Participatory Partnering
Emily Kramer-Golinkoff, who was trained as a bioethicist from the University of Pennsylvania, is “the engine behind a multi-level approach to changing the game for the advancement of treatments for patients with certain protein production mutations of Cystic Fibrosis (CF) called nonsense mutations — all while living with the condition 24/7,” according to an article by Joe Kim in Lilly Trial Guide. Kramer-Golinkoff provides funding for many phases of scientific research, advocates for patients with Cystic Fibrosis, participates in clinical research, influences the design of research, connects disparate scientific fields and scientists and inspires action through her public speaking, Kim said.
Kramer-Golinkoff has co-founded Emily’s Entourage (EE), a nonprofit foundation that raises funds and awareness to accelerate research for new treatments and a cure for nonsense mutations of CF. She has a number of clinical programs in process.
Lilly attempts to partner with patients in as much of the clinical research design process as possible through its CoDESIGN capability. Kramer-Golinkoff is using her influence as a patient in this activity, especially when scientists may not have the tools to engage patients at this level.
While 90% of the CF community may benefit from life-changing breakthroughs, patients with rare forms of CF caused by nonsense mutations have few treatment options. They want to get into research and help to accelerate medical advancements for CF. EE wants to demonstrate the “laser focus of creating clinical research opportunities for this majorly underserved population.” According to Kramer-Golinkoff, “Clinical research leads to new drugs, which can bring life-saving advancements…clinical research equals hope.”
EE is now funding interventional drug research. It is also helping researchers to design patient-centered trials. It starts with defining the entry criteria for research participation. Another important area of influence for EE is broadening the research viewpoint. She can elucidate the patient perspective that may be missed because certain populations are systematically excluded from trials. As he explained, “There are some correlates that are not reflected in the research…and sometimes [the researchers] just don’t know…there is no mechanism to listen or learn.”
In addition, EE has brought attention to other relationships among various conditions and symptoms and CF. They include diabetes, sleep patterns and other conditions affected by declining lung health. Scientists can use this information to find solutions that could improve the lives of CF patients.
In the last few years, EE has developed “a sophisticated audience of like-minded patients and advocates devoted to the mission of bringing much-needed therapies to patients with nonsense mutations of CF.” Besides “growing a community of patients organically through social media, local and national media and speaking engagements,” EE has also developed a patient registry for people with nonsense mutations of CF to help researchers expedite development of treatments.
All of these factors offer great value to the CF community of people who have nonsense mutations. Another huge accomplishment of EE is the way it has changed research for this field. As a result, the whole “toolbox of research has been brought to the next level.”
