Since my Kuvan responder trial last August, I’ve had many conversations with BioMarin representatives who say they are not yet ready to “throw in the towel” when it comes to deciding whether I am a responder to the first PKU prescription drug. The results of my first attempt were determined inconclusive; however, as a PKU patient currently off-diet, my eating habits are certainly more complicated than those folks who normally embark on this journey.
Needless to say, I’ve given the matter much thought and do feel that it might be worth a second go-around with the Kuvan responder trial later this year. A couple things will be done differently though. Here’s a look at some of the changes that will hopefully lead to a more definitive result.
Obtain baseline data of phe-levels prior to Kuvan responder trial—Since my unrestricted diet complicated matters, I am sending my clinic a blood sample and comprehensive food diary once a month in order to establish a baseline phe-level. During my first Kuvan responder trial, I was manually tracking protein using my iPhone’s notepad app. This time, however, I’ve starting using a different, free iPhone app called MyFitnessPal which tracks all sorts of nutritional data including protein. I just completed my baseline phe-level for the month of March. For that day, I had consumed 83 grams of protein and my phe-level came back at 17.6mg/dl. By the time I make a second attempt at a Kuvan responder trial, I should have 4 or 5 of these baseline phe-levels to work with.
Collaborate hand-in-hand with a Kuvan clinical coordinator—Communication issues and the lack of a well-defined trial process may have impacted the results of my first Kuvan responder trial so I was happy to hear that BioMarin has created a new position called the Kuvan clinical coordinator. This person serves as a liaison between PKU patients who are exploring the use of Kuvan and the metabolic clinics that prescribe it. A quick Google search found a BioMarin job posting which described the new position’s responsibilities as collaborating “with assigned patients to ensure compliance with the clinic’s protocol, assess response to Kuvan therapy, advocate for patient needs, conduct patient-level education, and…to address specific issues and needs that arise following the decision to initiate the Kuvan trial.” I have already met my clinic’s assigned Kuvan clinical coordinator and do feel she will bring some much-needed structure to the process.
Less variation in food consumption—The last adjustment currently being considered for my second Kuvan responder trial is the possibility of eating the same foods and same portions throughout the duration of the trial. As I understand it, this second trial may need to extend as long as three to four weeks. Eating the same breakfast, lunch and dinner for that amount of time might be rather tough, but to help make it easier, one alternative I’d like to explore is re-purposing the food data I’m already tracking in the MyFitnessPal app. That same information can be used to create a list of meal combinations I can alternate between during the trial while still consuming the same approximate amount of protein.
As you can see, preparation is key! It sounds like a lot of work—which is why I am waiting until after grad school to attempt a second time—however, if it works and I am determined to be a Kuvan responder, the benefits can far outweigh a couple weeks of inconvenience.