Hypothetical - Medical Trial

[BlsdMama]

Northwestern is doing stem cell trials on Stiff Persons.

To be clear, I am *not* at the point of making this decision. 

 

We are still waiting on an assay from Mayo to absolutely confirm or deny SPS and we have a couple more trials to do - #4 being plasma exchange and #5 being chemo.  However, the particular type of SPS Mayo suspects I have and/or will progress to (glycine receptor positive / PERM) doesn't meet good ends if it doesn't respond to treatment.  But this has, in the past 1-2 weeks, clearly advanced and is becoming more and more like SPS and less like Motor Neuron Disease.  (MND is painless, SPS is painful.  I am actively spasming in my back daily and last night was pretty rough as I had painful leg spasms for the first time ever.)

How desperate would you have to be?  A chance at being completely and totally well (your own stem cells ironically) and a 25% chance of fatality due to secondary infections.  Obviously your chances are better the healthier you are.   I would meet all the qualifications - I am antibody positive and IVIG and Rituxan trials have failed.

It's kind of a catch 22.  The earlier on, the better chance of surviving.  But 25% fatality - wow, that's a hard thing to sign up for.  How do people even make these decisions? Especially people with itty bitty kiddos.  ?

[Katy]

I have a relative who's going through a stem cell trial.  He's been disabled for years and his quality of life has declined so much he's losing the will to fight.  Deciding to go for it is as much, I think, an acknowledgment to him that it's worth the risk because he won't live long anyway, and quality of life is more important than lifespan to him.  His children have been grown for more than 25 years, they are stable adults who don't need him.  He sold his business so he has no employees depending on him anymore. He's in a different stage of life than you are. His older relatives are all dead. He's very devout in his religion and isn't at all concerned that death is the end. I think he is concerned with being a burden to his wife rather than helping her.

If/when you progress to the point that you're seriously wondering how soon this might kill you, you might feel dramatically different about the risk/benefit ratio.

[Pawz4me]

I'm confused, I think. Are you trying to decide between a medical trial (still technically experimental, recruiting patients, being conducted at various hospitals across the country or world, etc.) or are you choosing between two treatment options? The first is how "medical trial" is used in our experience in dealing with DH's cancer. But the way I'm reading your post you mean those are your two current treatment options?

But for either scenario I'd want my doctor(s) advice. They know you and your personal health situation the best. I'd want to know the odds of a cure (or complete/durable response--however it's termed for your condition) versus the odds of fatality, What are the odds of developing a non-fatal secondary infection that requires long term treatment? Would the necessity of treating a non-fatal secondary infection potentially mean withdrawing from the trial/stopping the treatment? Are there other potential side effects besides secondary infections? I'd probably have many other questions after researching each trial/treatment option, but those are the basic ones that pop in my mind right off the bat. Before joining a trial or deciding on a treatment I'd want to thoroughly examine and weigh ALL the available information (obviously), not just the fatality risk. I would also think that possibly someone would be able to give you some more detailed info on that fatality risk--were the patients who developed fatal infections health compromised in some other way, did they tend to be older than you, etc.

Contrary to what many people believe, medical trials are often far from the last resort for a patient. More often than not they're a chance to get either the current standard of care (treatment) for a condition versus a newer, more promising treatment with a LOT more monitoring than one might get with standard of care treatment.

[Pawz4me]

1 minute ago, Arctic Mama said:

Well,  Benny is going into a stem cell trial at Duke, and he’s just a baby. There fantastic treatments and fairly low risk, for the potential payoff.  We are comfortable with it ?

AM, I'm less than 90 minutes from Durham and in a few weeks we'll be moving DS22 into an apartment there. If there's anything we can do for you while you're in the area--do not hesitate to contact me! 

[prairiewindmomma]

We wrestled with some of those same questions. You make the decision with the best info that you have and then be at peace that you did the best you could at making the decision.  Dd did two phase 3 trials, and one phase 1 trial.  We turned down a couple of trial offers after extensive discussions with the investigators.

[BlsdMama]

2 hours ago, Pawz4me said:

I'm confused, I think. Are you trying to decide between a medical trial (still technically experimental, recruiting patients, being conducted at various hospitals across the country or world, etc.) or are you choosing between two treatment options? The first is how "medical trial" is used in our experience in dealing with DH's cancer. But the way I'm reading your post you mean those are your two current treatment options?

 

Not too much is known or understood about the glycine receptor antibody.  It was discovered about ten years ago.  It was thought that being GlyR+ meant PERM.  Now they know 4% of folks walk around with it with no issues.  *But* it appears I have Stiff Person.  So currently Mayo believes (right or wrong) that my Stiff Person will progress to PERM unless they stop it.  PERM is fatal - though sometimes acute and sometimes long term.  They die from an autonomic crash usually - meaning you don't always (often, but not always) get sicker and sicker until you crash. So it's a little tricky deciding to go for a trial because you can't define, "Well, if I experience X, Y, and Z, then I'm doing it."  Does that make sense?

The first thing they tried was a small dose of IVIG for 11 weeks.
The second was two rounds of Rituxan (rituximab)
Now I am on a much higher dose of IVIG weekly.  There is no improvement.
Next they will try plasma exchange - mine out and someone else's in.
Finally, cyclophosphamide (a form of chemo) will be on the table.

The Northwestern trial is new to me - I only just heard about it.  My Mayo team is not medically conservative.  My (local) neuro here commented a couple of weeks ago, "They are either going to kill you or cure you," and he spent eight years at Mayo. 

 

[ktgrok]

1 hour ago, Arctic Mama said:

Thank you so much, I will keep that in mind!   We have to travel up to four times to Duke during the duration of the study.  Benjamin was slightly too young for the criteria but they are way low on qualified volunteers and we are crossing all fingers they they’re willing to expand  the age criteria (since he needs to be two and will be so in just a few months, and it’s a multi year study trial).  So far it’s looking like a yes!

 

Stem cell treatments for cerebral palsy have shown fantastic results and we could never afford this on our own, so it’s an amazing opportunity.

As an aside, if you will be staying in a hotel make sure to tell them you are there for the hospital - most in the area give a discount for Duke medical center patients. My mom had her lung cancer treated there and raves about the place. They still go up every 6 months for rechecks. 

[Jean in Newcastle]

If I met the requirements and was at the beginning of a diagnosis and thus healthier than I would be if it had progressed, then yes, I would do it.  The medical trial that my dd is in gives us the right to drop out at any time.  Are you allowed to drop out if needed?  

[ktgrok]

I'd want to know if there were other factors that influence who gets a secondary infection. And what the protocol is to minimize that, and is it something you can adhere to. A friend is looking into a similar thing for her MS and she has to stay at the hospital, quarantined, for a while I think, which would be hard with your kids. So look into that? 

[Pippen]

Is the 25% fatality rate for all sites involved, or the fatality rate at Northwestern?  Be sure and ask for that percentage, because it may vary considerably. 

[ktgrok]

10 minutes ago, Arctic Mama said:

I didn’t know that, thank you!  There is some travel reimbursement, but it won’t cover it all.  Small price to pay for the treatments, though.

You are welcome! We've used it when traveling to Shand's hospital, it seems many larger teaching hospitals have this arrangement with the hotels. Or the hotels just offer it, something. The travel allowance with our insurance about matched what the reduced rate was, if I remember correctly. 

[Desert Strawberry]

I did a drug trial, years ago, before marriage and kids. It didn't work, and I had a bad reaction, but it was well worth and one of my three treatments did work. It was a low risk trial and super worth it. I'd do another one in a heartbeat. 

I think at this stage, you don't have a truly definitive diagnosis yet? So you can't say for sure that you would be eligible or that it would work, if I'm reading correctly? If that's right, obviously, you have to wait until you know for sure? 

If you are eligible and the other treatments are not working, by all means do it. I can't tell you what to do, but in your shoes I would absolutely do it. Yes. It's a chance worth taking IMO.