Inside a Drug Trial for a Rare Childhood Disease: Drs. Sarah Tehseen & Katie Felton

Most childhoods don't involve sitting at the hospital for an infusion of medication, transfusions on weekends, or worrying that classmates will comment on the colour of your skin. For one Saskatoon teen with an ultra-rare blood disease, that's everyday life. She was diagnosed with a form of anemia so uncommon only a handful of cases have been identified globally.  In this episode, Sarah Tehseen (MD) & Katie Felton (MD) share how they're working to change her "normal" by opening a phase 3 pharmaceutical trial and fighting for a better quality of life.   We hear how Tehseen and Felton each got into medicine, why they love working with kids, and what it's like to be there for families on "one of the worst days of their life."  "It's getting them through the next day, week, month and years ahead," said Felton. "So even though, yeah, I deal with blood disorders and cancer, which are really can be difficult conversations with families, we still have fun."   They pull back the curtain on the effort it took to bring a this drug trial for an ultra-rare form of anemia to Jim Pattison Children's Hospital. From having to respond to 40 or 50 e-mails a day, to forfeiting vacation time as their patient goes through blood draws and clinic visits, it's a heavy lift. "Definitely, it requires some changing and plans for us at times, to be able to accommodate and facilitate that," said Tehseen.  "Having two physicians doing it together, rather than being doing it alone, is has, has been super helpful."   They discuss the hidden financial realities of rare drugs, and the importance of blood and stem cell donation. Both physicians say they find true joy in detective work, and in finding the right treatments for their patients. And even simple things like learning a child's favourite video game or doing bunny-hop races down the hall can help kids coping with rare diseases feel a little less alone. Tehseen says it's worth learning more. "If she's your classmate, if she's your student, know what it is, how it's affecting her. Because the more you know, the better you're able to show up in the life of that person," she said.     OneMatch Bone Marrow Registry - https://www.blood.ca/en/hospital-donors-and-volunteers/become-donor/one-match Canadian Blood Services - https://www.blood.ca/en