The NeuRx Diaphragm Pacing System,

[notgivnup]

NeuRx Diaphragm Pacing System Approved for ALS

I just saw this from MDA,

The NeuRx Diaphragm Pacing System, a device that may forestall the
need for invasive ventilation, has been approved by the FDA for ALS


Article Highlights: •The NeuRx Diaphragm Pacing System, developed by Synapse Biomedical, has been approved by the U.S. Food and Drug Administration to treat respiratory insufficiency in ALS.
•The system is designed to supplement breathing efforts and help preserve diaphragm muscle function; it may delay or negate the need for invasive tracheostomy ventilation and can be used in conjunction with noninvasive ventilation.
•The pacing system will help people with ALS who still have some preservation of the diaphragm (the main muscle used in breathing) and also in the nerves that normally control its movement.
•The device will be available for physicians to prescribe in the near future.

 

[MuonOne]

notgivnup, et alia;

Here is their full web page on this news:

NeuRx Diaphragm Pacing System Approved for ALS
NeuRx Diaphragm Pacing System Approved for ALS | MDA/ALS Newsmagazine

ALSA also announced this news:
FDA Approves NeuRx Diaphragm Pacing System (DPS)®
FDA Approves NeuRx Diaphragm Pacing System (DPS)® - The ALS Association

 

[sadiemae]

I will try to find the article about this where is says that only about 10% of PALS will actually benefit from this. Christopher Reeve had one, and was able to go off his vent for short periods when the Pacer was turned on. I was so excited about this two years ago, however the doctors at several ALS clinics were not so excited. The research head at Forbes Norris was not very enthusiastic about it. She said Les would not be a good candidate because:

"In our experience, it worked for some people and not for others. It did not appear to work well for people who had already lost a lot of diaphragm function, like Les."

This was over a year ago, and his FVC was at 70%.

Here is the article: http://alsn.mda.org/news/diaphragm-pacing-system-awaiting-fda-approval-als


Receiving a Humanitarian Use Device (HUD) designation is a first step toward a possible FDA Humanitarian Designation Exemption (HDE), which allows a company to market a device to a specific population. The HUD designation establishes that the NeuRx diaphram pacing system is a medical device intended to benefit people with a condition that affects fewer than 4,000 people in the United States per year. (ALS affects greater numbers than that, but only a fraction of those have stimulatable diaphragms, meaning they have some preservation of the diaphragm muscle and the nerves that normally stimulate it.)

For those who do not have remaining phrenic nerves, which go to the diaphragm, or whose diaphragm muscles are too deteriorated, the device is not likely to have any benefit. So, as good as this may sound, it is only going to benefit a very small portion of PALS.

 

[mare]

I'm still very interested in this; have been following it.
As with most things, it is thought to be better to get early. Will continue to explore.

 

[sadiemae]

Same here, we see the Pulmo at the VA on Monday, and I have been discussing this for 2 years with him. Up till this point, they wouldn't even do the EMG of the Phrenic Nerve, since it had not been approved. I did SO much research on this 2 years ago, and am glad to see it is finally approved, but disappointed still that it will only work for less than 10% of PALS. I, however, have not done any research in a year on this, so maybe things have changed.

 

[MuonOne]

The ALSA website reference says ". . . an estimated subset of 3,300 with both respiratory problems and intact phrenic nerves that could benefit from the NeuRx DPS® treatment," which I think means about 500 to 600 people each year who develop ALS in the United States are expected to be medically suitable for this 'treatment.' I suppose you could multiply that by twenty to reach a worldwide guestimate or divide by ten to reach a Canadian guestimate (approximately fifty to sixty Canadians per year)?

 

[MuonOne]

I just realized that apparently you cannot edit posts here . . .

In the last post I intended to add that I think these figures are conservative, that in practice more than this number of people will find the practice to be helpful. The news from those attempting to participate seemed to imply that the ineligible count was much lower than 90%, further, fewer PALS are likely to disqualify because they are 'too progressed.'

 

[sadiemae]

More info:

How is the diaphragm affected by ALS?
With ALS, your nerves’ ability to signal your muscles decreases over time. This also affects your breathing nerves (phrenic nerves), which lose their ability to carry the message from your brain to your diaphragm. Ultimately, ALS weakens your diaphragm. As your diaphragm weakens, you develop a condition called chronic hypoventilation, which means you’re no longer drawing enough oxygen into your lungs.
Your ALS physician determines when you have developed chronic hypoventilation by using standard breathing tests. These tests include;
Forced Vital Capacity (FVC)
Maximum Inspiratory Pressure (MIP)
Arterial Blood Gas (ABG)
Oxygen Saturation (SaO2)

What are the Main Tradeoffs and Risks?
In order to obtain the potential benefits of NeuRx DPS®, you will need to undergo a routine laparoscopic surgical procedure with four short incisions to have the electrodes implanted in your diaphragm (breathing muscle). All surgery involves risks including the risks of anesthesia and infection. With this procedure, there is a risk of abdominal air getting through the diaphragm and going into the chest cavity during the surgery (capnothorax). Most of the time, the problem goes away on its own but sometimes it requires placement of a small tube in the chest for a short time and extra time in the hospital. With ALS, there is also the small but real chance of respiratory (breathing) failure as a result of surgery, at which point a ventilator would be needed.
http://www.synapsebiomedical.com/als/als-patientInfo.shtml

 

[John1]

A fuller quote from the website reads:

Approximately 30,000 people in the United States live with ALS. More than 5,600 new cases are diagnosed each year, with an estimated subset of 3,300 with both respiratory problems and intact phrenic nerves that could benefit from the NeuRx DPS® treatment.

It is ambiguous but my reading of that indicates that well over half of new cases would benefit from the pacer. That is, the subset refers to the 5600 cases, not the 30,000. If it refers to the 30,000 then Lori is right and the device is being hyped too much.

 

[sadiemae]

John, that is what the folks at Forbes Norris led us to believe. It is being hyped to much. The way I see it is it is for the newly diagnosed only. Which is good(actually GREAT) but for the more progressed PALS, it is just like the the drug trials, "You have had ALS to long, and we can not help you." For all the recently diagnosed folks, take advantage of everything you can NOW. A year from now, you will not be eligible for anything. I pushed SO hard 2 yrs ago to get Les the Pacer, but it was not approved for PALS yet and we could not get it. Now it is too late, but for those of you who CAN qualify, GO FOR IT. And also see if you can get into the NP001 trial. We did the Phase 1 , and were promised we could do the Phase 2, but, they lied to us, and we do not meet the criteria. And, good to see you again John! HUGS Lori ( So cool to have people call me by my real name. Neat that you remember it.)

 

[MuonOne]

John1, yeah I think you have identified the confusion in the various news sources and my interpretation is that the estimate is intended to be represented as 3,300 per year. This new method may be more popular that traditional vents but everyone is eligible for those. I doubt that all those who are eligible for the NeuRx DPS will go for it but I wonder if more people who are eligible for NeuRx DPS will go for it than people have gone from traditional vents. Traditional vents have been reported to be less than 50% acceptance and at least one source indicated its actually pretty close to only 10%. There are a few people who are doing both but I wonder if eventually there will be more people going NeuRx DPS than there are traditional vents . . . or will the NeuRx DPS be more comparable to 330, that is about one per day.

Of course, the fda has not said this technique is effective, the recent news is that the fda has said that this technique is not unduly dangerous and therefore is use is eligible for some promotion . . . I guess it is similar to being in a Phase III status.

 

[tmasters]

I asked about this at my ALS Clinic appointment today. Dr. Mozaffar said there are no local (So Cal) surgeons yet trained on the procedure but he's hoping that this gets rectified soon. But he feels I am a candidate for this and is hopeful that by the end of the year he has a better answer and can get me scheduled soon after that.

Another option is travel to Stanford or Cleveland.

He likened the benefits of the pacer to early bipap usage in ALS, in that it helps slow the decline of the diaphragm muscle. But he said it does not halt progression.

Now as to whether my HMO will cover it, that's another matter!

-Tom

 

[rknt50a]

I'm not a healthcare professional, but it seems to me that if the pacer can help a person maintain strength and operation of the diaphragm muscle, that would be all for the better even if decisions regarding bi-pap and venting were to follow later. A thought fwiw.

 

[BrianD]

I've been in contact with the closest facility to me, which is Madonna in Lincoln, Nebraska which is currently in process for receiving approval to do the procedure.
The information I received was that to qualify for the procedure you need to have an FVC of 50% or lower OR an MIP of 60 cmH2O or lower. In addition, an EMG to determine if your diaphragm can be stimulated.
So I'm not close enough to those numbers yet, so I'll see where I'm at the first of the year to see where my numbers are then.

 

[John1]

...So I'm not close enough to those numbers yet, so I'll see where I'm at the first of the year to see where my numbers are then.

Brian, I think the recommendation is to get the pacer sooner rather than later. I believe it acts to slow the inevitable decline of diaphragm function so the sooner you get it, the longer it has to slow the decline.