Pemphigus vulgaris patients are sought to participate in the Phase I trial of a peptide that, if it works as hoped, may mean an end to large doses of the systemic steroids used to treat the disease and the severe side effects steroids cause.

Peptimmune, a biotech firm based in Boston, Massachusetts, is asking PV patients in four cities across the United States to participate in tests sanctioned by the Food and Drug Administration, (FDA). The peptide, designated PI-0824, is delivered into the patients blood stream intravenously in two sessions.

The patient is monitored closely for eight weeks prior to the infusion and for several months afterwards. Fifteen patients over 18 years of age and on a maintenance dose of prednisone are sought for the study.

The clinical trial is structured in 3 phases with the safety of the patients being the most important factor. The dosage level in the first phase is considered low and its only after all 15 patients complete the first phase successfully that the decision will be made to find a new group of volunteers and enter Phase II at a slightly higher dosage.

The first to volunteer was Matt Koenig of Valley Stream, New York, a 46-year-old Engineer who developed PV in 1991.

I had some good feelings of accomplishment about the whole thing, although there was a certain amount of wait and see anxiety for potential side-effects, but that faded by the next morning. (Read Koenigs personal feelings about the trial.)

Designing peptides and other molecules to improve the treatments for a wide range of diseases was a $2.2 Billion industry in 2003 and its expected to grow by at least 50% in each of the next three years. See Peptides, the basics.

Before any trial of a new medicine, the manufacturer reviews the chemical properties and process of the test to insure the highest level of safety possible. Animal studies precede testing in humans. The FDA then does its own evaluation. Because of this multi-tiered safety net, serious problems are rare.

Patients interested in additional information are urged to contact one of the four principle investigators. They are:

Location and Contact Information

California
University of California San Francisco, San Francisco
M. Kari Connolly, MD, Principal Investigator
Christine Antolos 415-502-6229

Maryland
Johns Hopkins School of Medicine, Baltimore
Grant Anhalt, MD, Principal Investigator
Anita Guidos, RN 410-955-3422

New York
NYU Department of Dermatology and School of Dentistry, New York
Bruce Strober, MD, PhD, Principal Investigator
Lorrie Jondreau, RN 212-263-5244

Ohio
Case Western Reserve University, Cleveland
Neil Korman, MD, PhD, Principal Investigator
Zsa Zsa Porter 216-844-5899

 

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