I’m excited but apprehensive to write this post. Someone recently told me the expression, “knock on wood” came from the men who were healed after knocking on Jesus’s cross as he carried it up to Calvary. Makes me feel better about knocking on wood – not a superstition but rather a blessing.
So let’s knock on wood – ok?
We spoke with Dr. Yazigi yesterday regarding the results of Levi’s biopsy and MRI.
Here is what we understood going into the call:
The BSEP (Bile Salt Export Pump) results mean Levi is not eligible for the clinical trial because there was NO BSEP protein in his liver.
The MRI showed no “detectable” C.
His current labs show improved numbers with a Bili at 2.2. AST and ALT (liver enzymes) numbers in the 100’s (it is normal for this disease to have high numbers and low numbers as we have seen over the last year).
Here are the questions we asked and her responses (as best I can remember them):
Us:What are the results of the Biopsy?
Dr Y: The biopsy shows NO PROGRESSION of the disease. At 4 months Levi’s biopsy showed stage 2 fibrosis and his current biopsy shows stage 2 fibrosis. This is very encouraging and makes me feel a lot better. I do not believe that Levi’s liver will fail at any moment. He has met all of the benchmarks we needed him to and his liver disease is not progressing rapidly. This is all very good!
Us: Do these results show advanced liver disease to the point where we need to request additional points?
Dr Y: No, Levi’s liver is showing stability. At this time there is no need to request additional points. In fact, while we aren’t comfortable removing him from this list we do want to make him inactive (we ALL agreed to this!!!).
Us: Do these results change anything about the conversation we had on 6/26 regarding the fear of C?
Dr Y: No, you have to think of these two things separately. His liver disease is stable but because his liver is overworking and turning over cells very rapidly, he is at a greater risk of C. In order for me to sleep at night we need to monitor him with quarterly MRI’s (we will also only be doing quarterly labs) which is the best imaging we have available.
Us: When we were at the hospital we asked Dr. Kaufman if there was any reason to perform the same BSEP dye test on Levi’s current biopsy and he said no, the results won’t change. Is that true?
Dr Y: We don’t know Levi’s second genetic mutation. We have to treat him as an individual so I think we should absolutely stain his new biopsy. The body CAN CHANGE so we may find in this stain that he has the BSEP protein (recall from this blog post that Levi needs the BSEP protein to qualify for the clinical trial).
Us: Is the team at Georgetown moving forward with the clinical trial?
Dr Y: Yes, we believe this medication is “THE FUTURE OF THIS DISEASE.” It looks very promising and safe.
Us: Because Levi does not have the BSEP protein is he completely eliminated from this trial? Can we ask for compassionate use of this medication?
Dr. Y: The current BSEP results we have for Levi show no BSEP protein which means he is ineligible for the clinical trial however we have BSEP protein in other areas of our body (like in our intestines). I’ve asked the trial investigator if they believe this medication can attach to the BSEP proteins in other parts of the body to assist the liver. I’ve specifically asked if Levi can participate outside the trial under compassionate use (THIS WOMAN IS AMAZING)!
Us: Are there other clinical trials?
Dr Y: Not that I am aware of however there are two big transplant conventions coming up in September and November. If anyone presents anything that could help Levi, I will find out about it there and let you know where to go so Levi can participate.
Us: What is the plan going forward?
Dr Y: Change Levi’s status to inactive on the transplant list. Monitor him quarterly with labs and MRI. Manage his itchiness as we have been unless there is notable change. Stain his current Biopsy to confirm he does not have BSEP protein in the liver. Wait for the trial investigator to provide us with answers.
So What Now?
To say we are extremely encouraged is an understatement! This is an answered prayer y’all! Levi’s liver is stable. This doesn’t mean he won’t ever need a transplant, this just means he can keep his liver longer (unless he is eligible for the medication and then he may never need a transplant).
In all the research I’ve done and support groups I’ve polled, the longer Levi can keep his native liver, the better. Most people feel that you are trading one fatal disease (PFIC2) with another (transplant) because immune suppression is no joke. So if we can avoid transplant for as long as possible, that is what we will do, and we will PRAY that God will provide healing through His mighty hand or through the hand of the scientist who created this medication.
For I will restore health to you, and your wounds I will heal, declares the Lord. -Jeremiah 30:17
The Clinical Trial
Many of you have asked questions about the clinical trial so I will do my best to provide as much information as possible! Please remember that I am not a medical professional and at this point have LIMITED knowledge about this.
The clinical trial presented to Georgetown is for a medication. It was presented to them by a Pharmaceutical company. The medication attaches to the BSEP protein in the system (from what I understand), even non functioning protein, to carry the bile out of the liver. It does what the liver cannot do itself. This clinical trial is ONLY testing to see if the medication will reduce itching with “side effects” to include reduced risk of fibrosis and cirrhosis, no need for transplant and reduced risk of C (because it’s helping the liver get rid of the bile).
This clinical trial is in Stage 3 and Dr. Y believes the medication to be very safe for pediatrics.
So what about Levi? The biopsy from when he was 4 months old shows NO BSEP protein in his liver. We are testing his current biopsy. We have BSEP protein in other areas of our body (like in our intestines). We are waiting to find out if this medication can attach to the BSEP protein in other areas of the body to assist the liver. Dr. Y has asked for compassionate use of the medication for Levi if he does not have the protein in his liver! We are waiting for answers from the trial team.