A New Era for Sickle Cell Disease

June 1, 2019 11:00 am

Do you know what it’s like to “go from having a normal day in the morning to, by 3 p.m., being in debilitating pain, unable to walk, barely able to speak”? Mattie Robinson shares this experience and what it’s like to live with sickle cell disease in a new video released by the Alliance to Protect Medical Innovation.


The video, A Case for Innovation: Sickle Cell, features Mattie Robinson, a single mother and entrepreneur, as well as researchers at Global Blood Therapeutics, Inc. (GBT) telling stories of life with sickle cell, of innovation and of how the United States’ competitive environment has, so far, driven new cures and treatments for rare diseases.


Sickle cell disease is a rare disorder that affects approximately 100,000 Americans and, according to a 2014 study, leads to about 113,000 hospitalizations annually. The cumulative cost of these stays each year is at least $400 million.


Despite these costs to the system, for years, the amount of research dollars into sickle cell disease “lagged” behind other diseases, according to Dr. Josh Lehrer, senior vice president of development at GBT. When asked why he decided to focus on sickle cell, Dr. Ted Love, CEO at GBT, said he devoted himself to this research because treatments for rare diseases “have not gotten the attention they deserve.”  This understanding gives the researchers at the company a “sense of purpose and a sense of mission.”


Lehrer and Love explain that GBT engaged patients to understand the nature of the disease and its symptoms. This interaction allowed researchers to move its own treatment more quickly through development. GBT’s treatment for sickle cell could be available as early as next year.


This story is not only about GBT, Robinson, or sickle cell, however.


According to the National Institutes of Health, there are more than 7,000 rare diseases and some of these affect only a few dozen individuals. Cumulatively, however, rare diseases afflict about 30 million Americans.


Still, research funding lags. Any policy measure that erodes that pool of available research dollars will have profound effects on patients like Robinson who are longing for a cure for a disease that isn’t currently a household name.


Threats to innovation abound, but for now, there is hope.


As GBT CEO Love explains, “The U.S. has done an amazing job leading in innovation in healthcare.” He predicts that, over the next five years, “we’re going to win” because there will be “increasing numbers” of treatments on the market for rare diseases.


Robinson concludes, “It’s obvious that things are coming to a new era.”