One drug, many rare diseases. Inside the movement to make rare drug development more profitable

CNBC Advisory Board member Dr. Anna Greka has built her career on finding connections between different rare diseases. Greka, a Harvard professor and physician at Mass General Brigham, is also a member of the Broad Institute. At her lab, she oversees the Ladders to Cures Accelerator, an initiative designed to speed up the development of treatments for rare disease patients. It’s a model she hopes is able to attract more investment to the space. 

Greka and her team are working on identifying where different rare diseases converge and share a single biological mechanism. Greka calls this approach “nodal biology”. By identifying areas where rare diseases overlap, Ladders to Cures hopes to develop new types of drugs that can be used to treat multiple conditions. By developing single drugs capable of treating multiple diseases, the addressable patient population of those drugs becomes much bigger. That can make a drug more profitable, which Greka says is key to building a rare drug development model that is economically sustainable. 

Greka has already shown that the nodal approach can work. In 2019, she and her team published a paper outlining a mechanism they discovered for a rare kidney disease that also plays a role in diseases that impact the eye and brain. With the Ladders to Cures Accelerator, Greka hopes to discover even more nodal connections and start to develop drugs that can treat them. 

On October 27th, the Broad Institute will host the 3rd Annual Ladders to Cures Symposium. Greka will be there, along with her colleagues Dr. David Liu, Dr. Winston Yan, and MIT economist Andrew Lo.  

Ahead of the event, Becky Quick sat down with Greka to ask about the work being done at Ladders to Cures and the impact she hopes the accelerator can have on the rare community.

CNBC Cures is underwritten by Alexion, AstraZeneca Rare Disease.