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.