MB Civic

By Robert H. Rubin  |  May 8, 2006  |  The Boston GlobeTHE AVERAGE person has only a cursory awareness of the great progress made in the past 50 years in the prevention and treatment of life-threatening disease. Drugs developed during this period have greatly improved the chances for recovery from invasive infection, acute cardiovascular disease, and a variety of endocrine and immunologic disorders. Today, new classes of molecules hold great promise for the treatment of previously intractable disease. However, several challenges must be met if these new molecules are to be clinically useful:

An international shortage of clinical scientists, particularly clinical pharmacologists, capable of transferring laboratory discoveries to the bedside in the safest and most efficient manner.

The absence of widespread arrangements for side-by-side collaboration among academic, pharmaceutical industry, and government scientists to work together on new medicines.

The availability of measurement strategies to evaluate candidate therapies.

For acute diseases, measurement of the safety and effectiveness of most new treatments is relatively straightforward. Not so for the envisioned new therapies that offer particular promise for chronic illnesses such as degenerative neurologic disease, arteriosclerosis, schizophrenia, and certain cancers. A new development strategy is needed to demonstrate the utility of these new therapies. The importance of these issues is underlined by the attention paid them by the National Institutes of Health’s Roadmap and the Food and Drug Administration’s Critical Path for bringing new approaches to the bedside.

Everyone agrees that the most important individual to be considered in the development of new therapies is the patient. Yet, patients today hear about new drugs and, particularly, revolutionary drugs-in-the-making before they are available. News flashes too often announce advances prematurely and misleadingly, raising false hopes that some dread disorder can be cured or eased.

With all drugs we need to be assured they can safely do the job they are intended to do.

As we move forward, trying now to treat disorders long thought to be almost untreatable, new drug development becomes significantly harder. We are entering the realm of complex, degenerative processes for which we have little experience in devising effective therapies or in testing them for acceptable use. How are we going to pre-prove safety and efficacy of the biologically diverse therapies — therapeutic monoclonal antibodies, stem cells, and gene therapy — that are emerging? What new approaches to assessing these novel interventions will be required? We contend that new collaborations will be required among the stakeholders.

For too long, a barrage of ethical and legal conflicts have dominated the interactions among pharmaceutical companies, academic research laboratories, and such governmental agencies as the NIH and the FDA. To our embarrassment and regret, serious conflict of interest issues and outright lawsuits have been involved.

But with so much at stake for seriously afflicted patients, we must develop new 21st century models of collaboration that take advantage of the strengths of each of the parties while adhering to the highest of ethical standards. We need to become fully open about our singular and independent roles in the midst of mutual scientific goals.

Recently, we staged an intensive ”immersion” course in clinical pharmacology for the benefit of 20 medical fellows — physicians in various specialties who are enrolled in a special Clinical Investigator Training Program of the Harvard-Massachusetts Institute of Technology Division of Health Sciences and Technology and the Beth Israel Deaconess Medical Center.

The pharmaceutical houses of Pfizer and Merck not only funded the immersion course, but also sent their scientists to participate. More important, they made it possible for senior scientists to teach alongside academic faculty for the benefit of the trainees.

The importance of this ”experiment in collaboration” goes well beyond the extraordinary success of the Clinical Investigator Training course. The experiment provides tangible evidence that new forms of academic-industrial collaboration are both possible and desirable. If we are to take on research not only for the creation, but also the application, of new therapies, then collaborative models will be necessary.

It is in such new-style arenas of collaboration where tomorrow’s therapies will be generated and properly tested.

Dr. Robert H. Rubin is a professor at Harvard Medical School and associate director of the Division of Infectious Disease at Brigham and Women’s Hospital.