Dear experts, my son has DDD and have been treated with Soliris since January. He feels much better, his sC5b-9 value has gone down from 560 to 319 within 2.5 months, although the C3 value has dropped from 27 mg/dl to 18, but proteinuria and haematuria are almost away. So, obviously, Soliris is good for the kidneys, but it further confuses the immune system. Anyway, the most important thing is that my son feels much better.
However, our insurance has approved the costs only for 6 months. My question is: In case we fail to prolong the treatment, what is a correct way to get off Soliris? Can we stop the treatment abruptly or should we prolong the intervals before we stop? What happens with the immune system after we stop? Is it true that the kidney function gets worse after getting off Soliris compared to if one has never taken Soliris? And is it true that if we get Soliris some months or years later again, it will not work the same wonderful way like this was at the first time?
Thank you so much in advance!
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MPGN-C3G
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Our Expert Panel's response to your questions are below. We hope this will be helpful!
These are very challenging and very important questions.
At this point we do not have conclusive data about how well eculizumab works for C3G. Nor do we know for sure how long it should be continued. Many physicians who treat C3G have seen cases that seemed to get better after eculizumab was started, similar to what you describe for your son. Patients like him - who get better quickly after starting treatment - encourage us that the treatment is helpful in some cases. It is important to remember, though, that it is possible that these patients might have gotten better anyway. Similarly, there are patients who do not get better with eculizumab, but it is possible that the treatment was started too late in those cases or that the disease would have progressed faster without treatment. Given all of these uncertainties, we think that it is very important that more clinical trials are performed.
Eculizumab blocks a portion of the complement system (part of the immune system). When the treatment is stopped, the entire complement system can become activated again. If eculizumab has been helping, it is therefore possible that the disease will become more active after treatment has stopped. There are some clinicians who taper the treatment off, prolonging the intervals. However, even if you do not taper treatment, when you stop eculizumab, it takes many weeks for the drug to disappear. Therefore, there may not be any benefit to slowly discontinue the drug. None of the members of this panel tapers the drug off or prolongs the dosing intervals, although this question has not been specifically studied.
Because we do not know with certainty that eculizumab is beneficial for this disease, we also do not know how well it works for retreating disease flares or disease recurrences. Patients who seemed to benefit from treatment have stopped treatment without any adverse effects. There are also patients who have successfully been re-treated after their disease recurred, but others in whom re-treatment has not been helpful.
Given how many questions about the disease remain, however, we believe that all patients should be followed by a nephrologist (even if they are in remission). The members of this panel are also strongly supportive of new studies so that we can learn the answers to the questions you have posed. Fortunately, there are new studies underway to test new treatments, some of which might be better suited to treat C3G, as they are designed to block the complement system in a way that will focus on the cause of C3G. We are optimistic that our understanding of this disease will continue to improve through these studies.
Dear David, dear Experts, thank you so much for the detailed and honest answer! Although it does not clear up what exactly we should do, it helps me a lot. I know there are too few studies to prove something, but my feeling has been and remains that we should try everything to prolong the renal function until better drugs are on the market. And: in some cases Ecuizlumab does not help because it is too late. So, if we do not think about the extremely high costs, I think trying Ecuilzumab is worth it before it is too late because it might help. My next question to the Experts coud be (but I do not want to bother them again): When can we expect the wonderful new drugs for C3G?
My son is 7 and too young to take part in a study. So, we have to wait and hope that his kidneys are not completely destroyed by then. So, in case anybody in this forum knows more: When will the new drugs be available? (In particular in Europe!) Rather in 2 or 5 years or longer? Will they be affordable? Should we expect that they will completely switch off the immune system by blocking the process of opsonization?
Dear David, thank you so much for managing this wonderful forum! I wish you all the best!
I cannot give you solid answers on time frames - I know it can take a few years to complete trials and get approval for new drugs, but I believe there are also ways to expediate this process for diseases, such as ours, with no other treatments available & high need. And, there are going to be differences in the way this happens in different countries. Plus, that assumes the drugs actually work as intended, which isn’t always the case. Overall, I think it is just hard to predict. Maybe David or someone with more experience with new drugs could give more info on that.
I do know one drug started patient trials several months ago, and another is getting ready to start right away. I have heard that this second drug is expected to be targeted more at European countries, though a US trial is being done as well. I believe (but could be incorrect) that it may be a little faster/easier to get the drug to market in Europe vs the US, hence the reason for more of a “push” there.
Although children aren’t included in trials at this point, it is something that has been discussed to some extent. So, keep your eyes and ears open, as the opportunity may eventually come.
My understanding (that again, could be wrong!) is that the drugs currently in trials work to block the effects of the convertase proteins that extend the C3 cycle in C3G patients. This is what leads to the over-production and over-use of C3 that ultimately causes damage. I believe the theory is that this will not suppress the immune system’s ability to fight infections as eculizumab does.
As for cost - my thought is that drugs for rare diseases are always going to be expensive. The time required to research and develop, & a small patient market to profit from, is going to drive up cost. There also isn’t much competition, with no or few alternative drugs available to patients. I’m not sure a low-cost treatment will ever be available for us, at least not for a long time until competition can drive down cost. Maybe someday.
The good news is that the new drugs offer more variety and convenience in dosing - pills, injection, etc, rather than strictly IV infusion.
Dear linfuller, thank you a lot for your answer! Are you a doctor yourself?
I think, too, that the drugs tested in Europe will come to the market here sooner than others. In Germany, e.g., all new drugs also have to go through the ethics commission and this takes months and years.
It is interesting that German nephrologists whom I have met do not know a lot about the new drugs. When I heard about ACH4471 and Avacopan for the first time a year ago, no one knew about them here. Even now they ask ME about news in this field. It looks like I have a better access to the information through social media and my personal communication with Carla Nester Or maybe because they have only a few patients with our disease they cannot spend too much time for this topic.
So, please keep us further informed via C3GN/DDD Warriors. Thank you very much for your efforts!
On behalf of the Expert Panel, thank you for your post after their response to your question. It is very gratifying to know that the Expert Panel feature is appreciated.
I can answer your question about timing of new drugs- it’s the same answer as linfuller gave in her excellent response to your most recent post: it’s not possible to say when these drugs will be available to patients. Bringing drugs to the market (i.e., to patients) is fraught with difficulties and uncertainties. One never knows how effective a drug will be (and therefore, when it will be ready for use by patients) until it’s been tested in lots of patients for an appropriate amount of time.
The good news is that, as was noted by the Experts, the drugs being developed for C3G are specific. This means they block specific molecules in the complement system. Drugs that target specific molecules often have less side effects.
You can find out more about clinical trials in C3G by visiting clinicaltrials.gov. This is not the most user-friendly site, but with patience and attention to detail, you can figure it out. You can search for C3G, glomerulopathy, DDD, or the drug company of interest.
Regarding including children in clinical trials, linfuller is correct- there is talk about doing this. I don’t know whether the current trials for C3G therapies will include kids. None of the trials include children under 12 years old.
Thank you again for being engaged in the C3G online community!
Dear David, thank you so much for the quick reply and the valueable information! It is hard to find a nephrologist who knows our disease well. Our doctor said she would be greatful for any advise. So, the Expert Panel is a very important sourse of information for us and, I believe, will soon become it for many more people worldwide. Thank you for this opportunity!
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