Looking from the macro level, we are entering a potentially magnificent era in the treatment of cancer and disease. We can begin to realistically envision a time when drugs will be designed to specifically target the causes of cancer. These drugs, known as genomics, will prevent, control or fix illnesses and designed to optimally match every individual according to their particular genetic code.
From a theoretical viewpoint, the long-term future of the science of cancer treatment has never looked more promising. The future will not be illness-free but looks likely to be illness-manageable-and-get-on-with-your-life; chronic conditions instead of lingering uncertainty.
Leading thinkers in the research world are sure of only one thing: genomic, molecular targeted medicines and therapies will radically change how we treat and manage virtually all diseases. It is only a question of when. And the answer to that question will determine the volume of output of drugs and therapy patients can use.
Genomic Drug Development
BIO is the association that represents biotechnology companies, many of which focus on genomic drug development. Late last year, I was fortunate to attend their meeting in Stuttgart, Germany to learn more about how their members functioned as businesses. This was a meeting designed to allow small biotechnology companies to present their work and look for partners to continue more research or to market compounds through larger pharmaceutical companies.
BIO is in many ways the great frontier of emerging drugs. Some of the member company ideas won't pan out; others will be blockbusters that will change our outlook on life itself. A couple of terms stuck out which, to me, demonstrate the tensions of transitioning to a world of genomic drugs and therapy—market share and delivery platforms.
Every company touted their ideas in relation to potential disease group market throughout the world. With respect to cancer, those market shares were categorized by body part or tumor type. Potential market share is an important standard by which drug development tends to be prioritized. In body part thinking, myeloma is a small market, unlikely to pull in the share that other cancers have.
On the other hand, many of the presentations focused on genomic delivery platforms. Once pathways and targets and the appropriate drugs for particular cancers are identified through research, researchers apply that knowledge to determine how to deliver the correct drug down the proper pathway to the targeted cancer.
Are we ready for this shift? Researchers are looking at cancer more systemically, searching for the molecular targets causing illness, and determining the best pathways to those targets. Yet our research infrastructure is still categorized by body part and tumor-type. Indeed, most pharmaceutical company representatives readily admit that they are not prepared to deal with new potential markets based primarily on genomics. We are likely years away from that happening on a large scale, but the serious thinking, planning, and implementation must continue at an accelerated pace. Unfortunately, funding follows increasingly archaic categories.
More than research money is at stake. It is also a question of emphasis. The federal mandate for NIH and NCI requires them to follow the paths of science, not markets. Genomics makes the various types of myeloma research even more attractive because of the number of untested pathways and targets associated with the disease. Perhaps discoveries in the genomics of myeloma patients will help patients with other cancers and diseases.
Distressingly, however, this long-term good news is tempered by the scary trend of the administration's medical research funding proposals outlined in last week's Myeloma Minute. So, what can we do?
Education = Advocacy
It will be up to us to be responsible for spreading the gospel of genomics. By educating policy makers about the future implications of genomics, we will shape a policy program that makes the best science of genomics accessible to all patients.
We must advocate and educate our members of Congress about the essential need to fund NIH and NCI at the levels needed to sustain progress against cancer. The current administration proposals will not accomplish this goal.
To be successful, it will require consistent engagement from advocates throughout the nation. Only then will our research institutes be able to expect consistent funding to be the hubs for basic genomic research that biotechnology companies can turn into drugs for patients.
Now is the time action, more so than ever before. In the next few weeks, we will discuss some advocacy issues and tactics for the coming year.