My research identified Mass General Hospital (MGH), in Boston, Massachusetts, as one of the facilities involved in BrainStorm’s Phase 2 clinical trial. Although MGH was a 6-hour drive from home, I was determined to be involved in the Phase 3 clinical trial and felt that my best opportunity would come as a patient there. I met my doctor at MGH in March of 2017. After a full review of my medical records and additional examinations and tests, she reconfirmed my ALS diagnosis. I made my desire to enroll in the upcoming Phase 3 trial known to her at that time. After a few long months of waiting, the trial was approved to start in August of 2017 with MGH being the first site to enroll patients.
On the day the trial was approved to proceed at MGH, my doctor called me and scheduled screening tests and a full review of the trial procedure. Just a few weeks after our 15th wedding anniversary, we found out that I had been selected as one of the first patients to enroll in the Phase 3 clinical trial. I completed my screening visits and was informed I was eligible to continue in the trial in October of 2017. During that two-month period my ALSFRS-R declined enough to meet the inclusion criteria and, although it meant that I didn’t have an extremely slow progression, my family and I were so excited to be able to continue in the trial.
At my fifth visit later that month, I had my bone marrow aspiration done at MGH. This is a relatively commonplace procedure and is not unique to BrainStorm’s NurOwn® treatment. Two small incisions were made on the left and right sides of my lower back and two technicians simultaneously removed stem cell-containing bone marrow from my pelvis bone. The procedure was performed under general anesthesia and after a short recovery I was discharged later that same day. I was expecting more discomfort from the procedure but overall it wasn’t painful at all- I was back home that evening.
Even before I left MGH that day, my cells were already on their way to City of Hope Hospital in Duarte, California. City of Hope is the where all the stem cell-containing bone marrow samples are sent for all the participants in the trial, regardless of whether patients ultimately end up receiving the NurOwn® treatment or placebo (Dana-Farber Cancer Institute in Boston was added as a second manufacturing site in October, 2018). No one at MGH or any of the trial sites knows who is getting treatment or placebo, or is in any way involved in the decision-making process. That process is absolutely necessary to ensure the integrity of the trial results- neither the patients nor their doctors can know who is receiving treatment in a double-blind study.
In early December, five weeks after the bone marrow aspiration, I returned to MGH for my first intrathecal injection. This was done right in my hospital room using local anesthetic. The procedure itself took about 15-20 minutes and then I was required to lay flat for at least 4 hours to avoid a headache. I was observed for 24 hours for any adverse events and then released. I only had minor back ache in the middle of the night, more from stiffness than anything procedure-related. I returned home and resumed my life but with a new sense of optimism. With ALS, you guard your optimism very closely, but it felt good and exciting to be doing something.
In early January I had my first lumbar puncture to take a sample of my cerebral spinal fluid. This was done in the doctor's office, again under local anesthetic. My wife was even in the room. I had no pain and just had to lay flat for about 45 minutes after, again to avoid headache. After that, I was free to leave and we drove back home.
I had my 2nd injection in early February, with a follow-up lumbar puncture at the end of that month. These procedures went as smoothly for me as the first ones, but unfortunately, I then hit a road block in my trial schedule. Right after this trip I developed a blood clot (DVT) in my leg, completely unrelated to trial procedures. It was extensive enough that BrainStorm required I take some time off from the trial to give the clot time to resolve.
I got the all-clear in June and went back in July for my 3rd and final injection, followed by my last lumbar puncture in August. I then had a follow-up visit in September to do some blood work and an ALSFRS-R rating scale measurement, and had my last follow-up visit on October 19, 2018. Although I'm glad to have finished the trial, my wife and I will miss seeing the staff at MGH on a regular basis. They are a first-class organization and have given me the best care, attention and support an ALS patient could hope for.
As for the results, I can't say that I've seen any evidence that I received the stem cells. I feel that my disease progression has been steady for the duration of the trial, and I would've expected at least a temporary pause in progression for a short period after each injection. Of course, I could be wrong and perhaps my progression is slower than it would've been otherwise. In the end, I am choosing to believe I received the placebo, because I want to believe that this treatment will be powerful enough to be noticed. For my sake and for all the ALS patients who need something to hope for I would prefer that I got a placebo rather than an imperceptibly effective treatment. And despite feeling this way, I don't regret the time I spent in the trial or its restrictions. I was able to be involved in what I believe was the most promising ALS treatment trial available to me and I will be forever grateful for the opportunity.