Originally published by our sister publication Specialty Pharmacy Continuum
For more than 30 years, Taylor John experienced debilitating pain crises from sickle cell disease that frequently sent her to the hospital. Last year—following two injections of gene therapy in 30 minutes—she became pain-free.
Such life-changing experiences represent the future of pharmacy, said Kim Tedesco, RPh, the director of gene and cell operations at Walgreens, during a presentation at the NASP 2025 Annual Meeting & Expo, in Denver. She discussed several points for pharmacists to keep in mind as Ms. John shared her patient journey.
Cell therapy is the transfer of live cells—grown or modified outside the body—into a patient via infusion to help mitigate or cure a disease, Ms. Tedesco explained. The cells can be from a patient or donor. Gene therapy replaces, deactivates, or repairs the faulty DNA underlying a disease, and often uses a virus to deliver the newly corrected DNA into the cell nuclei.
About 2,000 cell and gene therapies are in clinical trials, she said, for “every disease state that you can think of,” from Alzheimer’s disease to type 1 diabetes. What began as rare therapies for rare diseases is morphing into therapies for high-volume disease states, Ms. Tedesco said: “Specialty pharmacy is really poised to be at the center of walking these journeys together with the patients.”
Because gene therapies are still new, 94% require 15-year, long-term care follow-up for the collection of data on delayed adverse events, she said. “Making sure that we have ways to connect with patients in a meaningful way will be really important with pharmacy,” she noted.
Social determinants of health play a significant role in accessing cell and gene therapies, which for now are only delivered in authorized treatment centers, Ms. Tedesco said. “There only may be a few treatment centers across the country that are treating a specific disease,” she said. In most cases, patients must travel for an extended period, making transportation, housing stability, financial and employment security, and caregiver support important considerations. “All of these are things … pharmacy [needs] to think about in a different meaningful way as we approach cell and gene therapy,” Ms. Tedesco said.
A 30-Year Quest
Ms. John, now 31, was diagnosed with sickle cell disease in the 1980s through a newborn screening. She was too young to recall her first crisis resulting in hand and foot pain around age 1, but her mother reported Ms. John held her hand and refused to stand. The crises continued at least four times per year. “I missed a lot of school due to sickle cell crises. During each hospitalization, I was in the hospital for five to seven days at least,” Ms. John said. “Each episode took a lot out of me.”
These events were unpredictable, and Ms. John’s younger sister would frequently have to bring her homework to the hospital ER while Ms. John was being evaluated and treated. Pharmacists need to recognize that these diseases affect not just the patient but family and caregivers as well, Ms. Tedesco said.
Although Ms. John was prescribed hydroxyurea to lessen or prevent crises, her disease worsened. By the age of 15, she began to experience vascular necrosis and pain in her hip joints, as the cartilage there began to deteriorate. “Just walking throughout high school to get to classes was very painful. At one point, I was in a wheelchair because it was just unbearable,” she said. By age 16, doctors told her she would need both hips replaced. By her late teens to early 20s, Ms. John started to see other friends with the disease die. It lit a fire in her to find a transformative therapy.
During the COVID-19 pandemic, she learned about gene editing through a television news program, visited the clinicaltrials.gov website, and started calling participating centers. She was accepted to the trial at TriStar Centennial Medical Center, in Nashville, Tennessee.
Even then, Ms. John still faced a long journey, starting with lung function tests, echocardiograms, and more to ensure she was strong enough to endure the procedure. Next came 10 sessions of cell collections where Ms. John was connected to a machine for six to eight hours per day. One round of collections was a bust, and the six-week wait between collections took an emotional toll on Ms. John.
After collection, her cells were sent to Switzerland for preparation, with a wait time of 23 weeks. Finally, on her 30th birthday, Ms. John was informed that her gene therapy was ready. She broke into a dance in the hospital hallway. But she still needed 16 rounds of chemotherapy to prepare her body to accept the therapy.
The day of her therapy, Ms. John said she felt like a kid on Christmas morning. “I went through 31 years of pain and suffering, and my entire life was changed in 30 minutes, with two 50-mL syringes of pink cells. It was mind-blowing.” After three months in the hospital, she was discharged to a new life.
Today, Ms. John feels well but returns to the center annually for checkups. She completed a course to become a medical assistant and is now able to travel freely.
Along the way, Ms. John had to adjust to her life-changing event and thinking of herself as a healthy person, with a mere half-hour mental health consult and few predecessors to offer her advice. This is another lesson to pharmacists to be proactive in helping identify any mental health concerns among patients undergoing cell and gene therapies, Ms. Tedesco said.
Throughout the process, pharmacists supported Ms. John by sending medications for pain management to her hotel so she could rest following long days of treatment.
Ms. John offered her advice to pharmacists.
“Treat these patients like they’re your family members,” she said. “Look at them as an actual person and not just a name on a bottle that you’re filling. … Each and every cell and gene [therapy] patient has been through so much to get to the point where they’re in front of you. Please try to make their experience a lot easier if you can because you don’t know the road they’ve traveled.”
The session garnered many positive comments from attendees, commented Rebekah Anguiano, PharmD, BCPS, BCACP, the director of education at the National Association of Specialty Pharmacy. Pharmacists can sometimes lose scope when caught up in the details of their daily work, she said: “[Ms. John’s] story realigns what we think about, what we do. It brings you back to the patients, their journey and their hope, and that’s what it should all be about. It’s really important to listen to stories like Taylor’s and be reminded of the importance of what pharmacists do on a day-to-day basis.”
Dr. Anguiano and Ms. John reported no relevant financial disclosures. Ms. Tedesco is an employee with individual and restricted stock in Walgreens. She is a former employee with individual stock in CVS Health and Johnson & Johnson.