I have quite a bit of news today. There has been a fair amount of conversations happening since the conference call in December but I didn’t want to post until all the information was gathered. Soon after the conference call and after some time had passed as to when we were expecting to hear about the efforts being made to get CDCA into the US, we got in touch with Rare Disease Therapeutics(RDT) and asked them for any news. We were able to speak to Bo Allen’s assistant who informed us that RDT was not working on any such effort. We were told that it is Dr. Kenneth Setchell who is spearheading the effort.
We were a bit surprised as this differed from what we had discussed during the conference call. Dr. Setchell is the director of the Clinical Mass Spectrometry Laboratory at Cincinnati Children’s Hospital Medical Center. Not content to leave it at that, we contacted Dr. Setchell to get his side of the story. He did corroborate what Bo’s assistant said that he was in fact working on getting CDCA into the US in bulk form and organizing an operation to get CDCA to patients. He is the Dr. whom I have heard is treating some patients with Cholic Acid. We asked him about this and he stated that he does treat his younger patients with Cholic Acid, apparently they have a good response with Cholic Acid. He said that as a patient gets older, he recommends moving to CDCA therapy. He mentioned that his supply of Cholic Acid is drying up which, along with the scarceness of CDCA has motivated him to undertake this effort. He did say he is also willing to help our cause in any way he can. I have a feeling we will all get to know Dr. Setchell in the future.
Angie and I discussed this information and were unable to put it all together to determine what was really going on with everything. It was nagging at me and so I persuaded her to call Bo Allen at home last night. Her conversation with Bo was very enlightening. It turns out that Bo has left RDT and is now partnering with Dr. Setchell in forming a company to get CDCA made here in the US. This corroborates an e-mail I sent on Wednesday to Dr. Salen asking about the differing bits of information that we were geting. Bo said that Dr. Setchell is currently in Australia trying to raise money to this off the ground. Bo said they need about $1M to get going.
There are 3 different drugs they are going to target with this effort. One is Cholic Acid, one is CDCA and I can’t remember the third but it’s used to treat pulmonary hypertension in infants. There are of course numerous government regulations and processes that must be followed to get this to all fall into place. One of those processes involves a detailed manifest about CDCA. To get this manifest together is quite expensive and time consuming, up to 3 years. Bo was hedging on not having to draft a new one but to be able to obtain one already created. He intends to contact Solvay who previously manufactured Chenix which was the retail name for CDCA when it was available within the US.
We spoke with Bo over the weekend about this and it turns out that he is 99% sure he will be able to get this set of documents from another source and not have to draft his own set. Some other news to report is the cost of CDCA once it becomes available. According to Bo, there is a government requirement that each patient have a ‘maintenance'(not the official term) fee each year of 5 to 8 thousand dollars. This would price most, if not all of us, right out of being able to obtain the medication. We suspect that grants will be responsible for keeping the medication attainable to patients with such a barrier in place. I should also mention that even though this is moving now, that it could be 2 years before the first set of capsules rolls out of the lab.
Bo said that he thinks that the Falk foundation will likely manufacture 1-2 more batches of CDCA before they halt production. We did not ask how much a batch is and how long it would last. In th event that we are facing imminent unavailability of the medication, Bo said that there are some emergency measures we can take under the guise of his new company to get the bulk CDCA and distribute it prior to all of his requirements being completed for his company. He says that Dr. Setchell’s lab has the capability to put it in capsule form in such a situation.
Lastly I should mention that Dr. Salen did send us a list of patients who are interested in becoming involved in our organization. We are going to be contacting each of them and will be posting information about that as it comes in. That’s all I can think of for now. If there are any details I left out, I will update later.