• Effectiveness

  Major (for amyotrophic lateral sclerosis)
• Side effects

  Mild (for Overall) (Increased fasiculations during & after infusions, drowsiness, low red blood cell count, headaches, increased muscle twitching (fasciculations) during and post infusion, fatigue)
• Adherence

  Always
• Burden

  Somewhat hard to take

Dosage: 1 other Monthly

Advice & Tips: 11/28/11 I am now in the 5th week past the very last trial infusion.  Since infusion cycles are generally 28 days, I think of the first 4 weeks after the last infusion to be within the dosing portion of the trial.  I just "missed" the first repeat cycle last week.  As had occurred all 6 cycles, I sensed the drug wearing off beginning about 3 weeks after infusions.  Unfortunately, this time I get nothing to stop the resulting negative effects. This graph below shows my ALSFRS-R history, with the x-axis adjusted to 0 days as my first NP001 trial infusion. Points are plotted through the complete dosing portion.  The green vertical line was the first infusion.  Red is the last infusion. My FRS progression was historically very linear, with a correlation ceofficient = 0.94.  On this trend, my natural progression should have resulted in an FRS = 32 at the completion of the dosing phase, but I ended at 38, 3 points higher than when starting.

Cost: < $25 monthly

Side effects: headaches, drowsiness, fatigue, low red blood cell count

• Effectiveness

  Major (for amyotrophic lateral sclerosis)
• Side effects

  Mild (for Overall) (Increased fasiculations during & after infusions, drowsiness, low red blood cell count, headaches, increased muscle twitching (fasciculations) during and post infusion, fatigue)
• Adherence

  Always
• Burden

  Somewhat hard to take

Dosage: 1 other Monthly

Advice & Tips: It's been 6 days since my last infusion cycle, #5. I am now 120 days into the trial. My speech recovered some the first day, and more the 2nd and 3rd days. My swallowing has still been better the entire trial. My walking improved some again, from the low point last break. My hands do seem a little weaker than when I began the trial. Overall, my progression has certainly at least significantly slowed over 17 weeks, and I say this versus improved, since my hands seem weaker. But my ALSFRS-R is truly higher than when I began, by 4 points. My progression had been steady to where I should have been expected to lose 3 points.

Cost: < $25 monthly

Side effects: headaches, drowsiness, fatigue, low red blood cell count

• Effectiveness

  Major (for amyotrophic lateral sclerosis)
• Side effects

  Mild (for Overall) (Increased fasiculations during & after infusions, drowsiness, low red blood cell count, headaches, increased muscle twitching (fasciculations) during and post infusion, fatigue)
• Adherence

  Always
• Burden

  Somewhat hard to take

Dosage: 1 other Monthly

Advice & Tips: I am currently 4 days away from my next infusion cycle, #5. For the last three days, my speech has worsened to where I am having to repeat a lot more with family. It has been 24 days since my last infusion, and this speech worsening has very predictably occurred now 4 times, about 1 week before the next cycle. But, in May, I was exclusively using Proloquo2go to talk, and for months. So, I maintain an improved condition, even at the low points. My swallowing is still great, and I don't notice a cycle with it. My walking has gotten more stiff over the past week. I plan to, again, video record my current condition (talking, drinking water, walking, standing from sitting and crouching) and then again 1 week after dose cycle 5. I did this a out 6 weeks ago, at a high point, and long distance relatives were amazed at my improvements since seeing me months earlier.

Cost: < $25 monthly

Side effects: headaches, drowsiness, fatigue, low red blood cell count

• Effectiveness

  Major (for amyotrophic lateral sclerosis)
• Side effects

  Moderate (for Overall) (Increased fasiculations during & after infusions, drowsiness, low red blood cell count, headaches, increased muscle twitching (fasciculations) during and post infusion, fatigue)
• Adherence

  Always
• Burden

  Somewhat hard to take

Dosage: 1 other Monthly

Advice & Tips: It has now been 13 days since my 4th cycle ended. I am 14 weeks into the trial, 100 days tomorrow, and 14 total doses. I am still BETTER than I was in May! I have always gotten very tired each infusion cycle, and have generally napped for about 3-4 hours later each day to recover from extreme tiredness and often a headache. Circumstances prevented naps on days 2 and 3. On day 3, I rushed to the airport and travelled solo. I had an all day meeting on the next day, then travelled home solo that same day, getting home about 10 pm. I was greeted by 9 out-of-state relatives ready to help us move out of our home the next day. The combination of all of this was brutal! The day after infusions, while incredibly excited about the meeting I participated in, I felt ill. My speech was very bad, walking very difficult, etc. I was very concerned. After finally settling in to our new home, one week after the last cycle, everything is back to the state it was. No further improvements, but I still maintain those realized earlier. My parents were among my visitors and stayed for over a week. Even as far back as December 2011, they could understand nothing I said. We had many conversations during their visit with my own voice. Repeating was necessary, but what a difference from 9 months ago!

Cost: < $25 monthly

Side effects: headaches, drowsiness, fatigue, low red blood cell count

• Effectiveness

  Major (for amyotrophic lateral sclerosis)
• Side effects

  Mild (for Overall) (Increased fasiculations during & after infusions, drowsiness, low red blood cell count, headaches, increased muscle twitching (fasciculations) during and post infusion, fatigue)
• Adherence

  Always
• Burden

  Somewhat hard to take

Dosage: 1 other Monthly

Advice & Tips: Completed cycle #3 three days ago (11 total infusions now) . Each cycle seems easier. I still had tiredness each day and a headache one day, but the short lived side effects seem less severe with each cycle. I mentioned previously that the effects plateau and then some regress slightly. The minor regression was quickly restored after only 1 infusion. My speech, after being improved for most of the time between cycles 2 and 3, regressed a few days before cycle #3, but improved again to where I am not using a computer to speak to family. For the first time since meeting the study nurse at the screening visit in May, I was able to converse without a computer, with some repeating. I've used my own voice to talk to my wife and mother 3 times on the telephone recently, for the first time in perhaps a year! My walking also seems more natural than it did after the last cycle. Overall, I have continued improving, but probably less significantly at cycle #3. My FVC/SVC has shown steady improvement as well.

Cost: < $25 monthly

Side effects: headaches, drowsiness, fatigue, low red blood cell count

• Effectiveness

  Major (for amyotrophic lateral sclerosis)
• Side effects

  Mild (for Overall) (Increased fasiculations during & after infusions, drowsiness, low red blood cell count, headaches, increased muscle twitching (fasciculations) during and post infusion, fatigue)
• Adherence

  Always
• Burden

  Somewhat hard to take

Dosage: 1 other Monthly

Advice & Tips: It has now been 22 days since the end of infusion cycle #2 (8 total infusions).  It is 4 days before cycle 3 begins.  As an update to the above: I have not used a cane to walk in 22 days.  I have no sense of imbalance any longer.  My gait is still abnormal and walking is slower, but is not a worrisome experience as it had been.  I have been using an iPad and Proloquo2go (AAC application) to speak for months now.  This had become necessary even for close family over the last couple months.  In the last 2.5 weeks, I have not needed to use my iPad to speak with family, until the last 2 days on occasion.  I even talked quite often throughout each day.  Swallowing liquids and solids has even improved slightly since the previous update. It has been months since I couldn't crouch without loosing balance, and had been impossible to stand up without assistance from that position for several months.  I now can again, and can even lie on the floor and stand up unassisted. I also believe my emotional lability is improved, as I can now contol laughter, without continuing for embarrassingly long durations. I have sensed improvement at each cycle so far, both during and shortly after.  Improvements seem to plateau soon after each infusion cycle and maybe even regress very slightly prior to the next.  But, overall the net result is definite overall sustained improvement each cycle.

Cost: < $25 monthly

Side effects: headaches, drowsiness, fatigue, low red blood cell count

• Effectiveness

  Major (for amyotrophic lateral sclerosis)
• Side effects

  Mild (for Overall) (Increased fasiculations during & after infusions, drowsiness, low red blood cell count, headaches, increased muscle twitching (fasciculations) during and post infusion, fatigue)
• Adherence

  Always
• Burden

  Somewhat hard to take

Dosage: 1 other Monthly

Advice & Tips: Update after 7 Infusions (not quite 2 full cycles): Efficacy: Substantially improved swallowing, increased tongue strength (moving food), increased tongue range of motion (eliminated food from collecting in areas, firmer muscles around lips (restored partial pucker and eliminated slight drool and leaking when drinking, blew out candles for 41st birthday in late June, couldn't at 40th!), more productive cough, increased range of motion with right arm at shoulder (vertical possible, was limited to horizontal), increased range of motion with neck (was less than full leftward, now normal), increased leg strength (restored ability to rise from sitting without pushing with arms), increased arm strength (much improved ability to sit up from lying flat and to turn in bed), slight increased leg coordination and balance (no reliance on cane for balance), greater dexterity in left hand (was difficult to sign number 3 with all fingers together, now easy), intermittent clear enough speech to talk fluently without reliance on iPad. Side Effects: Tiredness and occasional slight headache within hours of infusion. Wake rested, but repeats at each cycle ad builds up. After multiple doses, tiredness lasts a couple days and slight hung-over feeling. Fasciculations worsen during dosing. Frequent smiling due to amazement over unanticipated improvement!

Cost: < $25 monthly

Side effects: headaches, drowsiness, fatigue, low red blood cell count