Daratumumab for my SFN?

I will try daratumumab for my small fiber neuropathy

Hi everyone good to see you back here! 🌺   

I have some good news, so i thought I'd write a quick update.  

I just received a letter from my health insurance yesterday, saying that daratumumab has been approved for an off-label treatment trial of my autoimmune small-fiber neuropathy - yeii! 

But let's take a step back....

What is daratumumab?

Daratumumab is an antibody treatment, which is licensed to treat people with a specific kind of blood cancer, where cells of your immune system, which usually produce antibodies to protect you from infections, start to grow out of control and clog different organs in your body. This cancer is called multiple myeloma. 

This is daratumumab. Brand name of the drug is Darzalex and it is produced by Janssen

This sounds scary, but actually many treatments that are used to treat autoimmune diseases have originally been developed to treat blood cancer.... This is because the origin of autoimmune diseases, are cells of the immune system, which are mainly present in blood. 

Why daratumumab?

In case of my neuropathy, it is assumed (but there is not so much research out there yet) that these same cells, called B-cells, for some unknown reason, started to produce antibodies, which attack the small nerve fibers in my skin. This is why it's called an autoimmune disease - because cells of my own immune system produce antibodies which attack a part of my own body. 

This is also what happens in different autoimmune diseases, some of which you might have heard of, such as lupus, multiple sclerosis, auto-immune kidney disease, or rheumatoid arthritis. In every disease, these cells produce different kind of antibodies, which attack different systems in your body, and in my case they attack the small nerve fibers in my skin. 

Of course it is more complex than this and there can be many ways how an immune system can go awry. A lot of it is probably still unknown even to the experts, but this is the basic concept of what happens in my case. 

So recently, there have been reports about other types of autoimmune diseases, which have been treated by daratumuman. Here is a link to a case report of two women with lupus who were treated with daratumumab - just for the science nerds amongst you. 

How did we get the idea to try daratumumab?

Daratumumab is not a drug you usually come across as a pharmacist, because it is used in a very specialized field of medicine (hematology mainly) - so it was obviously not my idea to use it. 

But one of my lovely friends, who is a hematologist (so she treats people with blood cancer and other blood diseases) had previously used this drug to treat people with autoimmune blood diseases (I know I have really clever friends💕), in which antibodies attack blood platelets or red blood cells. And hematologists know this drug, because they use it to treat blood cancer. 

So she was actually the one, who suggested that daratumumab may be worth a try for my neuropathy. So i told my neuro Prof. about this drug, and he was actually really interested. He agreed that this approach made a lot of sense, but he also warned me that getting this approved by health insurance will be quite tricky and he had never used it before.....

What were the chance health insurance would pay?

Antibody treatments are usually pretty expensive, and daratumumab has never been used to treat my type of neuropathy at all before. It's not even licensed to treat any autoimmune diseases (yet). So we never expected that insurance would  pay without trying to get out of it. 

So far I had been very lucky with my insurance, and they had paid for a lot of expensive off-label treatments, which they could have tried to get out of it too (very thankful for this!!), so we figured we just give it a try. 

The plan always was, that in case they deny our request, we will go and talk to Janssen, who produces the drug, and they might pay for it. Producing companies have an interest in getting the drug out there to treat different diseases (got all this info from my hematology friend who had discussed a lot with the representative of this company before....). 

First insurance refused our proposal straight away

Within a few days after sending in the first proposal, I received a letter from health insurance saying they don't want to pay for daratumumab, because there is no evidence showing that this particular drug would work for my particular disease. 

It´s true that there is no evidence at all that this drug will work in my case, but then again, when you have a rare disease that has hardly been studied, that´s kind of in the nature of things. So we kind of expected this answer, but it's just a very tiring and emotionally draining process trying to fight for a treatment that you would rather not have to get anyway. 

It is quite strange, because you hear about these kind of problems all the time if you work as a pharmacist at a university hospital, but you don't really realize the dimension and complexity of such situations until you see it from the patient side.....but anyway, enough of that for this week and I will talk more about this process later.

We submitted a request for re-evaluation

So Prof. Wexler and I (after being advised by my excellent hematology friend) have submitted a request for re-evaluation. We explained why we think that trying this drug would still be the best option and that we think the application needs to be re-evaluated by a physician. 

In our request for re-evaluation, we asked the insurance company to either pay for the drug themselves or to get in contact with the pharmaceutical company that produces it to see if they would sponsor a trial treatment. The companies name is Janssen. Apparently this is what they did, and they wrote us now that Janssen has agreed to cover 2 months of treatment (ca. 20-30k Swiss Francs/USD). The insurance company said they want a progress report, so i'm hoping they'll keep paying for it in the future if it works...(they did not explicitly say that but I'm assuming this is why they want a report). 

This actually happens a lot, especially when treating rare diseases. Health insurance is not obliged to pay for a treatment if there is no evidence out there to show it has a good chance of working. Rare diseases are obviously uncommon and it's not easy or very lucrative to run studies on them. Often times when insurance doesn't want to pay, the drug producer will sponsor a trial of the treatment if the request sounds reasonable.

Obviously the company who produces the drug does not do this out of generosity and kindness only. They have an interest to get the drug out into the marketplace and in it being established in the treatment of different diseases. I think it is an aspect we often forget when we complain about evil 'big pharma'. They are actually paying for the treatment of many patients with all sorts of different rare diseases because health insurance is unwilling to pay. Of course one can argue whether or not this is a good system - but right now I am greatful for this option. 

Thank you Janssen, for paying for my treatment trial. 

So while this is great news, getting a new treatment is also kind of scary. I don't know if it will work, if I will tolerate the infusion, or if I will have any side effects from it and and and. I will discuss all the open questions with my prof next week and will keep you all posted on that. 

One of the main points we have to discuss is when we will start the treatment and whether or not to wait until I've received the COVID vaccine. Getting this new drug will reduce my reaction to a future COVID vaccine (for a while), but then again I'm not high-risk enough to get the vaccine right now. At the end of the day, I guess you have to treat the current problems first. I will not wait until summer to try this new drug just to get the COVID vaccine (Switzerland is being a bit slow with their vaccinations atm😏). 

THERE ARE ALWAYS SOOO MANY QUESTIONS THAT COME WITH NEW TREATMENTS, but they are not today's problems. Today I am going to celebrate the little win and have an Apero in the mountains 😀 We are going to play cards via Zoom with some friends....Apero is what Swiss people really like to do: it is basically just a nice word for having a drink before dinner, and Swiss people are very good at finding reasons to have an APEROOO.....🥂

So on that note I wish you all a great weekend and am sending you a hug from the mountains where we are still hibernating. 

BTW: most the photos I post are taken by Steve (he is a much more talented photographer than I am)

Categories: treatment