September 1, 2021
A Rare Disease Patient Sheds Light on Journey for Treatment
Written by: Amy Ripston
4G Clinical’s Senior Director of Marketing, Amy Ripston, had the opportunity to interview Renee Man, a brain cancer patient and mom to three beautiful children, on her journey from diagnosis to clinical trial participant.
The journey of a rare disease patient is seldom a simple one. Delays in an accurate diagnosis coupled with the lack of treatment options for advanced Meningioma turned her world upside down.
When Renee first discovered her Meningioma she was told it was benign. Something to watch, but did not require immediate treatment. It wasn’t until her craniotomy that she was informed it was in fact malignant and she rapidly started radiation. Results were positive, she was given hope of 10-15 years of good living. A chance to see her children grow and graduate high school. Years of memories ahead of her.
For a year and a half, it was just that. No tumor growth. Birthday parties, everyday moments, laughter.
In August 2020, her scans revealed that not only did the tumor grow, but there was a new one. It was aggressive. Doctors didn’t know how much time they could buy her. In a moment, she went from watching her children graduate high school to being lucky to see them graduate elementary.
That’s when her physicians fought to enroll her in a clinical trial using a failed breast cancer drug that had showed promise in brain cancer. Having started in 2017 with only 27 patients to date, trial results appeared promising but there had not yet been an interim analysis of the study.
With another promise of hope, comes more uncertainty. Since starting the trial the manufacturer has stopped making the drug as several of their other phase II trials in more common cancers have failed to show improved survival. Thus, Renee’s trial is set to end in Q2 2021 as they will no longer have new drug supply.
This was painful to hear. Even if the drug works, it only buys her six months unless the manufacturer decides to keep making the drug. Despite this news, you could still hear the positivity and strength in her voice few would be able to muster. To her, 6 months is 6 months – even if it comes with its side effects: seizures, fatigue, headaches, nausea.
While Renee’s disease is rare, this situation is not. Many patients participating in critical exploratory treatments face a similar truth when clinical trials end midstream. Rare disease advocacy has come a long way, although there is still so much to be done.
It was an absolute honor to speak with Renee. Our thoughts are with her as she continues her journey and we applaud her willingness to raise awareness for other rare disease patients.
For more resources for rare disease patients and advocacy, check out the links below:
Tag(s): Industry Voices