Neuronata-R is a stem cell treatment produced by Corestem that is very similar to the NurOwn treatment (by BrainStorm) for which I was in the on-going phase 3 trial. A treatment cycle starts with a bone marrow aspiration (BMA) to collect mesenchymal stem cells (MSCs) from the patient’s own bone marrow. Those MSCs are then manipulated by Corestem’s patented process and re-injected into the patient’s spinal fluid 4 weeks later. The process is repeated so one treatment cycle consists of two BMAs and two stem cell injections. The schedules for each BMA/injection combination overlap so that one treatment cycle looks like this:
Week 0: First BMA
Week 4: Second BMA, First MSC injection
Week 8: Second MSC injection
The main treatment difference between Neuronata-R and NurOwn (separate from the treatment’s potential effectiveness) is that BrainStorm has developed a means to effectively freeze the MSCs (either before or after manipulation-not clear on that) so that one BMA can provide stem cells for many treatments. That would obviously be a huge advantage for NurOwn, as Neuronata-R currently requires one BMA 4 weeks prior to each and every injection. Again, however, that is not considering or comparing each treatment’s effectiveness but only the practical aspect of the burden of the treatment.
Published results here show only with one treatment cycle (2 BMAs and 2 injections), the ALSFRS-R scores of those receiving the treatment changed by a mean of -1.36 points, while those on placebo changed by a mean of -4.67 points after 16 total weeks from the first injection. Looking at the graphical data provided by Corestem it appears that there is hardly any change in ALSFRS-R scores 2 months after the first injection for the treated group compared to about -2.5 for the placebo group. This shows a significant impact by Neuronata-R on ALS disease progression, although the effect appears to wear off after about 3 months (12 weeks) after the first injection. After that point the rate of ALSFRS-R score decline appears fairly similar between treated group and placebo.
Neuronata-R appears to be a very powerful treatment (perhaps unparalleled during the first 8 weeks following the first injection) but it is extremely expensive and would require ongoing treatments that are currently only available in Seoul, South Korea. This trial did permit the use of riluzole, so one major question to answer is whether adding the other treatments in the NeufStrong protocol will improve the benefit of Neuronata-R or extend it so that treatment cycles can be spread apart to save treatment cost and travel expenses.
This treatment is the primary reason for our fundraising efforts. My doctor at Mass General Hospital presented this option to me as one to research. As I investigated I could not find a good source of information online from a patient’s perspective, so I decided to create my own as I expect others like me would be interested.
The process started with a letter of support from my doctor to her contact at the Cell Therapy Center at Hanyang University Hospital in Seoul. Since I will not be told whether I received stem cells or a placebo in the BrainStorm trial until it is complete in mid-2020, we had to first confirm that still being “blinded” in this way would not preclude me from receiving the Neuronata-R treatment. Previous stem cell treatment presents an unknown potential interaction risk with any subsequent stem cell treatment, but fortunately this was explained as a Neuronata-R product warning and not a firm exclusion factor. I then requested to start anytime after October 19th, the day I (finally) completed my BrainStorm Phase 3 trial experience.
About a week later I was emailed several documents including:
It is from the Estimated Total Costs ($90,000-$100,000 USD minimum) that the amount for our fundraiser was developed. I selected a tentative schedule option and then a copy of my medical record was also requested, in addition to the required lab tests. I arranged for the blood test samples to be taken at an upcoming previously-scheduled clinical trial visit at MGH and those tests were covered by insurance. Knowing that transferring medical records is difficult enough from hospital to hospital just in the U.S., I requested a copy of just my ALS-related medical records and scanned and emailed them to the clinic myself along with the lab test results when they were complete.
I received confirmation that my lab results and records were satisfactory and that I was approved to move forward with my first visit in November 2018. I left on November 21 for my first visit to the clinic on the 23rd. I’ve documented all of my visits; see the links below.