Chapter 14: Boston
The good guys seek to find a new champion.
I’m flying through the sky at 600 mph and I wish it were because I’m actually Superman. He surely wouldn’t have had trouble putting his luggage in the overhead compartment above our cramped seat.
Meanwhile, somewhere 40,000 feet below me is a father, son, mother, daughter, husband, wife, sister and brother who all woke up on the wrong side of serendipity. If you are truly connected to ALS then you know the news I speak of.
The promising stem cell therapy, now ten years in the making, known as NurOwn received another devastating set-back as the Food and Drug Administration advised the drug’s parent company, Brainstorm, to reconsider submitting its Biologics License Application due to failure to exceed the traditional threshold of “substantial evidence” from its recent phase 3 trial data.
Jamie and I were both disheartened to hear this statement from the FDA whom many passionately plead to on our behalf. Others in the ALS community, whom we consider to be friends and allies, were downright outraged. And rightfully so in my opinion.
The tales stemming from this trial over the last decade bordered on miraculous. Legends were born as men stood from their wheelchairs to walk again. BIPAPs were disposed. Progression appeared to be halted.
Alas, when the trial concluded there were too few responders to meet the trials primary endpoints and subsequently the baby was thrown out with the bathwater. NurOwn was labeled a total failure by the medical community at large. No mind that a subgroup of early onset PALS actually saw improvement over placebo.
Can you imagine being diagnosed with a terminal illness, then thrown a lifeline only to have it snatched away upon seeing actual improvement in your condition? I personally cannot fathom that frustration, but it’s a reality for the responders in that trial.
Jamie and I from our room at what would soon become my home away from home, The Liberty Hotel in Boston.
Now I’m looking out the window and the magic of human flight is not lost on me. Even in the daytime, at this height above the clouds the sky looks almost black, a reminder that we’ve transcended what we once thought was the entirety of our universe. What we once thought was impossible. A setting sun only adds to the heavenliness of it all.
For all our flaws, we are ingenious creatures; consumed not only by survival but also by curiosity and progress. I have no doubt that we will transcend reality again and with that the constraints and limitations of human biology. It’s only a matter of time. The question is whether or not it will it happen in my lifetime?
At this moment, somewhere in between Boston and Kansas City, I’m wearing a smile because my wife and I just met the woman who can help us take flight so to speak, and accomplish the gravity defying miracle equivalent; beating ALS.
I’ve already discussed at length our issues with local neurology and the unacceptable stance of complacency and acceptance. You should not be surprised at this point that Jamie and I did something about that. A family connection and a very pregnant wife both expedited what we once thought an impossible task; an appointment with the world’s leading ALS specialist at the epicenter of all ALS research, Massachusetts General Hospital.
Together with our new quarterback, we hashed out our strategy going forward and cast a wider net around all the promising research trials in the pipeline. I even discovered another medication that could potentially help with my speech and swallowing present day.
She too shared in our disappointment about the NurOwn news. With pictures of patients covering her office wall and a hand in the trial itself, this doctor knew what it meant for the community. That all important hope. We even discussed our common grievances about the many flaws in ALS research. Namely, that in the eyes of the regulators, ALS is still seen as one disease which it most certainly is not. How else can you explain the extreme variability in onset, progression, and drug response? There’s also a BIG problem with trial efficacy endpoints hinging on a subjective 12-question scale that has no nuance or relevant biomarkers.
But my discouragement was tempered after our appointment at Mass Gen. It would appear clinical trials are becoming more sophisticated and tackling pathology head on (where it used to be more downstream). Soon subgroups and potential responders will be identified earlier from onset, allowing potential therapies to find their true targets and consequently leading to better data and better results that even the FDA cannot deny.
When we touchdown in Kansas City I expect the scenery will look a little different than before we set out. This week we signed our very own Patrick Mahomes. And the future just got a little brighter.