing Translational Sciences, can be applied to other organizations, and other diseases. “Translation is a team sport. It needs to be approached that way, and very consciously and deliberately managed that way.”

Documents critical to the Collaborative formation will be made available on FasterCures’ TRAIN Central Station soon.

Resources for Medical Research – We’re Gonna Need a Bigger Boat

By Margaret Anderson, Executive Director, FasterCures

It’s appropriations season in Washington, DC, and we are having an early spring. Why does that make me think of Jaws (the movie)? Because I love the beach and sometimes have nagging anxiety about what swims beneath me when I swim in the ocean? Perhaps. (And yes, I know that sharks attack rarely and they are minding their own business.) More so because I think we have allowed ourselves to forget what the real issue is out there lurking in the water. Resources and the future.

Yesterday, the House Appropriations subcommittee on labor, health and human services, and education chaired by Rep. Denny Rehberg (R-Mont.) convened a distinguished panel that focused on investments at the National Institutes of Health (NIH) and the newly created National Center for Advancing Translational Sciences (NCATS). A lot of the discussion centered on NCATS’ value proposition. And some discussion was about the pros and cons of the NCATS approach. (Let’s remember that NCATS is exactly 2 and a half months old now.)

NIH Director Francis Collins reiterated that NIH support for basic research remains consistent and strong at about 54 percent of the agency’s budget. He said he does not expect that percentage to change. He also emphasized that 98 percent the $575 million funding for NCATS comes from preexisting NIH programs. “We believe we could do a lot with modest resources at this point simply by putting the focus on bottlenecks in the drug development pipeline,” said Collins. Threading the needle? That sounds pretty rational.

Acting NCATS Director Thomas Insel has emphasized that NCATS was created to “complement—not compete with—the private sector.” NCATS pools together existing NIH resources and capacity in translational research to foster greater efficiency. It does not develop drugs; instead, it streamlines and improves processes to increase the odds of getting to therapies faster.

Todd Sherer, CEO of Michael J. Fox Foundation for Parkinson’s Research (MJFF), also providing testimony, noted that in pursuit of a Parkinson's cure, MJFF has funded more than $285 million in research since inception, of which 90 percent has gone straight to translational research. "Based on my experience of what can happen when substantive investments are made in translation, the total contributions NCATS can make to drug development may well be greater than the sum of the parts," he said.

At FasterCures, we support NIH’s efforts to create NCATS. Solutions can be tough to develop, and tougher to implement. Groups like MJFF and other outcomes-driven medical research foundations that are part of our TRAIN network, have demonstrated that innovative approaches to disease research are critical to speeding the R&D process. We must try new, promising avenues. NCATS is one tangible way to get moving. So what about the bigger picture?

We should not forget the larger menace on the horizon, the one that should cause the famous John Williams tune from Jaws to be playing in your head now. And that is the need to continue to keep our eye on the overall NIH budget, and the implications of flat or negative funding for NIH, as well as the threat of sequestration in January 2013. 

NCATS, new and important as it is, only accounts for a very thin slice of the NIH budget. Our national investment at the 27 institutes and centers at the NIH have yielded great scientific discoveries that have and will continue to improve health.

NIH also plays an essential role in sustaining our economy. A report released yesterday by United for Medical Research found that in 2011, the NIH directly and indirectly supported 432,094 jobs around the country. While significant, this is actually 55,000 fewer jobs than in 2010 – which, according to the report, “demonstrates that the lack of sustained investment in the agency is beginning to have an impact.”

The ripple effects of a constrained NIH budget go far and wide. Should sequestration (mandatory budget cuts should Congress not reach a budget agreement) happen, the NIH will lose 7.8 percent of its budget – $2.5 billion. Collins said that this means that “2,300 grants we had planned to give in fiscal year 2013 would not be able to be awarded. It would be devastating.”

Do we need to keep innovating our research system? Of course, but we also need resources. Resources feed that important basic science part of the continuum. Resources keep our young investigators in the field.

As the appropriations season unfolds, we urge all members of Congress and the medical research community to keep our eyes on the prize: a strong NIH will not only protect and sustain nearly half a million high quality jobs. This agency is also our biggest chance of having more “shots on goal” in the search for treatments and cures.

This is about resources. This is also about patients – patients today and tomorrow. This is also about the future. Let’s get our priorities straight. There are big fish – maybe even some really big ones – out there we need to be focused on, and as Roy Scheider who played Amity Police Chief Brody told us in Jaws, “We’re gonna need a bigger boat.”