Medical Research: When Scientific 'Breakthroughs' Get Lost in Translation

Margaret Anderson
Executive Director, FasterCures, The Center for Accelerating Medical Solutions

Every day we see stories in the media about the latest medical "breakthroughs" that could lead to treatments or cures for dreaded diseases. We are overwhelmed with snippets about stem cells, genes linked to Alzheimer's disease, autism and diabetes. We hear that cancer drugs are being tailored to treat an individual tumor. And yet, many breakthroughs like these have not borne fruit for patients.

Whether it's because science is hard and unpredictable, or that resources are limited, or there is lack of prioritization -- too many great scientific ideas sit at the laboratory bench waiting for translation. But, there are successful models that have turned a basic discovery into an effective therapeutic option. These models can provide pathways to a healthier future.

We are at a critical inflection point in current discussions within the biomedical research establishment about what actions need to be taken to push the science toward cures where possible. We need to take advantage of this moment, and we need to bring patients, the public and policymakers into the conversation. Next week from December 13-15 FasterCures is convening in New York City all of the sectors involved in medical research to do just that. Partnering for Cures is a meeting like no other, a place to forge collaborations and participate in outcome-focused dialogue about the challenges facing medical research. We have always maintained that each of the sectors plays a vital role, whether it is government, industry, philanthropy, academia, finance or the non-profits. And the need for their ability to seamlessly pass the baton from one to the other has never been greater or the stakes higher.

Successful models have integrated all of these sectors. Everyone needs to be at this table. Few life-saving therapies have come to market without the resources of private industry. Increasingly, patients have become more sophisticated and disease groups are ever innovating with new models for collaboration with academia and industry. The U.S. government is recognizing that it can play a special and critical role in providing an environment where successful partnerships can grow and proliferate. Partnering for Cures provides an opportunity for all sectors to productively collide, creating an ultimate open source opportunity to shine a light on these models so others can learn and build on them.

One area of keen interest to us is identifying solutions and models to cross the so-called "Valley of Death" -- an ever-widening gap in funding and support for the kind of research that moves basic science down the path toward treatments.

In a new report released today by FasterCures, "Crossing Over the Valley of Death," we highlight the productivity gap that currently exists today, how research moves from molecule to marketplace, how we can traverse the Valley of Death and what all of the sectors are doing towards that end. Many players are marching into that valley, but we are far from reaching the other side.

The need to keep marching with the resources required to make the passage is recognized by advisors to U.S. National Institutes of Health (NIH) Director Dr. Francis Collins, who have recommended that a new translational medicine and therapeutics development center be created by the federal agency post-haste. This proposed center would bring together existing NIH activities in translational research and medicine and allow greater coordination and collaboration internally as well as externally, and ultimately, we hope, produce greater outcomes.

We need to support this recommendation, which if implemented would shine a light on the critically needed but under-resourced and under-appreciated area of translational research. In our jump to embrace this incredible opportunity, however, we need to ensure that Peter is not robbed to pay Paul. NIH's other strengths include supporting the nation's basic biomedical research enterprise, and that focus cannot be lost or diminished in our impatience with the pace of progress. Basic science is still as important as ever, but we also want and deserve concrete outcomes.

Dr. Robert Beall, President and CEO of the Cystic Fibrosis Foundation often talks about the Foundation's own model for de-risking research as providing "more shots on goal." Never has the need been greater to have all of the sectors implement that same approach, to take bold steps to move forward swiftly.

Our nation's wise and prolonged investment in basic science has produced discoveries that now need translation. Knowledge gained from basic discoveries allows us to take more strategic and informed shots on goal. This new and important focus on translation no doubt makes some in the research community uncomfortable, as they worry about focus, changing research priorities and competition over scarce dollars.

We recognize those concerns, but at the same time, the need for accountability and outcomes prevail. Patients need to know that we, the collective "we" in the medical research system, are doing everything we can to get new preventive, diagnostic and treatment options through the pipeline and into the clinic. The alternative to this change is the status quo -- 15 years for an intervention to go from bench to bedside. Clearly that isn't acceptable. Is it?

Join us at Partnering for Cures, December 13-15 in New York City. .

Are we optimizing our health IT system to improve patient outcomes?

Dozens of public and private stakeholders in medical research joined FasterCures last week at a forum about health information technology (health IT) policies – what these mean, what these represent, and how these will impact patient outcomes. The discussion focused on our national health IT infrastructure and how it is designed (or not) for clinical research. FasterCures Executive Director, Margaret Anderson, drove the discussion with Adam Clark, Ph.D., Director of Scientific and Federal Affairs at FasterCures. Adam sits on the policy committee of the Office of the National Coordinator for Health Information Technology (ONC) as a consumer advocate.

Key points raised during the forum include:

• The lack of communication between different sectors of the medical field despite the ever-growing state of health information technology.


• The current efforts by the ONC to establish standardized electronic health record (EHR) systems as a means of enhancing the quality of clinical care and


• Utilizing patient data to advance research and development.


• Addressing the challenges of patient privacy and security in implementing standardized EHR systems.


• The need for patients to have access to information in EHR systems, not only clinicians and providers.


• The importance of health information technology encompassing clinical research moving forward, creating effective avenues towards scientific innovation.

This effort is part of FasterCures’ Think Research program that supports the utilization of medical records and clinical datasets towards research on the progression of diseases and the development of treatment methods for them. An updated version of the FasterCures’ white paper entitled “Think Research: Using Electronic Medical Records to Bridge Patient Care and Research” will be released in early 2011.

Late-Breaking Partnering for Cures Addition: Hamburg and Woodcock to Discuss Drug Development in the Age of Targeted Therapy

We are excited to share with you a late-breaking addition to the already stellar Partnering for Cures program! A new plenary session now features News from FDA: Drug Development in the Age of Targeted Therapy.On December 14, 2010, FDA Commissioner Dr. Margaret Hamburg and Director of the Center for Drug Evaluation and Research Dr. Janet Woodcock will discuss how advances in science and technology, including genomics, offer exciting new opportunities to transform the drug development and review process. They will address how academia, companies, and FDA can speed the development of targeted treatments for cancer and other devastating illnesses, including innovative trial designs and regulatory policies.

With less than two weeks until Partnering for Cures, we have plenty to look forward to:

• a dynamic program (12 panels and four plenary sessions) with an exemplary speaker roster.
• a customized partnering system that eases the process of finding the right contacts and establishing meaningful relationships (already live and active!).
• a new addition to the program, the Expert Consultations component allows participants to schedule free, one-on-one consultations with scientific, technical, and financial leaders.
• an innovator presentation track featuring 31 case studies of cross-sector collaborations.
• focused networking opportunities according to specific therapeutic affinity areas.

Register now and benefit from productive collisions with nontraditional allies from across sectors of medical research.

HHS Releases Awards for Biotech Programs Funded Through Healthcare Reform Act

Adam M. Clark, Director, Scientific and Federal Affairs
The Department of Health and Human Services recently released approximately $1 billion in funds through a new program called the Qualifying Therapeutic Discovery Project (QTDP). Included as part of the Patient Protection and Affordable Care Act (also known as the Healthcare Reform Law), QTDP is unlike traditional NIH peer-reviewed projects as it uses an expedited review process to fund small biotechnology companies producing potential products in the biomedical pipeline. It specifically directs funding to companies developing products that will treat unmet medical needs, reduce long term health care costs, or advance the goal of curing cancer within 30 years.

Approximately 3000 companies from 47 states and the District of Columbia received funding through the program through either tax credits or grants, with California topping the list with nearly $300 million in awards followed by Massachusetts with $125 million. Projects ranged across the spectrum of diseases including approximately 30 research programs in Parkinson’s disease, 60 in Alzheimer’s, 80 in diabetes, and more than 100 in cancers.

Quantifying the impact of this funding on the medical research paradigm will be challenging to measure in the short-term. Here’s hoping that this funding boost will provide financial incentives for small biotech companies to pursue development of much needed therapeutics, and spur greater efforts that will accelerate translation of scientific discovery into clinical products.

At FasterCures, we firmly believe that patients need results-oriented research that may be high-risk but with the potential of high rewards, similar to those supported by this new effort. After all, the real value of our national investment in scientific discovery should be measured in terms of improved patient outcomes: better health, improved quality of life, and overall wellness.

Creating Opportunities in Drug Design: Applying 21st Century Technologies to Drug Safety Testing

Adam M. Clark, Director, Scientific and Federal Affairs

Advancing medical progress can sometimes be akin to putting together 3,000-piece 3d puzzle. Each piece is critical and must be in its appropriate place. In medical research, we have many of the most critical pieces such as technological achievements in molecular and cellular biology that are revolutionizing research capabilities and expanding our understanding of disease.

• Once hailed as the pinnacle aspiration of clinical genomics, the $1000 genome now seems to be right around the corner.
• The application of protein sciences, aptly named proteomics, has demonstrated clinical effectiveness in evaluating for heart damage, as in the measurement of troponin, and in identifying responders to targeted treatments like Herceptin in certain breast cancers.
• Advances in bioinformatics are providing researchers the ability to integrate large and complex datasets and model molecular networks.
• And just this month, researchers presented a novel breakthrough using RNA to reengineer human fibroblast cells to pluripotent stem cells in a manner that is almost 100 times more efficient than gene transfer and does not alter the cell’s genome, bringing about tremendous potential for using differentiated stem cells for cellular modeling of disease or treatment response.

And yet, we have yet to position these key pieces in their appropriate places, see how they connect so they can build toward the ultimate goal: to improve patient outcomes.
Consider that the number of new drugs being submitted to the FDA for approval has fallen from 53 drugs in 1996 to 19 just last year. Very few drugs (only about 8%) make it from pre-clinical testing all the way to market and among the reasons for a drug’s failure to move ahead early is toxicity concerns in animal models during early stage testing.
Recently, I participated in a panel that focused on this issue as part of the Brookings Institute and Friends of Cancer Research Conference on Clinical Cancer Research. The discussion focused on the value 21st century technologies could add to improving drug design, screening, and approval – values that benefit both the drug developers and the patients receiving promising new therapies. In an issue brief the panel put together to complement the discussion, we highlight two case studies (an integrated approach to organ injury and oncology drug-induced cardiovascular toxicity) that may help shed some light on real-world implications of safety assessments.
Among the key concepts from the panel and echoed throughout the day-long conference were:

• Current in vivo models for drug toxicity testing have changed very little in decades and have not taken into account advances in molecular and systems biology.
• Animal models were inadequate predictors of toxicity responses in humans, both in terms of generating evidence for biologically accurate mechanisms of drug toxicity, as well as identifying concerns for low-incidence, but dangerous, toxicity responses in our diverse human population.

Panelists proposed that the U.S. Food and Drug Administration should prioritize toxicity testing, noting that improvements in this area could increase the number of drugs making it into clinical trials, decrease the time in pre-clinical testing, and provide a more accurate and biologically-based profile of the benefit:risk ratio for new drugs.

We await anxiously where this conversation may lead and look to the FDA to start picking up these puzzle pieces - genomics, proteomics, bioinformatics, and other scientific breakthroughs – and begin piecing them together to improve patient outcomes. Drug toxicity profiling, as technical as it may be, is a critical piece to help complete the cure puzzle.

Adam Clark joined FasterCures in September to lead its scientific and federal affairs programs.