By CAC2 Individual Member Jennifer Flowers
Rhabdomyosarcoma became a common word in my family when I was three years old. By the time of diagnosis, the cancer had metastasized from the middle ear into the mastoid and eustachian tube. The University of Michigan’s C.S. Mott Children’s Hospital oncology team told my parents there was less than a three percent chance for me to live out the year. There was no definitive treatment protocol to defeat this rare form of cancer, and the survival rate was extremely low. Over the next two and a half years, I underwent surgeries, full head and neck radiation, and a four-drug chemotherapy cocktail. Just before my sixth birthday, I became one of the rare survivors and entered remission.
Routine check-ups resulted in clean bills of health until shortly after my fourteenth birthday when the oncology team discovered a tumor wrapped around my brainstem and cervical spine. Entering surgery, there were high risks of respiratory failure, total-body paralysis, and the possibility of another malignancy. Thankfully, outcomes were less severe – paralysis of only one arm (later reversed with physical therapy and stubborn determination) and pathology reports identifying the tumor as a benign schwannoma.
Late effects from treatment increased as I entered adulthood. In my mid-twenties, symptoms of extreme fatigue, loss of lean muscle mass, and rapid weight gain led doctors to discover radiation damage to my pituitary gland prohibiting adequate production of human growth hormone (HGH). I rely on daily injections of somatropin to counteract the deficiency and will depend on it for the rest of my life.
In the fall of 2023, the national drug shortage impacted my ability to obtain monthly refills of this essential-to-life drug. My specialty pharmacy began receiving reduced quantities of Nutropin AQ from its maker, Genentech. Every month, my case manager was unsure whether she would be able to secure one of the rationed injection pens for my refill. We scheduled the refills and then held our breath until the pharmacy’s shipping notification came through, indicating that one had been sent out for delivery. These notifications brought great sighs of relief and guaranteed another month of medication.
In early 2024, the pharmacy only received 5mg injection pens of Nutropin AQ, and no 10mg pens for my prescribed dosage. Due to insurance coverage restrictions, I was only allowed one 5mg pen at a time with no guarantee that the pharmacy would still have medication available two weeks later when I would need more. My endocrinologist had me start rationing the medication. Rather than taking the daily amount required to maintain normal levels of HGH in my body, I took a reduced dosage every other day. This was extremely stressful. Without sufficient daily doses, my HGH count begins to drop within two weeks, and symptoms of HGH deficiency resurface within a month or so. Thankfully, Genentech was able to reinstate production of the 10mg pens within that time, and I was able to resume adequate daily dosages before deficiency symptoms surfaced.
From the beginning of the shortage, my specialty pharmacy and I had been proactively planning ahead by searching for answers as to whether or not the Nutropin AQ shortage meant an imminent discontinuation of all HGH drugs in the United States but found no solid answers. Then in the spring of 2024, Genentech sent letters to patients stating that on December 31, 2024 they would “discontinue all…. formulations [of Nutropin AQ] in the United States.” I was devastated by this and scared about what it could mean for the future of my health.
My endocrinologist, upon hearing the news, said it was time for me to start advocating for myself at a higher level. I did not know how to do this, but one thing was for certain – I was not going to let the chemotherapy and radiation that had saved my life now take my life. I had come this far. I was not going to stop fighting now.
The formation of cancer advocacy groups mostly occured after I was released from my oncology team’s care. I had never been involved with one. As a survivor much older than the children and young adults for which the groups appeared to be focusing their work, I was not sure that anyone could or would help me. However, if one does not ask, the answer is always “no.” After researching multiple groups, I sent emails to the first six on my list asking if they could provide me with any guidance. Alex’s Lemonade Stand Foundation (ALSF) replied to my email. Although ALSF does not provide such services, they had contacted CAC2 on my behalf for possible recommendations.
It amazed me that an agency had just stepped in to help me in this way! Within 24 hours, ALSF connected me with Vickie at CAC2. Vickie immediately began an investigation of the shortage via her contacts at the White House and in the Senate. After hearing my story, the White House connected us to Laura Bray, Chief Change Maker at Angels for Change. This organization had been helping HGH patients with the somatropin shortage for the past year. Within 72 hours, Laura and I met via Zoom and developed a plan of action. Since my doctor had approved a switch from Nutropin AQ to Pfizer’s Genotropin, Laura set up a meeting with Pfizer’s entire Genotropin team to inquire about their 2025 production plans. Aware of the shortage, Pfizer had already made a commitment in their upcoming five-year plan to continue providing for HGH patients.
Changing specialty drugs is a laborious and stressful process of prior authorization paperwork and waiting for insurance approvals. During the changeover process from Nutropin AQ to Genotropin, there were roadblocks that my pharmacy case manager, endocrinologist, and I could not get past. CAC2 and Angels for Change immediately stepped in and notified their contacts in Congress and the White House. Shortly thereafter, approvals were granted and my first prescription for Genotropin was filled.
Throughout my childhood, my mom constantly advocated for me. She was a momma bear who protected me while never letting me give up the fight. As I grew older, she mentored me as I took on the role of advocating for myself. Survivorship is uncharted territory and times come when the expertise of others is needed to help you advocate for your needs. There is a saying, “You never stop needing your mom.” One momma bear fought for me throughout my childhood. As an adult, CAC2 and Angels for Change have become like two extra momma bears who are there to fight with me and for me when I cannot do it on my own. It is an amazing feeling of relief and gratitude to be entering 2025 with such phenomenal support and knowing that I am not alone!
Pfizer has given HGH patients the hope of five more years of somatropin-based drug production. This is an encouraging start, but it is not the solution. Survivors with HGH deficiencies need the guarantee that somatropin-based drug production will continue throughout our lifetimes. As a member of CAC2, I hope to give back even an ounce of what has been given to me as we look for solutions that give pediatric cancer survivors many more birthdays with quality of life!