Gene therapy for autoimmune disease?

Patients Multiple sclerosis

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Posted on

Hi Dr. Seignez,

Happy Thanksgiving!

From what I've understood over the years, an autoimmune disease like RA is reaction from the body "turning against" itself.

So would it be pointless to consider gene therapy? I've heard so much about it in recent years so I was wondering if it can be applied to conditions like RA. Is there any research going on or planned in it?

Also, can you tell us what the differences (besides the exorbitant price) are between biologics and chemotherapy drugs like methotrexate in treating RA? Is one better than the other? 


Beginning of the discussion - 11/29/21

Gene therapy for autoimmune disease?

Posted on

Hi @karm!nR‍,

In autoimmune diseases, the immune system, which is usually in charge of eliminating germs and preventing tumors, attacks certain parts of the body. For example, in type 1 diabetes, the immune system destroys part of the pancreas, which stops the production of insulin (a hormone made by this part of the pancreas). In multiple sclerosis, the immune system damages the nerves, which causes sensitivity and movement issues.

Gene therapy is a very promising therapy for many conditions, including autoimmune diseases. The very first gene therapies have arrived on the market (mostly for cancer). The field of research is very large, and some teams are working on gene therapy for autoimmune diseases, so there is good hope that specific gene therapies for autoimmune diseases will emerge. But it would be premature to try predict a specific time frame.

To answer your second question very broadly, biologics are newer, more complicated to take (via injection), more expensive, and generally less aggressive than chemotherapy drugs. In most cases, the practice is to start with chemotherapy, an older drug whose effectiveness and side effects are well understood, and, if the disease is not stabilized, to combine it with a biologic, or, if serious side effects appear, to replace it with a biologic. There is no specific time frame for considering biotherapy, but it is usually measured in months rather than years (the official recommendation for rheumatoid arthritis is at least 3 months of maximum-dose methotrexate). Biotherapy is not "the" answer for everyone, but it often provides a significant addition to treatment.

If you suffer from an autoimmune disease and your disease is not stabilized by your background treatment, whatever it may be, I invite you to consult your doctor.

Have a nice day!

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