Dupuytren disease has fallen through a gap in a system which ordinarily leads to better and better disease treatment. One side of the gap is that at its core, Dupuytren disease is a medical disorder, from some subtle biologic imbalance. The other side is that although Dupuytren disease does many things, Dupuytren hand deformity has hogged the spotlight and distracted from efforts to figure out how to stop the disease at its source.
OLD In the past, new treatments for medical diseases came from chance and observation. Someone noticed by trial and error that bark from white willow helped bring down fever and inflammation; another found that wintergreen did the same thing. Only much later the active ingredient (salicylate) was isolated from these and later modified to create aspirin. Medicine is based on these and other similar stories – trial and error, chance and observation. A common theme of these stories involves problems developing over hours, days or weeks – fever, inflammation, headache, infection, acute gout, heart failure, a long list. For these, the effect on just a few people over the course of a few days may be dramatic. In contrast, this list much shorter for chronic medical diseases, which vary between people and change slowly over years.
NEW As technology advances, trial and error is being replaced by sensitive tests and big data. These resources have led to targeted molecular therapies to attack the disease, not the patient. Targeted molecular therapies involve three stages of development. First, identify the genes or protein molecules at the core of the problem. Second, find or develop a custom molecule to lock on to the molecular Achilles heel of this core biology. Third, develop a means to deliver this medicine to those in need. Targeted molecular therapy has been a game changer in treatment of chronic medical diseases including rheumatoid arthritis, psoriasis, as well as many forms of cancer.
MOVING Chronic medical diseases have benefited from this new approach in part because their initial treatment is with medicines, reserving surgery only for medical failures. The new model fits neatly into the triage system for these conditions – medical doctors looking for better medicines.
STATIC A different triage system has trapped Dupuytren treatment in the past. Without any Dupuytren medicine, the role of primary Dupuytren caregiver has gone to surgeons with expertise in treating deformity. Not surprisingly, most Dupuytren research has focused on technical aspects of deformity, not its cause. Unless this changes, no progress will be made toward a cure for the cause of Dupuytren disease. It’s as if the treatment options for a dripping faucet were entirely confined to the choice of the best mop.
QUESTIONS This situation also misses a much larger problem: Dupuytren related health issues. Dupuytren disease is not simply a finger problem. Dupuytren disease is associated with higher rates of hypothyroidism, cardiovascular disease, frozen shoulder, cancer risk and early mortality. The reasons for these associations are unknown and unexplored. Medical treatment for Dupuytren disease could benefit general health and quality of life of Dupuytren patients far beyond their hands.
ANSWERS The goal of the Dupuytren Foundation is the big picture – to develop a cure for Dupuytren disease and all that comes with it, not just Dupuytren contracture. This demands a new approach. The most promising model is targeted molecular therapy, which is why this forms the basis of the International Dupuytren Data Bank. It’s time to close the knowledge gap of Dupuytren disease.