Three Significant Rhabdomyosarcoma Initiatives

         Jump-Started at Denver Workshop!

   Elevate Childhood Cancer Research and Advocacy, Inc. partnered with four leading rhabdomyosarcoma-focused foundations to bring dozens of the leading rhabdomyosarcoma physician-scientists and clinicians together for two days of non-stop, roll up the sleeves work to bring about improved diagnosis, treatment and survivorship for those impacted by the disease!

   Elevate CEO, Robyn Spoon, has been collaborating with Summer’s Way Foundation, Focus on Rhabdo, Maddie’s Promise, SebastianStrong, and Friends of TJ for over a year in advance of this biennial workshop established in 2018 by participating foundation members. Experts came together from all over the world, including Belgium, the UK, Germany and all across the US from Dana-Farber, Harvard, Johns Hopkins, Duke, NIH, Texas Children’s, Baylor, University of Washington, St Jude, and more.

So, what happened? 

These two days felt like we had all jumped into a therapy development sandbox for rhabdomyosarcoma, with physician-scientists, clinicians and basic biology scientists sharing what they are working on, and gaining insight from others on their early findings! It can take years for their work to become publicly available. These scientists and clinicians work in institutions and hospitals all over the world, and they thanked our foundations for providing them with a chance to set aside their other tasks for a few days and focus exclusively on gaining up to date information on rhabdomyosarcoma from their expert peers! 

The workshop also gave the opportunity to early scholars in the field to network with leading experts as these early scholars and post-docs are actively seeking research and clinical posts right now! It’s important to provide these young scientists a chance to stay in the field!

    We need them.

   Several of the early scholars had received grants from rhabdo-coalition foundation partners. Summer’s Way and Friends of TJ have sponsored some of these early scholar grants. In addition to sessions led by experts in the field, Workshop participants, including foundation participants, got to hear updates from the work being done by early scholars like Dr. Patience Odeniyide, who is working on RAS-directed therapies for rhabdomyosarcoma, and Dr. Yun Claudia Wei, who is working on better understanding a particularly aggressive form of rhabdomyosarcoma, ssRMS with MYOD1. 

   Elevate had the opportunity to gather critical information from participants through a pre-workshop survey to gain insights into short and long-term best hope for developing new therapies as well as the challenges these experts face while trying to treat patients and develop new therapies. The answers to this survey led our foundations to lead a Saturday morning session on:

   Breaking down barriers to data access for those in the front lines of treating our kids AND for those trying to better understand the disease in their research work.

This “all hands on deck” discussion led to the creation of the…

   Rhabdo Coalition Data Access Task Force, which will begin work in January of 2024! The Task Force will be identifying the most critical questions that need to be answered so that we can collaboratively find solutions to get this prioritized information in the hands of those who need it most. This will…

>Help decision-makers: better stratify risk categories for those diagnosed with rhabdomyosarcoma

>Help provide better data to clinicians in treating hospitals so they can go beyond their set of data in their own hospital to gain up to date info on best treatment options for patients,

AND

>Help researchers access critical information they need to assess matches for possible new therapies! 

   Elevate will also be participating in the second initiative coming out of the workshop: a Therapy Prioritization Task Force!

   This group will be determining the most promising options for new therapies, as a group, and then determining the best ways to get them brought forward through clinical trials. While this group is working on a start date, the Rhabdo Coalition members are already meeting collectively with industry partners in biotech and pharma to better understand how we can help overcome what has been dubbed “the valley of death”, which is the land between academic research and clinical trials. Gaining an improved understanding will help us tackle the problem head-on. The third goal coming out of the workshop is to help establish better access to needed tissue samples, cell lines and PDX (patient derived xenograft) models so that researchers can adequately test new compounds against various types of rhabdomyosarcoma. This task will be left to foundations, like Elevate, and other members of the coalition.

Our newly forming Elevate Rhabdo Workgroup will be tackling this issue head-on in the coming year.

 All participants left the workshop armed with updated information, improved collaborations and a list of next steps for 2024!