feel like a guinea-pig?

Ongoing trials for DM/PM patients

I have only participated in one clinical trial in my life. It was for a hypertension drug back in the early 1990’s. I have not participated in one for dermatomyositis. However, I have used medications when they were still considered novel treatments, such as Rituxan in 2007 and Acthar this year. Those times I have felt like a guinea pig.




The Annual TMA Patient Conference in Reno, which I attended, has just concluded. Dr. Lisa Christopher-Stine from the John Hopkins Myositis Center, click here,  gave a talk on Promising research PM, DM. Click here to see that presentation, I thought I would mention a few of the ongoing pharmacological trials.


Safety and Efficacy of BAF312 in Dermatomyositis

This is where I dangerously pretend 10 minutes of google searches is a perfect substitute to attending med school for 7 years and having a medical practice! From what I understand BAF312, also called Siponimod, acts on certain types of white blood cells (lymphocytes) which are involved in autoimmune attacks seen in MS and other disorders such as dermatomyositis and polymyositis. It binds to special locations (or receptors) on the surface of the lymphocytes, called sphingosine-1-phosphate receptors (S1P-R). This causes a larger proportion of lymphocytes to be retained in the lymph glands, and thus not reaching the target of such attacks, which is muscles in myositis patients.

The trial is currently recruiting. Click here and here for more information.



Safety and Efficacy of BAF312 in Polymyositis

Please read the previous section on how the drug works.

The trial is currently recruiting. Click here and here for more information.



Tocilizumab in the Treatment of Refractory Polymyositis and Dermatomyositis (TIM)

Again I will caution you to my lack of understanding of anything medical. The following is an excerpt I gleaned from how the drug works in rheumatoid arthritis.

“Tocilizumab works by blocking a cytokine known as interleukin 6, or IL-6, which is believed to be one of the factors that cause inflammation in rheumatoid arthritis. Tocilizumab is an antibody that blocks the spot where IL-6 attaches to the surface of cells. When IL-6 is unable to attach to these cells, it is unable to activate them or turn them on. As a result, the cells are unable to drive inflammation in rheumatoid arthritis.”

Dr. Chester Oddis from the Division of Rheumatology and Clinical Immunology at the University of Pittsburgh, click here, is one of the principle investigators. He referenced the trial at the annual conference.

Click here for more information.


Acthar in Treatment of Refractory Dermatomyositis and Polymyositis

If you have been reading our blog, you will know I did not have success with this drug. However I will add a couple of caveats. I met four other patients at the recent TMA conference who took Acthar. In all cases they had a positive outcome with the drug in treating their myositis. In one case when the standard dosage of bi-weekly injections did not work, which I did, the patient switched to a more frequent, lower dosage treatment which helped their condition.

I blogged a piece about the history of Acthar and how it works. Click here  for that article. The study is led by another TMA MAB member from the University of Pittsburgh, Dr. Rohit Aggarwal.

Click here for more information.



The manufacturer of this drug, Idera Pharmaceuticals, had a booth at the recent TMA Annual Conference. They were taking names for an upcoming trial for use of the drug in dermatomyositis and polymyositis patients. I was unable to find any info on the trial and it is not listed on the clinical trials web-site. Some details on how the drug works can be found on the Idera web-site by clicking here.  Nicole spoke to the representative quite a bit, and we have information cards we’ll hand out at our Picnic in the Park and November 2nd KIT meeting for those interested in signing up for the Idera trial.


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