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ShopGuy

Phase 1 Trial of New PD Treatment: My Experience

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ShopGuy    87

Last Friday, I finished the five-day inpatient portion of a Phase 1 clinical trial for BIIB054, an immunotherapy treatment for PD being tested for the pharmaceutical company Biogen. Details of the trial (still recruiting at some locations, I believe) are here:

https://clinicaltrials.gov/ct2/show/NCT02459886

Overall, the experience of participating in the trial has been very worthwhile. As the saying goes, there were stretches of boredom punctuated by brief moments of--okay, certainly not terror--but heightened anxiety.

To prepare for the trial, I was required to stop some medications, and undergo blood and urine tests, an EKG, an MRI, and a DaTScan. Blood, urine, EKG, and MRI were to confirm good health (apart from PD); the DaTScan confirmed my dx. Cost of all testing (including DaTScan) was covered.

Infusion of the study drug was done during a five-day, four-night inpatient stay in a research clinic. The study drug (or placebo) had already been administered in higher doses to 40 volunteers without PD, and a handful of volunteers with PD, with no significant side effects. My MDS is one of the study doctors--I felt the potential benefits for research and for myself outweighed the modest risks. I was well compensated for travel and time, and the catered meals were *far* better than hospital food.

Day 1 was a battery of blood and urine panels, plus multiple EKGs, blood pressure readings lying down and standing up, MDS exams, and collection of cerebral spinal fluid via lumbar puncture (spinal tap).

I was more than a little nervous about the spinal tap, but it turned out to be much less of a big deal than I imagined. Very little pain (just a little poke from the lidocaine injection, and a bit of pressure when the needle went it). After that, I was hooked up to radio telemetry to continuously monitor heart rate for the next 48, and spent the next several hours in the clinic's waiting room. By the evening, when we moved to the clinic annex where the bedrooms were located, I had a fair amount of discomfort in my lower back. This may have been a reaction to the lidocaine. I was given ibuprofen, which provided good relief, and was fine the next morning.

There was another volunteer participating in the trial at the same time--that, plus books and internet, was a good distraction from a whole lot of sitting around waiting for the next blood draw, the next EKG, etc.

Day 2 was infusion of the study drug (2/3 chance, two possible doses) or placebo (1/3 chance). Infusion was via IV and took about an hour. Some combination of fasting, lying flat on my back for an hour and who-knows-what caused pretty bad heartburn during the last 15 minutes of the the procedure. When I mentioned this, I was given immediate EKG and had blood pressure checked to rule out possible complications. For the rest of the day, we had regular blood draws to monitor the drug as it moved toward and crossed the blood-brain barrier.

Day 3 was more of the same. The telemetry came off, and by afternoon, we were given the chance to take a shower and get out of the clinic for a few hours. I met up with a friend in the area and we went for a hike in a nearby park. Good to move the legs after 2 1/2 days sitting.

Day 4 was another MRI in the morning, more routine blood draws and EKGs, plus another afternoon release (for good behavior?)

On Day 5, when the results from the MRI came back, we had final MDS exams, and were released. There will be a series of followups over the next 16 weeks, including two more spinal taps and another MRI.

I was told BIIB054 isn't expected to offer symptomatic benefits, but may (if it works, and if I got the active drug) slow progression by 4-5 times the rate without the drug. As I understand it, the drug is an antibody engineered to bind with the clumping form of alpha-synuclein, and clear it from the brain. The 'moonshot' (as my MDS put it), is that the drug will prevent the formation of Lewy bodies, and even reverse some disease progression.

Needless to say, everyone at the clinic is very excited about this trial (and very appreciative of the patient volunteers). Given the lack of negative side effects, a Phase 2 trial is expected very soon (perhaps this fall), and it appears Phase 1 patients will also be eligible for Phase 2. I don't know if Phase 2 testing will be as invasive or require an inpatient stay.

We often hear that new drugs take a very long time to pass human trials and get released to market--what I heard from clinic researchers was that with good recruitment and positive results, new drugs can be released quite quickly, perhaps 4 years or less from initial human testing. I feel fortunate to have an MDS committed to active research as well as top-notch patient care, and clinical research facilities located relatively close by. In fact, my MDS will be conducting Phase 2 trials of the drug Nilotinib soon. I don't know if I'll qualify, but it's exciting having cutting-edge stuff happening so close to home.

Finally. a little soapbox: with a number of promising PD treatments in the pipeline, this is an excellent time for PWPs to get serious about research, by volunteering for trials (not for everyone, I know), and/or by contributing to organizations that support research (like the Fox Foundation), and advocating for increased support and funding for basic research through NIH and NSF. The basic research underlying all these new treatment has been twenty years or so in the making--too long a horizon to payback to expect private companies to do it alone.

 

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PatriotM    797

I'm curious about the side effects of the immunotherapy that the researchers discussed with you.  Does immunotherapy cause a risk of developing cancers?  Does immunotherapy cause a risk of healthy organs being attacked by the immune system?  Other possible side effects?

I'm curious because I see all those commercials on TV where the possible side effects for various treatments are doom and gloom. 

Thanks for posting on this important trial.

 

 

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She-Ra    63

Sounds like you were quite the pin cushion!  Thanks for volunteering for this.

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MarcB    17
11 hours ago, ShopGuy said:

Last Friday, I finished the five-day inpatient portion of a Phase 1 clinical trial for BIIB054, an immunotherapy treatment for PD being tested for the pharmaceutical company Biogen. Details of the trial (still recruiting at some locations, I believe) are here:

https://clinicaltrials.gov/ct2/show/NCT02459886

Overall, the experience of participating in the trial has been very worthwhile. As the saying goes, there were stretches of boredom punctuated by brief moments of--okay, certainly not terror--but heightened anxiety.

To prepare for the trial, I was required to stop some medications, and undergo blood and urine tests, an EKG, an MRI, and a DaTScan. Blood, urine, EKG, and MRI were to confirm good health (apart from PD); the DaTScan confirmed my dx. Cost of all testing (including DaTScan) was covered.

Infusion of the study drug was done during a five-day, four-night inpatient stay in a research clinic. The study drug (or placebo) had already been administered in higher doses to 40 volunteers without PD, and a handful of volunteers with PD, with no significant side effects. My MDS is one of the study doctors--I felt the potential benefits for research and for myself outweighed the modest risks. I was well compensated for travel and time, and the catered meals were *far* better than hospital food.

Day 1 was a battery of blood and urine panels, plus multiple EKGs, blood pressure readings lying down and standing up, MDS exams, and collection of cerebral spinal fluid via lumbar puncture (spinal tap).

I was more than a little nervous about the spinal tap, but it turned out to be much less of a big deal than I imagined. Very little pain (just a little poke from the lidocaine injection, and a bit of pressure when the needle went it). After that, I was hooked up to radio telemetry to continuously monitor heart rate for the next 48, and spent the next several hours in the clinic's waiting room. By the evening, when we moved to the clinic annex where the bedrooms were located, I had a fair amount of discomfort in my lower back. This may have been a reaction to the lidocaine. I was given ibuprofen, which provided good relief, and was fine the next morning.

There was another volunteer participating in the trial at the same time--that, plus books and internet, was a good distraction from a whole lot of sitting around waiting for the next blood draw, the next EKG, etc.

Day 2 was infusion of the study drug (2/3 chance, two possible doses) or placebo (1/3 chance). Infusion was via IV and took about an hour. Some combination of fasting, lying flat on my back for an hour and who-knows-what caused pretty bad heartburn during the last 15 minutes of the the procedure. When I mentioned this, I was given immediate EKG and had blood pressure checked to rule out possible complications. For the rest of the day, we had regular blood draws to monitor the drug as it moved toward and crossed the blood-brain barrier.

Day 3 was more of the same. The telemetry came off, and by afternoon, we were given the chance to take a shower and get out of the clinic for a few hours. I met up with a friend in the area and we went for a hike in a nearby park. Good to move the legs after 2 1/2 days sitting.

Day 4 was another MRI in the morning, more routine blood draws and EKGs, plus another afternoon release (for good behavior?)

On Day 5, when the results from the MRI came back, we had final MDS exams, and were released. There will be a series of followups over the next 16 weeks, including two more spinal taps and another MRI.

I was told BIIB054 isn't expected to offer symptomatic benefits, but may (if it works, and if I got the active drug) slow progression by 4-5 times the rate without the drug. As I understand it, the drug is an antibody engineered to bind with the clumping form of alpha-synuclein, and clear it from the brain. The 'moonshot' (as my MDS put it), is that the drug will prevent the formation of Lewy bodies, and even reverse some disease progression.

Needless to say, everyone at the clinic is very excited about this trial (and very appreciative of the patient volunteers). Given the lack of negative side effects, a Phase 2 trial is expected very soon (perhaps this fall), and it appears Phase 1 patients will also be eligible for Phase 2. I don't know if Phase 2 testing will be as invasive or require an inpatient stay.

We often hear that new drugs take a very long time to pass human trials and get released to market--what I heard from clinic researchers was that with good recruitment and positive results, new drugs can be released quite quickly, perhaps 4 years or less from initial human testing. I feel fortunate to have an MDS committed to active research as well as top-notch patient care, and clinical research facilities located relatively close by. In fact, my MDS will be conducting Phase 2 trials of the drug Nilotinib soon. I don't know if I'll qualify, but it's exciting having cutting-edge stuff happening so close to home.

Finally. a little soapbox: with a number of promising PD treatments in the pipeline, this is an excellent time for PWPs to get serious about research, by volunteering for trials (not for everyone, I know), and/or by contributing to organizations that support research (like the Fox Foundation), and advocating for increased support and funding for basic research through NIH and NSF. The basic research underlying all these new treatment has been twenty years or so in the making--too long a horizon to payback to expect private companies to do it alone.

 

Thank you, thank you for such a well written and thorough description.

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Peace    160

Thank you for sharing your experience and for taking part in this trial.

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otolorin    28

ShopGuy,

you are so blessed,may this particular trial be one of the cures for PD,amen.A big thank you  for your courage.

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ShopGuy    87
On 6/26/2017 at 4:02 AM, PatriotM said:

I'm curious about the side effects of the immunotherapy that the researchers discussed with you.  Does immunotherapy cause a risk of developing cancers?  Does immunotherapy cause a risk of healthy organs being attacked by the immune system?  Other possible side effects?

I'm curious because I see all those commercials on TV where the possible side effects for various treatments are doom and gloom. 

Thanks for posting on this important trial.

 

 

Had the second spinal tap this past Tuesday (along with blood draw and ECG).

Patriot, with regard to side effects: the consent document is 22 pp long, and goes into detail about known side effects from DaTScan, MRI, lumbar puncture, blood draws, etc (all minor).

Regarding the study drug, the document states that since this is one of the first times the drug has been used in humans, "possible serious side effects are not known." In addition, the document makes it clear that BIIB054 has NOT been tested for increased cancer risk, or interactions with other medications.

I'm not a doctor or medical researcher, but my understanding is increased cancer risk is not expected from immunotherapy (since the immune system often plays a role in preventing cancers). Cancer risk *is* a concern with stem cell therapies. Sadly, some unproven, unregulated--not to mention very expensive--stem cell 'cures' have caused cancers. It's tragic that desperate people were suckered into paying tens of thousands of dollars for a fake cure that caused real cancer.

Of course, 'cancer' isn't a single disease. I think even slightly increased risk of a lethal, incurable disease like lung or pancreatic cancer would be a big deal. But maybe increased risk of less serious, curable cancers might be worth it. My dad was diagnosed and treated for prostate cancer at about the same time he was dx'd with PD. The prostate cancer never came back, or caused any significant problems for the rest of his life (15+ years). But PD disabled him physically, then complications from it killed him. 

Anyway, none of this seems applicable to immunotherapy. As it was explained to me, immunotherapy is basically one of two approaches: active, where the patient's immune system is stimulated to produce antibodies its own; and passive, where antibodies produced in a lab are injected in the patient. BIIB054 is the second type--passive.

In some Alzheimer's trials, early active immunotherapy approaches caused runaway inflammation and killed patients. This has made researchers very cautious about active approaches, although there is an active immunotherapy treatment for PD that has advanced to Phase 2, and thus passed Phase 1 safety testing. The advantage of active immunotherapy is that only a few treatments would be necessary, after which the body's immune system would be capable of clearing alpha-synuclein on its own.

Passive approaches are considered safer in principle, I believe. But it seems more frequent treatment is necessary, since the immune system doesn't actually make the antibodies. As an aside: the injection procedure I went through was pretty clunky--an hour lying down with an IV. If that had to be repeated say, weekly, I doubt it would be worth it to anyone without advanced PD. But I really have no idea how or how often BIIB054 will be administered if it becomes an approved treatment.

When I talked to my doctor last Tuesday, he said he was surprised none of the PD patients he has enrolled and treated (four so far) experienced significant headache as a side effect of the study drug. He expected it, as a consequence of minor inflammation of brain and spinal tissues. I suppose that could mean any number of things, including that BIIB054 is safe, but not particularly effective.

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PatriotM    797

Thanks for the info ShopGuy.  That was an excellent explanation and exactly what I was interested to know. 

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Lonnise    60

David,

Thanks so much.  Very informative and hopefully resulting in bodily responses that are exactly what you want.

Has your PD been "subcategorized"?   I'm thinking maybe so, since you have family members dx'd w/ PD and other genetic markers (?) identified in your signature information.  I'm reading a number of postings and articles that support that PD clearly has different "roots/origins" one-such being an autoimmune disease.  I intend to do more "research" but I suspect my husband's "type" of PD is exactly that, autoimmune "mutiny" if you will.  Not sure if immunotherapy works for autoimmune diseases, but I'll look into it.

Again, best of luck with everything and thanks for sharing.

LHG

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ShopGuy    87

Hi Lonnise,

Perhaps my signature is confusing--as far as I know, I have NO genetic markers associated w/ PD, and neither do my family members dx'd w/ PD. Here's part of another post I wrote that may clarify:

Most people with PD *don't* have LRRK2 or GBA mutations, or any other mutations strongly linked to familial PD. My dad had PD, and my sister was recently diagnosed. That might suggest a genetic cause, but we've have also lived in similar environments, associated with somewhat higher risk. And the specifics of the disease were somewhat different for us--my dad was diagnosed in his late 60s, tremor dominate, relatively fast initial progression (very typical PD profile). My sister has no tremor, but debilitating postural instability, and gait issues. Symptoms starting ~55 y.o., undiagnosed for 4-5 years until she went to an MDS. I was diagnosed at 46, tremor dominate, very slow progression to date.

Although my dad never had genetic testing, and I don't believe my sister has had any testing yet, I would be very surprised if either had LRRK2 or GBA mutations.

Re: sub-categorizing. My guess is we're still quite a way from treatments tailored to specific PD subtypes, since research into what constitutes subtypes and and the different causes is still at an early stage.

I try not to focus much on why I (in particular) wound up w/ PD. First, because there's likely no clear answer or single cause. Second, because for me it's a little too close to a 'why me?' kind of question, and too much 'why me?' seems likely to lead to dark places.

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Lonnise    60

Hi David,

Thanks for the clarification.  I'm NOT looking for answers to "why us", that seems like wasted energy.  I am interested however in helping to customize a management plan and anticipate the expected and necessary revisions thereto as part of my supportive awareness and partnership in this journey.  I just finished reading "Being Mortal" by Atul Gawande and the questions he identifies as critical throughout one's journey of worthwhile living resonate with me.  Accordingly, I'm looking for an MDS that embraces this type of relationship with his or her patients and while we as a society may not yet have the kind of "sub-category" tailored treatments for PD, I believe it will happen and I'm hoping to help it happen during our lives.

Best of luck with your continued journey and enjoyment in living and thank you again for your courage, participation and sharing.  Peace.

Lonnise

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