Reflections – The Day After

Hi, All

Yesterday – 15 November 2021 – I received my first infusion of Nexviazyme – the recently-approved Enzyme Replacement Therapy (ERT) from Sanofi-Genzyme.

This followed nine years of Lumizyme infusions (also a product of Sanofi-Genzyme).

This milestone transition followed over two years of discussions, evaluations, research, negotiation, and a whole new battery of baseline studies in preparation to move on from Lumizyme. This long process included me abandoning my former Pompe specialist and finding a new one who would be more responsive to my changing condition and needs.

In late 2018 – early 2019, I began to plateau on the beneficial effects of Lumizyme, a fairly common experience among Pompe patients who have received that drug for seven or eight years. Together with my doctors, I considered increasing my Lumizyme dose, as many Pompe patients have done after plateauing. By the time we actually faced that decision, a new drug was already in clinical trials under the lab name, “Neo-GAA” – an acronym meaning “new GAA.” GAA is the abbreviation for the enzyme, acid alfa glucosidase, the deficiency of which causes Pompe Disease.

My new Pompe specialist informed me that she had experienced denials from insurance companies when she tried to adjust the Lumizyme dosage for other Pompe patients who had plateaued. Because higher doses of Lumizyme deviated from the prescribed standard of care, some insurance companies would not approve an alternate dose. Because Neo-GAA was nearing the end of its clinical trials, my doctor invited me to consider waiting for the new drug. She felt that, because my body was already not responding as favorably to Lumizyme as it once had, administering more was probably just delaying the inevitable. Because Neo-GAA was an entirely different composition, my doctor felt that my body would likely respond more favorably. So we decided to wait.

Neo-GAA was originally scheduled for FDA approval in May 2021. For unexplained reasons, the FDA decided to delay that decision by three months. In August 2021, the FDA approved Neo-GAA at which time it was assigned the new brand name, Nexviazyme (avalglucosidase alfa).

A month later, I met with my Pompe specialist, and she agreed to write the orders for the switch from Lumizyme to Nexviazyme. I began this blog at about that time to document this process. Readers can review the blog posts to see how that process unfolded. It took a while.

 But yesterday, the stars aligned, and I received my first Nexviazyme infusion.

Today, I want to share with you the noticeable differences between the way my body has historically reacted to Lumizyme and the way I’m responding to Nexviazyme.

Ever since my first Lumizyme infusion in 2012, I have had a consistent response to that drug. Lumizyme infusions took about four hours to administer. Beginning about an hour or two into the procedure, I would feel a gradual onset of fatigue. The physical fatigue was accompanied by a state of mental and emotional sensitivity. It’s difficult to think of a suitable comparison but I would become sensitive to light and sound. I would also find it difficult to reason clearly or make decisions. After my first few Lumizyme infusions, I would set the stage for my homecoming by having my dinner planned and prepared so I wouldn’t have to cook or decide what I was eating. It was also common for me to get home, turn down the lights and maybe light some candles, and put on soft music. I would avoid anything, like TV news, that would trigger anxiety. I would basically retreat from the outside world and, if possible, remain so for the day following my infusion. That heightened sensitivity would typically last a full 24 hours.

I have a few Pompe friends who made the switch from Lumizyme to Nexviazyme over the past few weeks and they have reported feeling energized after receiving their first Nexviazyme infusion. Some have speculated that this may be due, in part, to the fact that Nexviazyme is mixed with a dextrose solution (versus the saline solution used with Lumizyme). Dextrose is a sugar which would reasonably give patients a mild energy boost. One Pompe friend asked her doctor about this theory and her doctor said that dextrose wouldn’t really explain much of an energy boost. Whatever the case, I went into yesterday curious to see if I would have a similar reaction.

Yesterday was a very long day, relative to my normal patterns. I was up at 5:00am and went through my normal morning routine. Because I was expecting a long stay at the infusion center, my sister came by in the morning and picked up COPPOLA, my service dog, so he wouldn’t be confined all day. I left for the infusion center at 9:00am and stopped to get some lunch to bring with me. I arrived at the infusion center a little before 10:00am and my infusion was underway by 10:40am. The infusion ended at 3:00pm and I had to remain at the center for a two-hour observation period. I left at 5:00pm and was home by 5:15pm.

I was very tired … but not at all in the way I’m used to after a Lumizyme infusion. I was just tired from a long day. I put away all my gear, my sister dropped by to return COPI, and I settled in. After getting a drink and catching my breath, I began to notice a distinct difference – not just compared to a Lumizyme evening but to any other evening. My mental acuity was much sharper. After about an hour at home, I felt motivated to prepare dinner, sort through emails, return messages, and check in with my family. My energy wasn’t only up, I was feeling exhilarated and social – VERY different from a regular evening and certainly different from an evening following a Lumizyme infusion.

I slept great last night and today, that mental acuity and energy have been sustained.

One element that, for me, is consistent with both Lumizyme and Nexviazyme is my dream time afterwards. Following Lumizyme infusions, I would have super-vivid technicolor immersive dreams with epic storylines. When I woke during the night last night, I was delighted to find that Nexviazyme infusions are followed by similar dreams. Fortunately, I’m not a nightmare person. I have marvelous life-affirming dreams … where I’m not bound by the constraints of Pompe Disease. In my dreams, I can run and leap and fly. So being able to enjoy turbo-charged versions of dreams like that after infusions is a perk!

Enzyme Replacement Therapy (ERT) is not really about an immediate response. ERT nourishes the deficient system of a Pompe patient with normal levels of enzymes that enable our bodies to efficiently process glycogen. ERT is a long-term project. So ultimately, the efficacy of this new drug – Nexviazyme – will be revealed over months and years. But after nine years of feeling completely depleted after ERT, if this new treatment means I’m going to feel mentally and emotionally uplifted, I can get excited about that. I already am!

So, this story will unfold as it does. But after one Nexviazyme infusion, my report is that I feel great … better than I’ve felt in a long time. And since this blog is intended to inform fellow Pompe patients of what they might expect when facing the transition from Lumizyme to Nexviazyme, my report is decidedly favorable.

~ Brian

Infusion Day 1.8

5:30pm (PST) – 15 November 2021

Home Sweet Home

My first Nexviazyme infusion is complete and a success.

I completed my two-hour observation period at 5:00pm and am now home and unpacked from a very long day.

I’ll debrief further tomorrow. There are some very noticeable differences already between Nexviazyme and Lumizyme … rather positive ones, I think.

For now, though, I’m going to settle in … turn on Monday Night Football … and call it a day!

I’d like to thank all of you again for the good wishes, affirmations, and encouragement today.


~ Brian

Infusion Day 1.7

3:00pm (PST) – 15 November 2021

Nexviazyme Infusion #1 Is On The Books!

My second bag of meds just finished up. I’m now starting my two-hour observation period.

They’re keeping my IV in with a dextrose solution drip for the first hour of my wait. This is to keep my infusion line open in case I have a reaction. That way, they can immediately infuse me with Solu-Medrol. I rather doubt that’s going to happen. Today has gone very smoothly.

It looks like my total infusion time on this new drug, even with the slower increases in the first hour, was just about four hours. By comparison, the total infusion time for my Lumizyme infusion was about three hours and forty minutes. So pretty comparable.

I’ll admit, though, that I’m not feeling particularly energized. I had a lot of energy invested in this big day and I’m pretty tired. But I’m also feeling like today was a big success.

I’m sure gonna sleep well tonight!

Thanks again for all of the good wishes on social media, emails, and messages today.

I’ll plan to do one more entry to end this long day, when I’m settled in at home. Fortunately, I live less than ten minutes away from the infusion center.

~ Brian

Infusion Day 1.6

2:00pm (PST) – 15 November 2021

In the Home Stretch!

About an hour ago, we completed the infusion of my first bag of Nexviazyme. Remarkably, it took just two hours and twenty minutes to finish that half of my overall infusion. When infusing with Lumizyme, my first back typically took about two hours. Because we were using a slower infusion rate with Nexviazyme, I had expected that first back to take considerably longer.

Typically, my second bag of Lumizyme would take a little over an hour and half. I’m now almost an hour into my second back of Nexviazyme. So we’ll see how long it take to finish this up.

This first day on the new drug requires that I stay at the infusion center for a two-hour observation period following the completion of the treatment. So I have quite awhile yet to go today. Even though I’m not that excited about a two-hour wait afterwards, I’ll be feeling a great sense of satisfaction in having gotten through this first treatment.

Timing aside, all is going well with this first infusion of Nexviazyme. All of my vital signs are stable and at good levels and I’m feeling fine.

There has been so much anticipation around this day and I’m sure I’m going to be pretty tired when I get done today.

My spirits are really high, though, and I’m looking forward to getting home this evening after this long day.

Infusion Day 1.5

12:15pm (PST) – 15 November 2021

I’ve reached my maximum infusion rate of 210ml per hour.

All vitals are good.

I’m feeling good too!

I’ll stay at this rate until this first Nexviazyme infusion is done. Probably in an hour or so, I’ll reach the end of my first bag of medication and we’ll switch me over to the second.

Hope is Flowing, big time!

~ Brian

Infusion Day 1.4

11:40am (PST) – 15 November 2021

Just completed my first hour of my first Nexviazyme Infusion!

All systems GO!

We just increased my infusion rate to 150ml per hour. In a half hour, I’ll be at my maximum rate.

All vital signs are good!

The hospital pharmacist just stopped by to say hello. She is relatively new to Sierra Nevada Memorial Hospital but we’ve met and talked before. She reported that mixing Nexviazyme is so much easier than Lumizyme. Both of these medications need to be reconstituted by hand. Lumizyme was packaged in smaller vials and the pharmacy had to hand-mix forty vials for my dose. Nexviazyme is packed in larger vials so the pharmacy only has to mix about half that number. So they’re very happy about that. The Pharmacist also told me that my second bag of meds is already mixed and delivered. So when this first bag is done, we’re ready to move right over to the second. No waiting!

My first four years of Lumizyme infusions were administered at UC Davis Medical Center in Sacramento. They’re a huge operation and have three separate infusion centers. During my time with them, they were infusing eight or nine Pompe Disease patients as well a several with other Glycogen Storage Diseases (e.g., Fabry Disease, Gaucher’s Disease). They were a well-oiled machine at UCD.

Since I moved back home to Grass Valley in 2016 and relocated to Sierra Nevada Memorial Hospital, I’m the only patient at this hospital with Pompe Disease (or any GSD, for that matter) and it’s a big deal for this relatively small hospital to be treating me here. Over these five years, all of the infusion nurses, the pharmacy staff, and the hospital administrators have taken a real interest in my case, my care, and these treatments. They’ve been remarkably embracing of treating me and I’ve made a lot of friends here.

When I lived in Chico, the local hospital – Enloe Medical Center – refused to take on this medication or to administer my infusions. That decision was based at least in part on the exorbitant price of these infusions – roughly $75,000 each. The fact that my hometown hospital didn’t bat an eye at taking on this treatment and the way they’ve always worked tirelessly to accommodate me has always made my infusions here seem special.

As I mentioned in an earlier post, there’s a lot of excitement here today as we start this new treatment!

Feeling very grateful today!

~ Brian

Infusion Day 1.3

11:10am (PST) – 15 November 2021

First 30 minutes completed without incident.

Infusion rate increased from 30ml to 90ml per hour. We’ll go another 30 minutes at this rate and, if all goes well, we jump up to 150ml per hour. The max rate for this infusion is 210ml per hour. When we hit that rate, we stay there until the infusion is done.

We take my vital signs at each rate increase – blood pressure, heart rate, temperature, oxygen saturation levels. Everything is in normal range and I’m feeling great!

All ahead full!

I’ll check back in 30 minutes at my next increase.

btw … thanks for all of the supportive comments on FB today!

~ Brian

Infusion Day 1.2

10:40am (PST) – 15 November 2021

Nexviazyme Infusion #1 Underway!

I’ll infuse at 30 ml/hr. for 30 minutes then ramp up to 90 ml/hr.

This is a slower rate than I was using with Lumizyme. My increases were every 15 minutes on Lumizyme but we’re starting with a slower rate on this first Nexviazyme infusion, just to be safe.

Fingers Crossed!

These past two years have led to this moment … and we’re off and running!


~ Brian

Infusion Day 1.1

10:20am (PST) – 15 November 2021

I’ve arrived at the Infusion Center.

My only premedication today is Tylenol, which I’ve now taken.

My IV is started and I’m on a drip of dextrose and water. I believe we run this dextrose drip for 30 minutes before beginning the actual infusion.

My first bag of Nexviazyme has been mixed and delivered so it’s ready to go when we reach 30 minutes. I’m a big guy. I’m 6’4” and weigh about 250 lbs. Because this medication dose is mixed based on body weight, my overall dose is relatively larger than the average. These enzyme infusions are very viscous and are known to clog the inline filters between the bag and my arm. Because my dose is so large, they divide my infusion into two bags, each with its own filter. That reduces the possibility of clogging the filters … which would require that we halt the infusion and replace the filter.

I had the timing of Lumizyme infusions down to about five minutes. Today, we start a whole new protocol, so I have no idea how long this is going to take.

There’s quite a bit of excitement at the Infusion Center today. I’ve worked with this team for over five years, and they are all as invested in the start of this new treatment as I am!

More later!

~ Brian

Infusion Day 1.0

8:10am (PST) – 15 November 2021

I’ll be leaving for the infusion center in about 45 minutes.

And as I sit in the quiet of my office, I can’t help but reflect on the path that has brought me to this place … this moment.

I was diagnosed with Pompe Disease in May 2012 after living 26 years believing I had Muscular Dystrophy. That discovery opened the door to a treatment that could stop this progressive condition from worsening. I received my first Lumizyme infusion that September.

I remember the enormity of that September day. I recall feeling like my dreams had come true. I had literally woken up one day to learn that I no longer had MD. I had something else – something called Pompe Disease – but this was treatable. I sat in that infusion center and received my first Lumizyme infusion and felt, like I do today, that things were about to change … that I was given a second lease on this life that I cherish so deeply.

Every two weeks for the past nine years, I’ve returned to an infusion center to receive an enzyme replacement. And these Lumizyme infusions have lived up to their promise. They saved my life from the rapid decline I was experiencing in 2012. I’m here today, writing this entry, because of Lumizyme. These treatments stabilized my condition and gave me a decade of life I otherwise would not have had.

A little over two years ago, though, that stability began to falter. The progressive weakness that had been an everyday part of my life leading up to 2012 started to return. Very slowly over a series of months, I wasn’t able to reach as high as I had been. My grip strength was failing, and I began to drop things more.

I promptly reported these experiences to my Pompe specialist. I came to learn that it was not uncommon for Pompe patients who had been receiving Lumizyme for many years – so-called long-haulers – were increasingly reporting a plateauing effect. The stability they (no, we) had been experiencing began to fade and the muscle weakness began to creep back in.

I learned that many Pompe patients responded to that plateauing effect by increasing their Lumizyme dosage. Some began to infuse weekly; some began to receive double doses. Despite my urgent pleading, my Pompe specialist wouldn’t consider making those changes for me. As I’ve had to do at several junctions in my life, I took it on myself to pursue some changes. I contacted the leading Pompe specialists at Duke University and consulted with them. They provided me with the latest published papers on this plateauing effect and how to adjust dosages to address the problem. I provided these studies to my Pompe specialist – to no avail. She continued to delay.

About a year ago, I found a new specialist. And for the past year, we have compiled all new baseline measures in anticipation of the release and approval of a new enzyme replacement therapy. Originally called Neo-GAA in clinical trials, that new drug was approved three months ago and given the name Nexviazyme. A month after Nexviazyme received FDA approval, I met with my new Pompe specialist, and she approved me to transition from Lumizyme over to this new drug.

These past couple of months have involved a lot of medical bureaucracy … approval of the new drug by my hospital … insurance approvals … billing approvals … and volumes of paperwork to complete each of those steps. My first Nexviazyme infusion was originally scheduled for October 18th. That was delayed to November 1st. And that, in turn, was delayed to today – November 15th. This past Thursday, in the final hour before my hospital had to order my medication for today, the final approvals came through and opened to door to today’s Nexviazyme infusion.

In the next couple of hours, I’ll begin that infusion. Today will be my 238th enzyme replacement therapy. Today will be my first Nexviazyme infusion. I’m confident all will go well today. Even so, I can’t ignore the enormity of this moment. Everything is riding on the success of this new treatment. It’s not an overstatement to say that today marks the threshold of the rest of my life.

Time to sign off. Time to leave for the infusion center. My next entry will be from there … with an IV in my arm … and a bag of Nexviazyme hanging from a post next to me.

Off I go!