Executive Director Jim Minow

Executive Director Jim Minow during a recent hike in support of Walk Strong Across America.

Cure JM families and friends,

I have received some great news. 
The most recent clinical trial of the new drug, vamorolone, has provided additional positive safety and efficacy data required by the FDA for approval in the U.S.

In short: It works. It is safe. And most importantly, a New Drug Application for approval in the U.S. will be submitted to the FDA in the first quarter of 2022. That will open the door for a trial in JDM patients.

If you know me at all you know that I believe the development and availability of new drugs for the treatment of JM is one of our organization’s highest priorities. The path to success is not an easy one. A new drug can take 20 years to develop, and several more to gain approval by the FDA. And most clinical trials fail.

But we won’t.

Vamorlone is a new type of steroid. This current clinical trial confirms that it is the equal of prednisone in terms of effectiveness, but most importantly does not have the side effects of growth stunting and weight gain, among others. While we have much more to learn about vamorolone and JM, we have been partnering with its developer, ReveraGen BioPharma, over the past four years to launch a clinical trial specifically for JM kids. Trial planning is taking place at Cure JM’s Center of Excellence at Duke University.

The FDA’s first approval of vamorolone (should everything proceed as planned) will be in Duchenne Muscular Dystrophy, another inflammatory disorder resulting in muscle weakness and atrophy. While the clinical trial in JM will seek subsequent FDA approval for juvenile myositis, the JM medical community may also look to smaller safety trials to speed access to patients.

We are also looking forward to the results from clinical trials on two non-steroidal drugs that show promise in treating JM—abatacept and baricitinib. I’ll share more information on these two trials in my next update. Today, I’d like to close out with a message of great importance:  Walk Strong Across America!

Cure JM needs your leadership in Walk Strong more than ever. We need 50 more Walk Strong teams if we are to reach our July 4 Walk Strong Across America Day goal. So, if you have not yet created a team, you can do so now here. Shannon or Lauren will be in touch and can help you set up your page. It’s fast and easy.  

Please do. As we wage war on JM for all our JM kids, I’ll paraphrase Uncle Sam’s famous recruitment poster here:  I Need You! And so do our JM warriors. Thank you for taking action today.

It’s important to remember that raising money is not our mission. Our mission is to find better and improved treatments, a cure, and to improve the lives of JM kids everywhere by funding and supporting high-quality care and medical expertise in every corner of the country.  
Fundraising is the necessary means to meet that end.

It’s a wonderful wish to think that some other entity than Cure JM will fund the research or the vamorolone clinical trial, for example, that must be accomplished to make these new drugs a reality. Unfortunately, as my mother (and maybe yours, too) would admonish me as a child, Wishing Doesn’t Make It So. If we want to meet the mission, it’s up to us.

So, thank you, from the bottom of my heart, for all you do to make Cure JM’s work possible.

July 4—Walk Strong Across America Day—is a very special day for Cure JM when our families create a common bond from coast to coast as we come together to assure a brighter, healthier future for JM kids everywhere. Please don’t miss it!


With appreciation for all you do,

Jim Minow
Cure JM Executive Director