There is an important kind of rhabdo clinical trial that is presently hindered by lack of bio-specimens. Specifically, the trials focus on creating individualized therapies for relapsed rhabdo patients. In order to consider designing such a trial, clinician-scientist ideally want to see that positive effects of specific drugs in patient-derived mouse xenograft models.

The number of bio-specimens from relapsed rhabdo patients is extremely small. If, and I can only hope in one way that you do not qualify, but in the other, if you do, please consider working with your oncology team - if any surgical procedure is being considered - to donating as much tissue extra (otherwise discarded) sample as possible so that these trials can begin.

The specimens for making patient-derived xenografts (PDX) need to be alive (fresh/not frozen and not in formaldehyde or wax). These samples can be sent to any of the labs that FoR has identified (many of whom have signed FoR MOU’s). Archived (frozen and wax-embedded tissue) is still valuable, but for other kinds of research studies. Dr. Keller's lab will accept live tumor tissue, but so does Dr. Houghton (perhaps the world’s authority on rhabdo PDX’s) and others can also (perhaps Dr. Rota might be able to make a PDX in her own lab… which is just as good an option.)

It all starts with a family expressing interest and letting their clinical team know that they wish that extra, leftover material sent to one of these labs. The researchers and clinicians can take on the details from there.

Thanks to all for your consideration. Should you have any specific questions, please feel free to contact me using the Contact Us form on the website or leave a comment below.

Alan Ehrlich