Laila’s Birthday Fundraiser for a Cure!

This upcoming week is Laila’s thirteenth Birthday! Last week we hit the two year mark in her fight against Rhabdomyosarcoma. To say my heart and mind have been battling high stakes and emotions as of late is putting it lightly. I posed the  question recently to her doctor “what next?”. Laila is on four drugs currently. The Temsirolimus that we do weekly maxed out at 12 months. The Doxil that we do monthly maxes out at 18 months. The two immunotherapy drugs which are no-weekly and every three months max out at two years of use. So “What next?”. We are six months into this investigational treatment plan. We also maxed out her lifetime dose in an unprecedented amount of radiation to kick start this plan of ours. Meaning we must attribute some of what is currently working to that fact. We cannot utilize that again. Well what is next is we continue to go and have hope that we have cured her of disease. We cannot treat Laila further than this unless she has another active relapse. We need BETTER! Personally and overall. Did you know that when we came up with this plan it was the first time in approximately four years that a briefing was held to inform staff about the news drug we would be utilizing and that this has opened the door to others who are now using the same drugs in their protocols?! Four years!!!! They have been using the same drugs and getting the same statistical results, per oncology across the board and it’s advanced(no fault of our wonderful team). I have to be honest I’ve found myself at my lowest at times throughout this second battle. If we had nowhere but up to go through frontline, relapse is a scary depth without words to describe our daily worries. However! That feeling….of pushing forward little by little and opening doors for better, for a cure for rare pediatric cancers is the fire that pulls me from those depths and fills my world with HOPE! The gasoline that moves us to every next day. Every appointment! Every scan! Every hard moment she has endured! This treatment is promising and it needs funding. My first HOPE is that my Laila will be cured with no more incidents moving forward. Still, we have no way of knowing. In the event of a relapse I would like this treatment to have been funded, proven effective and to be available to Laila. In the event she is cured by our current efforts I want this treatment to have been funded, proven and available to the next child who sits where Laila sits. So they have less to endure than she has, as well more and better outcomes. I will attach the email following this that describes the treatment projection down at CHOP. Keep in mind the drug irinotecan was a part of Laila’s frontline treatment as well as part of the beginning of her second treatment plan. It’s one of the more effective treatments we have available and this treatment plan is looking to improve cancer treatment for all. The Doctor who wrote this email explaining further their trial we are raising money for coincidentally hired Laila’s primary oncologist and worked with him for almost thirty years at CHOP! Along this journey EVERY time I have found myself lost or looking for our next turn it seems some “coincidence” or obvious sign lights the way. Dr. Phil (Laila’s oncologist) always airs on the side of caution. “We are treating her NOW” and what we have available is what he insists we focus on. He also held this doctors opinion with such high regards and to hear him state this is one of the most promising projected advances he’s seen in his time intrigued Dr. Phil or piqued his interest. And so. This is where it becomes our duty as the parents of these rare cancer fighters, to use our position and our voice to rally the masses. To help us find and fund better treatment options until the day there is a cure! Because no child! No child deserves to live this hell we call cancer treatment. 

Investigators at CHOP (Drs. Chorny, Brodeur and Alferiev) have developed a nanomedicine that promises to deliver much more drug to the tumor, and much less to the patient. Thus, it should be both more effective and less toxic than the same dose of conventional therapy. Our agent, called PEEL-224, is being developed for clinical trial by PEEL Therapeutics. It is related to the conventional drug, Irinotecan, so theoretically it could replace this agent in clinical trials, once safety and efficacy are demonstrated. So far, in our mouse models of neuroblastoma, rhabdomyosarcoma (RMS), and Ewing sarcoma (EWS), we are able to cure 80-100% of the mice with just four weekly doses of the drug, whereas conventional delivery of irinotecan at a much higher dose has little if any effect and cures none.

We are working with PEEL Therapeutics, and Dr. Joshua Schiffman, the President and CEO, to secure FDA approval of PEEL-224 as an Investigational New Drug (IND), and we are also applying for orphan drug status. We anticipate to submit our full and final application to the FDA by the end of the year, with phase 1 clinical trials to start in early 2022. As is usually the case, the initial trial will be done in adults, and the indications will be colon, gastric or pancreatic cancer. However, there will be another stratum for recurrent/refractory sarcomas, particularly RMS and EWS, and we expect that patients as young as 12 years old will be eligible for this trial.

The government works in slow and mysterious ways, so we cannot guarantee that things will happen on this timeline, but so far we have met or exceeded every deadline for submission or completion of preclinical studies that were required for the FDA submission. So, stay tuned, keep your fingers crossed, and perhaps write a letter to the FDA to support the approval of this agent.

If you’ve made it this far thank you!

If you can donate thank you!

If you think about us than you!

We love and appreciate your support deeply.  

Raise money for a CURE for Laila’s Birthday 💛