At the Jain Foundation, we are always looking for ways to improve our effectiveness by evaluating our own performance and measuring progress towards our goal of developing a cure for dysferlinopathy. However, given the unpredictable and non-linear nature of scientific research, finding appropriate metrics to track research advances and progress towards a cure is very difficult. Although we at the Foundation are confident that we have made progress, there is no clear method to measure distance traveled towards a cure or how many years remain before we reach that goal.
Thus far, we have no response for the most pressing question from patients: “How much longer?” Many members of the scientific community typically give an answer of 7 to 10 years, but this figure is not based on specific facts and is only meant to strike a balance between hope and expectation. The honest answer is that we don’t know. There are many possible paths to a therapy, and each is plagued by unforeseen and sometimes insurmountable hurdles. Therefore, it is impossible to predict whether an approach that is currently close to the clinic will succeed, or whether patients will need to wait for a new strategy to develop.
There is no other human endeavor where the complexity, uncertainty, and risk of failure are as high, and the process of measuring successes along the way as unreliable. Nevertheless, we will continue to search for reliable methods to measure progress in the dysferlin field so that we can continue to evolve and increase our effectiveness at pushing the field towards a cure. In the meantime, we have outlined below two indicators of progress and of the Jain Foundation's impact on dysferlin research: