The Very Real Potential of CAR-T Therapy in Myositis

The Potential of CAR-T Therapy in Myositis.

We expected to hear promising news about the potential for CAR-T therapy to treat myositis at the Global Conference on Myositis (GCOM).

What we heard was more than promising—it was astounding.

What if I told you that there was a therapy that would “reset” the immune system—virtually eliminating the autoimmune response in myositis that causes inflammation, pain, muscle weakness, and other JM conditions of which we are all too familiar.

That “reset” exists today and is approved for use of certain blood cancers. A next frontier is to target rheumatic autoimmune diseases such as myositis and lupus, and early clinical trial results are very, very encouraging.

The most notable results come out of Germany, where 15 patients including three myositis patients as young as 20, completed the trial. All of the patients saw remarkable and lasting improvement—some of up to three years of being “disease free.”

Results were reported by Dr. Georg Schett, head of the Department of Rheumatology and Immunology at Friedrich-Alexander University in Erlanger-Nurnberg, who led the trial and was the keynote speaker at GCOM. Schett presented a visionary scenario where in rheumatic diseases such as lupus and myositis, Car-T therapy will emerge as a “living drug” leading to the absence of disease eliminating the necessity for ongoing treatment. Current JM treatments, as we know all too well, suppress the immune system and usually lead to clinical improvement, but do not eliminate the disease.

We also heard from Dr. Carl June, a pioneer in Car-T therapies used in cancer, who noted that autoimmune diseases present the next frontier of research with an “explosion of research and investment” in just the last year. “We can learn much from CAR-T’s success in oncology,” June noted, likening the process to a bone marrow transplant. June is the Director of the Center of Cellular Immunotherapies at the University of Pennsylvania Perelman School of Medicine.

June also serves as a medical advisor to Cabaletta Bio, which has a CAR-T therapy platform specifically designed for autoimmune diseases such as lupus and myositis. Dr. David Chang, Cabaletta’s Chief Medical Officer, served as the moderator of the CAR-T “Achieving Remission” session. Cure JM families may remember Dr. Chang from the 2023 Family Conference where he shared Cabaletta’s progress with Cure JM’s Medical Advisory Board.

I will share with you more information on upcoming CAR-T clinical trials in myositis along with a guide on the process of “How CAR-T works.” Of the 15 patients in Dr. Schett’s trial side effects were minimal, short-term, and when they did occur, proved to be manageable.

The Potential of JAKs in Fighting JM

In our June 2024 “Ask the Doc” Town Hall, Dr. Julie Paik joins in a Q&A session to shed light on what JAK inhibitors are, how they work in JM, when parents might consider discussing JAKs as a treatment option, and the pros and cons of their use.

Helping Parents & Kids When They Express High Emotional Distress

This expert-led presentation is for parents about how to help their kids and themselves when their kids express high levels of emotional distress. Our presenters will talk about ways parents can calm themselves, share tips for talking to kids with age-appropriate information, and know when to find help from a professional. There will be a Q&A session immediately following the presentation.

Cure JM May 2024 – Virtual Symposium

The Cure JM Clinical Care Network hosted our second quarterly virtual symposium of 2024, “Juvenile Dermatomyositis – Advances in Clinical Care, Basic Research, and Translational Studies.” World-leading researchers and clinicians presented sessions on various topics, with the primary goal of enhancing the overall patient and provider experience through collaboration and best practice sharing.

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