This article includes information about three people dying in clinical trial for CAR-T. We understand this may be difficult for some people to read about, and encourage you to reach out for support if you need it. You can find links to places to find support and information at the very end if you would like to skip the article.
Two pharmaceutical companies (Novartis and BMS) have paused their CAR-T trials for autoimmune conditions, including lupus. This is because three people sadly died in the Novartis trial, and they need to review all of the information to decide if it is safe to continue. No one died in the BMS trial, but they have paused their trial because some people had similar side effects. There are also other trials of different types of CAR-T which have not reported similar side effects or stopping their trials. We will update this article when we have more information or if anything changes.
Our thoughts are with the families and loved ones of the people who died.
What happened?
There are lots of trials to see if CAR-T cell therapy (often just called “CAR-T”) can help people with autoimmune conditions, including lupus. Different pharmaceutical companies, who make CAR-T, are trying different types. You can find out more information about CAR-T, lupus, and trials in our video series.
Three people in one of the trials died from a rare side effect. That trial was run by a company called Novartis. They have temporarily paused their trial while they investigate what happened so that they can see if it is safe to continue. Another company, called BMS, also decided to pause their trial. No one died in the BMS trial, but some people had similar side effects so they want to make sure it is safe.
So far, no other companies have reported pausing their trials or similar side effects, but we will update this article if they do.
What happened to the people who died?
The three people all died due to a rare side effect where the immune system becomes severely over-active and attacks the body, called “immune effector cell-associated hemophagocytic syndrome”. This side effect was already known to be a rare complication of CAR-T, but we don’t yet why it happened in this trial.
The trials have been paused so that the teams can investigate why this happened in more detail. This will help them to decide if it is safe for their trials to start again, if they need to make changes, or if they need to stop their trial completely.
Did this happen in the UK?
The Novartis trial has sites in different countries. We do not know where the people that died were based. ClinicalTrials.gov, a website which lists clinical trials, does not include the UK as one of the sites for the Novartis trial for lupus, but does include the UK in their trial for myositis, which is an autoimmune condition which impacts the muscles.
Are all CAR-T trials for lupus stopping?
The Novartis and BMS trials are stopping temporarily. This is so that they can review what happened and make sure they understand why. They will then decide if it is safe to continue, but this depends on what they find out. It might be that it is safe to continue but the dose or way people are looked after needs to change, or it might be that it is safe to continue in the same way. They might decide it is not safe to continue and stop their trials permanently.
There are also other companies running trials using different types of CAR-T, including in the UK. So far, no other companies are reporting similar side effects or needing to pause their trials. We will update this article with any new information.
I’m taking part in a CAR-T trial, what should I do?
You don’t need to do anything. If a research team has any worries or they decide to pause your trial, they will contact you directly. You will not have to find out in the news. Even if a trial is paused or stopped completely, they would still make sure you have all the regular check-ups you need to keep you safe and well.
If you are taking part in a trial and you have any worries, you can contact your research team. On the information sheet they gave you, there is usually a contact number if you need to speak to someone. You might also have a named research nurse or research assistant you can speak to.
It is completely understandable to be worried or have questions. It may be helpful to know that there are lots of rules about safety in trials. The two trials being paused means that the safety of people involved is being put first. You can find out about some of the organisations that oversee clinical trials in the UK on the NHS website.
As well as your research team, you can also reach out to support services to talk things through more generally. This might be your local NHS services, or you can contact our Support and Information Line by phone 01708 731251 or email: [email protected]
Does this mean CAR-T is unsafe in lupus?
We don’t know yet. Clinical trials are there to find out if a treatment is safe, if it is effective, and who it is safe and effective for. Some early trials have shown CAR-T could be safe and effective for people with lupus, and we wrote about some recent positive results on our website. But this other trial has shown a rare but very serious side effect. More research and more understanding of what kind of CAR-T, for what person, when, and at what dose is needed, as well as more understanding of what caused the side effect.
What does this mean for lupus and CAR-T in the future?
We don’t know yet. At the moment, some CAR-T trials are continuing to research and there will be a review about what happened to the people who died. When there is information from the safety review, we will have a better understanding of what it means for the future.
CAR-T is still an exciting possible treatment which might help some people with lupus, but these deaths highlight how important it is that there is more understanding of what kind of CAR-T may be safe and effective, who it may be safe and effective for, and when. Research and trials take a long time, and that can be frustrating when you need a better treatment now, but understanding who a treatment is safe or not safe for, and keeping people as safe as possible on a trial, are important reasons for it taking that time. It is a sad and difficult announcement, but there is still a lot of hope for the future in lupus treatment.
Our thoughts are with the families and loved ones of the people who died.
More information and support:
Information on CAR-T and clinical trials:
- Our video series on CAR-T, lupus, and clinical trials
- NHS information on clinical trials
- Information on how research is checked and monitored from Be Part of Research
Talk to other people with lupus: