When Sanford Simon’s daughter was diagnosed with a rare and potentially deadly liver cancer (FLC), he used his skills as a scientist to gather a collaborative group of patients, families, doctors, and researchers to find a new way of looking at these very rare cancers.

He sees how research on one type of cancer can lead to insights about others:

“Both journals and study sections repeatedly judged that FLC is too rare to justify studying, ignoring the evidence that a well-defined cancer, even if rare, can provide critical insights to reveal general principles. For example, the study of glioblastoma led to the discovery of mutations in isocitrate dehydrogenases, which have since been implicated in many human diseases, including other cancer types.”

Patient and family input was critical to make progress. OurBrainBank founder Jessica Morris (1963-2021) passionately believed in the power of patients.

“I discussed the potential of these patient groups with Francis Collins, the long-term director of the NIH. He emphasized that the success of patient engagement should not be judged by the ability of scientists and clinicians to give the patients a seat at the table. Instead, he said we should bring them into the kitchen and involve them in the planning and preparation of the meal.”

Read the full article in Nature.