Science matters

Our economy, global competitiveness, and, most importantly, our health depend greatly on whether we prioritize our investment in and commitment to science. That sense of urgency and the powerful voice of patients was at the heart of our three-day event, Celebration of Science on September 7-9, held at George Washington University and the National Institutes of Health (NIH). As FasterCures Chairman Mike Milken put it in a Wall Street Journal opinion piece, “[September 7] in Washington, a town not known these days for widespread consensus, a remarkable gathering of leaders begins a weekend of events devoted to a single proposition: Science matters.”

We celebrated because we’ve come so far – the scientific and technological advances of the past few decades have transformed the way we live, what we know about disease, how we’re searching for treatments, and how we’re delivering them to patients. Throughout the three days, we saw glimpses of the transformative power of science and research:

• Progress against HIV. First, from the vantage points of many forces in HIV advocacy – including Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, and Earvin “Magic” Johnson, chairman and CEO of Magic Johnson Enterprises – two people who defied the odds of a disease that when it burst onto the scene 30 years ago was considered a death sentence. This progress was further crystallized as we heard at the NIH about “Yesterday’s Progess and Tomorrow’s Promise” from Timothy Ray Brown, the “Berlin patient,” the first person who has lived to tell the story of how he once had HIV.
• The power of the human genome. One of the largest collaborative efforts in science, which was led by NIH Director Dr. Francis Collins, is the sequencing of the human genome. Scientists were able to piece together, in order, the 3.2 billion nucleotides that encode our DNA, which is the blueprint for the cells of our body. Due to an incredible surge in the power of sequencing technology and plummeting costs, the idea that we may integrate sequencing into clinical care is closer than ever before. On the campus of NIH, we saw how the future of medicine is taking shape – today – thanks to the family of the 16-year-old Beery twins, Alexis and Noah – whose doctor used genetics to unlock the mystery of the twins’ affliction. What they learned led to a precision treatment approach for the twins’ rare muscular disorder, and they are leading virtually normal teenage lives today as a result.

We also heard about the development of the therapy Kalydeco, which treats cystic fibrosis patients with a specific gene mutation. Kalydeco, developed by Vertex Pharmaceuticals and the Cystic Fibrosis Foundation, is stunning proof that if you can get government, academia, nonprofit, pharmaceutical companies, and patients to work together for a common goal… you can literally change the world.

• Advances in rehabilitation medicine. We heard great optimism from Captain Dan Berschinski (retired), who lost both of his legs well above the knee and part of his hand in an attack in Afghanistan. He stood proudly in front of 1,000 leaders from across sectors, bluntly challenging us to stay focused on making medical research a priority, and in particular on his fellow veterans whose injuries are not as visible as his own, such as traumatic brain injury and depression. His remarks were further underscored by Paul Pasquina from Walter Reed and Geoff Ling from the Defense Advanced Research Projects Agency (DARPA), who showed us the vital importance of this research in treating combat-related injuries.
• Better understanding of the crafty cancer cell. Cancer researchers are in relentless pursuit of therapies for all unmet needs. We heard from the world’s leading investigators about progress in areas such as molecularly targeted cancer drugs, which give patients substantially greater hope than was thought possible even a few years ago. New understanding of the genetic events that contribute to cancer, the survival strategies of tumors and the body's immune responses, adoptive immunotherapy, and other treatment options emerging from their laboratories are achieving increasing success in holding back cancer progression and turning once-fatal diseases into treatable conditions. Susan Lindquist, an MIT researcher, described how a protein’s unique structure is critical to its function and how disruptions to that structure can lead to serious health conditions, such as cancer.

We also heard from Winter Vinecki, a 13-year-old triathlete, who is racing to find a cure for prostate cancer after it claimed the life of her father a few years ago. As crafty as the cancer cell might be, we know that determined advocates like Winter will not rest until we win.

These are among the many patient and researcher stories that came to life in discussions about the power of science. Transformative solutions to many of our most significant challenges are possible – but only if we make science a national priority and reaffirm our commitment to supporting it.

Scientific breakthroughs, combined with public-health advances, underlie what is arguably the greatest accomplishment in human history: World-wide average life expectancy has more than doubled since 1900. This has fostered major social benefits and economic growth in this country and many others.

However, this growth is in jeopardy because of the threat of sequestration, the automatic budget cuts that will happen soon if Congress and the White House do not act. Many in the scientific community are concerned it will cause irreparable harm and cast a pall on the future of medical research and patients waiting for treatments and cures. Last week the Office of Management and Budget (OMB) released a report that laid out the “devastating impact” sequestration would have on scientific research.

Progress we lauded during the Celebration of Science may be halted if federal dollars are cut to the NIH, Food and Drug Administration, and other agencies involved in medical research and advancement.

The Celebration of Science showed why science matters – because of the innovative research being performed in labs in every state, because of the treatments and cures reaching bedsides for a variety of diseases, and because all sectors concerned with medical research can come together with a focus on patients. There is nothing that matters more.