06/20/2010 05:12 am ET | Updated Nov 17, 2011

It's Time to Move Beyond Baby Steps on Cancer Research

It is a truism in business that when the economy slows and the going gets tough, the tough innovate -- by taking stock, streamlining operations, and focusing on what works. In recent years, we have seen companies from Sony to Estee Lauder make difficult but necessary improvements, with the hope that when the economy improves, so will their fortunes. The very best entrepreneurs -- think of Amazon and the Kindle -- launch disruptive new products or entirely new businesses.

In the scientific community, however, the impulse is exactly the opposite: when the economy slows and money gets tight, research funding tends to get more conservative. Rather than investing in cutting-edge new treatments and promising new therapies, our research establishment -- which, by nature, is structured for caution -- tends to hunker down and focus on established research that promises more incremental returns.

While understandable, this approach also compromises the possibility of more significant breakthroughs. In our current economic downturn, one of the casualties of this go-slow approach is the exciting and promising field of targeted cancer research -- where tighter public budgets and a national strategy focused on incremental progress have helped slow some of the most promising breakthroughs on cancer.

For decades, the debate in the cancer community can be summed up in the difference between a nuclear bomb and a cruise missile. A nuclear bomb destroys everything in its blast radius; a cruise missile, by contrast, destroys a specific target, while leaving the surrounding structure largely intact.

In cancer treatment, more-traditional chemotherapy and radiation are the equivalent of the nuclear bomb. Both seek to kill cancer cells by flooding the body with drugs or gamma rays -- but in the process, healthy cells are often damaged as well, leading to debilitating side effects.

By contrast, targeted cancer therapies are like the cruise missile: they are designed to travel through the body directly to the tumor -- and once arriving, to block the cancer's growth by attacking molecular weaknesses within the bad cells of the tumor itself. It does so without harming the healthy cells around it, and in most cases, without significant side effects.

By contrast, targeted cancer therapies are like the cruise missile: they are designed to travel through the body directly to the tumor -- and once arriving, to block the cancer's growth by attacking molecular weaknesses within the bad cells of the tumor itself. It does so without harming the healthy cells around it, and in most cases, without significant side effects.

At least a dozen targeted drugs have been approved since 1998 -- the most famous of which is Herceptin, which has already made a dramatic difference in treating both early as well as late stage breast cancer. In the past two months, media reports have provided Americans an exciting glimpse into the potential held by dozens of targeted therapies.

Take an example highlighted by a recent New York Timesseries: melanoma. As anybody who has had it knows, melanoma is a particularly insidious form of cancer, one of the few that manifests itself externally on the body, in the form of nasty black lesions on the skin -- matched by internal tumors below the skin. Researchers have identified a mutation on a specific protein -- known as the B-RAF protein -- that is thought to be responsible for the growth of this disease in certain people.

Working with a pharmaceutical company, researchers led the creation of a compound -- known by a moniker worthy of George Jetson, PLX4032 -- designed to bind to the B-RAF protein, interfere with its replication, and destroy it. The results were astounding. When a high enough dose of the drug was administered to patients in clinical trials, some had regression of their tumors. In other cases, improvements were so dramatic that doctors on the trial sent one another "before and after" CT scans of their patients, to show tumors that had all but disappeared. Patients found that they could taste food again, live without pain, and live normal lives. Many believed they had beaten their cancer.

But then, one by one, patients began to relapse. The drug was generally found to stop the melanoma for about six months. Rather than dwelling on the limited success -- after all, six more months of life is not insignificant -- trials like these give rise to more hopeful questions. Are targeted drugs the key to future advances? If blocking the protein made by one mutated gene led to a second mutation -- as seemed likely -- what if that mutation could be identified and blocked? What would happen if these drugs were taken in combination with others in the same way that cocktails of protease inhibitors have dramatically improved life for those with HIV? Could cancer be turned into a chronic condition instead of a death sentence?

At a time when one in four Americans will die from cancer, targeted therapies give dramatic new hope for treatment and possible cure. Yet, as a nation, we will not realize the potential of the most promising new cancer research until we begin funding cancer research at levels equivalent to the epidemic again; and until we move beyond the conservative, go-slow approach cancer strategy that has dominated the past few decades -- a strategy which seems content to focus on incremental progress, on generating second or third generations of traditional drugs that lead to marginal improvements, rather than funding disruptive innovation that could lead to dramatic leaps forward and many more lives saved.

A new sense of urgency needs to be injected into the debate, in three ways.

First, although the National Cancer Institute has recently increased the funding of R01 research grants from the 12th percentile up to in certain situations the 25th percentile, a greater focus of this should dedicate a higher overall funding to newer targeting concepts -- that are considered high risk, high reward, but may lead to dramatic improvements.

Second, government funding should be awarded to grants that emphasize innovation and impact in addition to feasibility, and private investment should look towards investing in more novel approaches that may result in greater impact. Tax incentives should be provided to urge pharmaceutical companies to take big bets on bold new treatment;

Third, the Food and Drug Administration should consider increasing the cases in which these "smart" targeted treatments are given to people who are less sick, without requiring, as the FDA does now in many cases, for patients to be at death's door before joining trials. From 1975 to 2000, the number of cancer cases across the globe doubled. By the end of 2010, cancer is expected to become the leading cause of death worldwide -- doubling again by 2020 and nearly tripling by 2030. We can't afford the go-slow approach anymore. It's time to launch a few cruise missiles into this debate.

Phillip M. Arlen is the CEO of Neogenix Oncology Corporation, a biotechnology company focused on developing state of the art diagnostic and therapeutic products for the treatment of pancreatic, lung, cervical, colorectal and prostrate cancers. Several of these products are currently being moved to commercialization. Neogenix Oncology is a privately held corporation with facilities in Great Neck, NY and Rockville, MD.

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