There are a number of research efforts occurring in the US and around the world investigating the causes of and potential treatments for leukodystrophy. It is important to understand the different stages of research and how the research continuum progresses towards the ultimate goal- finding a cure. This section provides a helpful overview of each research stage, showing how research goes from "bench to bedside."
For a current list of available clinical trials, please go to www.clinicaltrials.gov.
Basic research is about advancing new knowledge about a particular subject, with or without an intention of practical application from whatever is learned. In this stage, medical professionals such as physicians and researchers might search for how a specific metabolic process occurs, or what genetic mutations are the cause of a particular disease, but nothing is directly applied to patients in this phase. The information gained from this stage is the basis for all other research phases, and new insights can be gained from research curiosity that will potentially impact patients later on.
The findings from basic research are applied in translational research in order to advance and test a particular idea. Patients are still not directly involved at this point, as researchers may use lab or animal models to test a hypothesis or idea from previous research. The main point of this stage is to get closer to developing new therapies or medications, and it acts as a bridge from the traditional lab work that may have occurred in the basic research stage to the clinical trial phase where these new therapies are studied in patients.
Clinical Research (Overview)
Clinical trials are carefully designed, highly regulated studies to determine whether a particular intervention (such as a new drug or surgical procedure) is beneficial. In many cases, these trials are to find whether the new intervention is better than the current "standard of care" intervention. There are multiple phases of clinical trials, each focusing on a particular aspect of safety or effectiveness.
Clinical Trial- Phase 1
The first phase in a clinical trial focuses primarily on patient safety. A very small number of patient volunteers will be informed and consented about the trial and intervention. In the case of new medications, initially small doses of the drug will be given to patients, and the research team will study the side effects and dosage aspects of the drug. Other groups of similar patients will be given progressively larger doses to determine the appropriate tolerable dose. The actual drug effectiveness is a secondary focus in this phase.
Clinical Trial- Phase 2
The second phase focuses on the medication or intervention effectiveness. The drug may also be compared to a current medication or standard of care. A key aspect of this phase is to also to rule out ineffective medications. More patients are involved in this phase than in Phase 1 trials, but not to the extent of future phases.
Clinical Trial- Phase 3
Phase 3 trials are large-scale studies that may be collaborative efforts across multiple sites (such as hospitals). This phase is to determine whether the new intervention is safe, effective, and "better" than the current standard of care. A better intervention may mean more effective at treating a disease, resulting in fewer side effects, increasing patients' quality of life, etc. These studies can take place over multiple years, potentially involving thousands of patients.
Clinical Trial- Phase 4
After an intervention or medication has been proven through the above phases, it may be granted FDA approval through a very lengthy process. Phase 4 trials focus on on additional observations of safety, potentially for new populations of patients (such as children or pregnant women). Some medications may also be helpful for other diagnoses, and this phase may investigate other uses of previously-approved drugs.