As a principal scientist working on sickle cell disease in the Rare Disease Research Unit (RDRU) at Pfizer, Kelly Knee is a jack of all trades. She spends part of each day conducting experiments to test new ways to treat sickle cell disease, which is the most common inherited blood disorder. Knee also meets with researchers and patients to discuss Pfizer’s projects and priorities as well as innovations in treatment that have the potential to transform patients’ lives.
Patient-focused research is what drives Knee, and that extends well beyond the lab. At conferences and events organized by Pfizer’s RDRU, Kelly often meets with patients, interacting with them and their families as frequently as possible to ensure they are all part of the treatment conversation. Knee considers her interactions with patients suffering from sickle cell disease as a huge inspiration in her work. “It gives us a real connection to what we’re trying to do,” she says.
Knee points to the advent of greater patient-focused medicine development, combined with industry interest in studying smaller patient populations, as hugely powerful forces. “I think that patients afflicted with rare and orphan diseases have a lot of reasons to be excited and hopeful that there’s going to be therapies for them.” says Knee.
The most challenging part of her job is understanding where to start on complex, rare diseases. “The pile of stuff we don’t yet understand about how those diseases progress is just gigantic. There’s a lot of good ideas, and a lot of people are taking a lot of different tacks. And I think that’s the best way to go about it. I am optimistic that one of them is going to be the right one.”
I think that patients afflicted with rare and orphan diseases have a lot of reasons to be excited and hopeful that there’s going to be therapies for them.
Her optimism is in part because Knee sees the changes being made in the biopharmaceutical industry now and in the future. Growing patient engagement is a part of this, as is an increase in clinical trials. With more clinical trials, more ideas and projects will be able to be tested.
While developing medicines for rare diseases can be a complex and slow process, Knee emphasizes that the most important thing about her job is working towards making patients’ lives better. “What I would say to patients is: Keep talking to us. We are really interested in what you have to say.”
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