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Faster Cures For the Future

Robert Goldberg, Ph.D.
Medical Progress Today
May 13, 2005

On April 21, the Milken Institute held its annual Global Conference in Los Angeles where I was able to meet, talk and mostly listen to many of the world’s best medical researchers, along with the investors who have bank rolled the genomic revolution.

Anyone fortunate enough to be at the event could not help sharing Sir Isaac Newton’s sense of excitement when he settled upon his laws of physics: “I seem to have been only like a boy, playing on the seashore, and diverting myself in now and then finding a smoother pebble or prettier shell than ordinary, whilst the great ocean of truth lay all undiscovered before me."

The health care forums at the conference were sponsored by Faster Cures, the Milken Institute’s center devoted to medical innovation. These discussions highlighted the technological, financial and entrepreneurial transformations that will allow us to turn our biological information into a new generation of targeted therapies. Medicines in the future will be personalized or tailored to be highly safe, and cost-effective. These breakthroughs will benefit people suffering from some of the most devastating diseases of our time.

Unfortunately, there are diminishing resources for an increasing number of innovative projects. Venture capital firms are reluctant to fund medicines in the early stages of development, creating what both investors and rare disease groups call the “valley of death” between initial drug testing and commercialization.

Large pharmaceutical firms are regarded as ideal partners, because they have expertise in drug formulation and potentially large sources of cash. But pharmaceutical firms are still digesting many of the biotech alliances—and—acquisitions of the past couple of years. Drug companies have seen their failures mount as they try to develop novel drugs that genetic computations suggested would work, but flop in human trials. Now, U.S. biopharma companies of all types face a potential loss of profits in the form of price controls and drug importation, even as the cost of moving drugs through the FDA continues to climb.

The critics of drug companies won’t like it very much, but it’s true nonetheless: new and probably expensive drugs will be the route by which medicine will progress throughout the next century. Much of our biology – sequenced and mapped by supercomputers processing and making sense of our DNA – is distilled into a few thousand proteins. Lee Hood, of the Institute for Systems Biology, and Nobel Prize Winner Lee Hartwell, have noted that these biological insights can be in turn turned into tools for predicting and preventing disease. Nanotechnology will eventually allow us to make billions of tiny measurements to see how drugs work and how well they work. Craig Venter, who sequenced the Human Genome in 9 months, noted that well before then, it will be quicker and cheaper to develop a common set of molecular markers for similar purposes.

Either way – or both – we will move from trial—and—error treatments to predictive medicine in relatively short order. The biological information, computational power, mathematical models and engineering expertise now exist for a move to more personalized medicine. Already on the market are the first diagnostics that allow doctors to distinguish between disease and non—disease, different types of disease, progression of disease, which drugs work, and side effects.

Being able to predict how beneficial a medicine is, and whether it will work, is of great value, especially if your life depends on it. It was no surprise that many of the groups representing patients with rare diseases would be supportive of this fantastic voyage into the future. But it was startling to learn how many of the groups not only have active drug development programs but are leading the way in using biomarkers and other gene-based tools to improve the “batting average” of the drugs they are seeking to commercialize, and to insure that they are right drug, for the right patient, at the right time.

Then again, what is an orphan drug or rare disease but highly individualized or unique disease requiring a unique treatment? So non-profit foundations such as the Multiple Myeloma Research Foundation, the ALS Therapy Development Foundation and the Epilepsy Project have created a critical path to improve the efficiency of drug development, as well as the partnerships necessary to create new medicines for diseases that currently lack a cure.

Their solution is a new paradigm of commercialization, one in which drug development is more efficient, more predictive, and focused on personalized medicine. It includes rare disease groups and their partners in the academic medical community who, in seeking to develop a faster track to better cures, team up with drug companies of all sizes in order to tap into larger amounts of capital and potential profits.

This solution also consists of developing nations with significant public-health needs. Korea, India, and Singapore have spurned policies that include price controls, patent grabs, and bulk production of generic drugs, and have sought to stimulate investment in the highest value medicines. They too are seeking to promote profitable partnerships that will create their own biotechnology and pharmaceutical industries, using America as their model for promoting better medicine and prosperity.

The Global Conference brought together a unique group of scientists, advocates and funders who want to transform knowledge into progress. The Milken Institute also recognizes that commercialization is the “fuel of invention” that will bring these medical miracles to fruition. If Washington policymakers keep that fuel flowing, faster cures are sure to come.

Robert Goldberg, Ph.D. is director of the Center for Medical Progress at the Manhattan Institute for Policy Research.

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