Happy Birthday to me.
Phoenix Nest headquarters (HQ) are located at Industry City (IC) in what used to be known as Bush Terminal. A massive manufacturing and distribution plant built in the 1890’s, 16 buildings on 35 acers right off the Gowanus Canal. In the 60’s there was a mass exiting of the manufacturers at Bush Terminal and the buildings fell into disrepair. Today they have been built back up but are still in keeping with their industrial roots. My loft space is rustic; beatdown in a good way, the best feature it’s windows. It is the ideal location for a houseplant garden. PN HQ is a silver lining on the horrors of my job. I feel like I can breathe here. Jonah loves it too, there is a great Pizza joint downstairs and a foosball table in the arcade room, but mostly he likes it for the tv. My favorite addition to IC is the Little Pot of Soil, a plant shop down by the pizza joint. I think they put it in just for me. IC is my happy place; it must be as I pretty much live here. I received a B.day check in the mail from my mom yesterday and spent the whole thing on plants and pots for PN HQ. It was a good birthday.
I realize that I haven’t shared anything since Elouan passed away. I want to be able to get online and say we’re all done, Sanfilippo has been cured and we can all go home now. The reality is that research and drug development take a long time. There are hundreds of opportunities for setbacks. We try to control everything we can, but research is unknown, that’s why it’s called research.
For example, our IIID Enzyme Replacement Therapy (ERT), All-027 program enzyme didn’t like its food or temperature and wouldn’t grow. It took us a year to figure out the right food and temperature combination. That stretched our grant out an extra year. We ended up doubling the length of our IIIC mouse dosage study for JLK-247. Our mice were supposed to come down at 32 weeks, but we decided that the behavioral dated would be better supported with additional survival data. We extended our study to 70 weeks and just now took the rest of the mice down. Which again sucked up more funds. If only the money just flowed… Our third therapeutic study, AVP-6 hasn’t run into any major challenges. We’ve gotten lucky thus far. Knock on wood.
Last summer we won a NIH grant to support the observational study for ALL-127 (Natural History Study for IIID). The startup of this clinical trial has been exactly what I expected it to be, terrifying! I’ll go ahead and break it down for you. We have our main contract research organization CRO, Labcorp/Covance, these guys build the electronic Case Report Forms (eCRF) for us and store the data that comes out of the observational trial. They write the informed consent forms (ICF) and provide medical writers for our clinical protocol. They make sure we don’t miss anything and keep every letter in compliance with our regulator’s guidelines. The paper trail involved is astronomical. At the end of the study, they will do all the statistical analysis. All this costs just as much as the clinical trial itself. Secondly, we have the hospital site, they see the patients and perform all the assessments listed on the clinical protocol that we designed. After each patient they input all the medical report results into the eCRF. We have two additional CROs that have helped us design a real-world real-time app. We call it the ‘RARE’ app. For the app we chose several daily activities for the caregivers to video record, this too, is a methodical research project. On top of that we will have a retrospective study where the patients will provide our study site with their medical records. The primary investigator (PI) will comb through the records and pull out our needs. The patients will come to NYC for a total of 3 visits over the course of three days for two years. The kids will go through a battery of clinical and cognitive assessments. First Patient In (FPI) was supposed to be in March we’re looking at September/October now. A few things happened, first we decided to move sites from Yale to Columbia. Secondly, was the change of cognitive assessments for the protocol.
Our All-127 grant is milestone based, one of the milestones was to present our clinical protocol to the FDA, for comments. The comments took me by surprise. I must hand it to the FDA; they did a great job providing insightful and helpful ideas and advice. It appears that they have learned a lot from the Sanfilippo trials that have gone before me. The San A and B trials have had a rough go at it. Most of them have shutdown entirely or divested, not necessarily because the drug wasn’t working, but because the sponsors didn’t have the bandwidth to give the drug enough time to show change. I don’t think the sponsors fully appreciated how much time and resources that they would have to commit to their trials. For Sanfilippo it has become abundantly clear that we must put as much time and resources into designing the trials as we put into drug research and development. The FDA was kind enough to give me some ideas on how we might do things better than our predecessors.
Based off the FDA’s comments and my mother’s intuition, I pulled out all but one of the clinical outcome assessments that were considered the gold standard for Sanfilippo trials. Breaking the mold is terrifying, I’m ‘Scared all the time but doing it anyway’. This print is hanging in my hairdresser’s salon. It resonated with me. Wish us luck!
Thank you for all your support and generous Birthday
donations. I want to be able to translate our patient facing documents to as
many languages as possible. So that we can enroll more families into our RARE
app video assessment. I'm a little shy of my goal. Just saying. https://www.facebook.com/donate/965299684110213/10158889015667215/
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| Print by Margaret https://www.margaretstolte.com/ |
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| My new snapback, Bulbasaur hat, a Bday present from Jonah. |
