Dear Cure JM Families and Friends,
A few days ago, I shared with our Cure JM families an update that I was “Counting the Days.” I was counting the days because Cure JM can raise $1 million in our Annual Holiday Challenge.
I’m not counting any more, as today is December 31, the last day of the Challenge. The year-end response has been generous and heart-warming, and we are almost there. Three days ago, we had $150,000 to raise to reach the $1 million goal. Today, we need just over $50,000.
Reaching this goal is more important than ever, as moving forward with several game-changing research projects hangs in the balance.
Just last week I met with the research team that is planning an important clinical trial for a drug that has already shown great promise as a new JM therapy. This drug is proven to block the over expression of interferon which leads to muscle inflammation in JM. The team’s clinical trial plan for children and adolescents with JM is in place and has been approved by the FDA.
We are ready to go—almost.
I say “almost” because the pharmaceutical company that was to supply the drug as a clinical trial partner has now elected not to participate.
I’m not totally surprised by this turn of events. Juvenile myositis is a rare disease, and in the dollars and cents world of big pharma, a market like JM is all too often seen as too small to bother with.
We’re not giving up, even though this big pharma decision leaves the research team with a $270,000 budget gap that someone will need to fill.
Also last week, in an emergency meeting, the Cure JM Research Committee recommended an initial commitment of $75,000 a year: funding that will allow the trial to move forward without pharma’s participation. Without Cure JM as the backstop to fill the funding gap, this trial would die on the vine.
And so, I am reminded, once again, of just how important Cure JM’s funding and leadership has been to researchers in the U.S. and around the world. Each year, we understand more about what causes JM and therefore how to better treat it. We have grown the number of drugs that can be used as alternatives to steroids, methotrexate, and IVIG and that are often used as maintenance therapies in managing and controlling the autoimmune storm.
Because of these and other advances, it’s fair to say that Cure JM has helped return childhood to our children who deserve it.
You have made all this progress possible through your support of the Holiday Challenge. Reaching that $1 million December 31 goal will allow us to do everything possible to close the $270,000 clinical trial budget gap when the board of directors meets in January to assess our available emergency resources.
So, if you have yet to make your Holiday Challenge commitment, please do so now so we can move forward with this clinical trial and assure a brighter future for our JM children. And if you’ve already given, THANK YOU! You’ve made everything we do possible.
Tomorrow brings a New Year of Hope and Promise for Cure JM families. In 2023, Cure JM researchers added one new drug therapy to the growing number of options available to JM children in their journey toward remission. With your help, we can and will advance better treatments and more options in 2024 and future years.
May your family—and Cure JM—have a great year in 2024!
With appreciation,
