Many disadvantaged young Black people with sickle cell disease approach the healthcare system looking for pain relief only to hear providers quickly judge them as drug seekers — a reality that drives Global Blood Therapeutics (GBT) to make a difference for those suffering from the discredited condition.
Rare disease leaders are often directly affected by the illness their company is built on, and GBT is no exception. For example, the company’s CEO, Dr. Ted Love, developed a connection with sickle cell disease (SCD) patients as a medical student at Yale when he first observed the stigma often associated with the condition.
“This bias is something SCD patients often deal with their whole lives,” Love says. “That is why I jumped at the chance to join GBT and develop novel therapies to make an impact for SCD patients.”
GBT has now developed a robust pipeline of drug candidates aimed at changing the game for patients with SCD, a group of genetic, red blood cell disorders that can cause a range of painful and debilitating symptoms.
Despite a robust medical understanding of SCD, the condition is much more common in those of African descent and its patient community represents a historically underserved population in research.
“While the pathology behind SCD has been understood for decades, therapeutic innovation has significantly lagged,” Dr. Kim Smith-Whitley, executive vice president and head of research and development at GBT, says.
Earlier this year, the company notched a new approval for its lead candidate, Oxbryta.
Currently, there is no cure for SCD aside from bone marrow transplants. Yet, because of the risks involved in the procedure, it is reserved for children with a severe form of the disease.
Meanwhile, several medications to treat symptoms — such as the extreme pain associated with SCD — have been approved, but there has long been an unmet need for the anemia the disease can bring about. This is where Oxbryta comes in. On the heels of an FDA approval for children in December, the European Commission (EC) authorized the use of Oxbryta to treat SCD-related anemia in February.
Oxbryta, which won the 2021 Prix Galien USA “Best Biotechnology Product,” is the first medicine approved in Europe that directly inhibits sickle hemoglobin polymerization. Oxbryta increases hemoglobin levels and reduces sickling and hemolysis — the cause of long-term complications of sickle cell disease.
Following the subsequent EC marketing authorization for Oxbryta, GBT submitted an application to the MHRA for a Great Britain Marketing Authorization using the EC Decision Reliance Procedure.
“This is a key step forward in providing patients in the UK with an effective and innovative treatment for SCD,” Nicholls says. “It also reinforces GBT’s focus on reimagining what is needed to get important, groundbreaking treatments that provide hope to underserved patient communities into the hands of people who need them as quickly as possible.”
As GBT continues to advance its pipeline, drugs like Oxbryta are a testament to the company’s potential to modify the course of SCD for a patient community that has suffered from limited treatment options for far too long.
Given the historical lack of attention in SCD, listening to and understanding the needs of the community and supporting advocacy have been a core part of GBT’s strategy — even well before it had an approved medicine.
“GBT will continue to have a strong focus on supporting the SCD community through a variety of programs including Sickle Cell Speaks (SCS) which aims to inspire hope by showcasing stories of strength and dispelling misconceptions about SCD,” David L. Johnson, chief commercial officer at GBT, says. “In partnership with other community-based organizations, the campaign shares individual stories from a diverse group of patients and caregivers.”
With more than 65,000 followers on Facebook, SCS provides educational resources for understanding and managing SCD. The campaign also connects patients and caregivers to events and support communities.
"This bias is something SCD patients often deal with their whole lives. That is why I jumped at the chance to join GBT."
At the time of the approval of Oxbryta, GBT launched GBT Source Solutions, a comprehensive program — the first of its kind in SCD — for patients who are prescribed Oxbryta, that provides a wide range of practical, educational and financial support customized to each patient’s needs.
The GBT Foundation, a community-focused, charitable entity, is also aimed at improving the health and well-being of marginalized and underserved communities around the world, particularly for people living with SCD. The Access to Excellent Care for Sickle Cell Patients (ACCEL) Program, part of The GBT Foundation, provides grant funding to support novel projects aimed at accelerating the development of programs with the goal of addressing the gaps and disparities in care that people living with SCD face. Since its inception in 2019, GBT has provided over $2 million in funding to U.S. community-based organizations and institutions.
GBT has also forged partnerships that have a direct impact on the community, including joining with the Sickle Cell Disease Association of America to host the annual SCD Therapeutics Conference, a forum to discuss the latest advances and future trends for treating people with SCD.
In 2018, GBT began supporting the Access to Care Summit, a forum for those involved in the care of SCD patients to exchange ideas to improve the lives of people living with SCD and generate solutions for accessing quality care. The program is now managed by the Foundation for Sickle Cell Disease Research as an educational resource for the sickle cell community.
Love is not the only executive at the company who’s been impacted by SCD. Kobe Dufu, director of biology and one of the discoverers of Oxbryta, says he was motivated by the lack of innovation in SCD, and was one of the first scientists to join the GBT.
He was also inspired by his family connections with the disease. Two of his grandparents have the sickle cell trait, which means that a person has inherited the sickle cell gene from one of his or her parents. He also has two cousins with SCD — one who passed away at age 5.
Another cousin, who is a teacher in Ghana, has been a major influence on Dufu and informed him years ago about the extremely limited access to SCD treatments in Africa. These connections make his work at GBT intensely personal and meaningful.
The UK General Manager of GBT, Nigel Nicholls, says he has always been drawn to professional roles where he can make a difference for underserved patient communities.
His son Matthew lives with Peters Plus Syndrome, another rare disease, and has inspired him to seek ways to help improve the lives of others with rare diseases.
In those efforts, Nicholls led the company to secure a Promising Innovative Medicine (PIM) designation in the UK for Oxbryta, the first SCD treatment to receive it. Like many at GBT, Nicholls says that it’s the personal connection to a rare disease that gives him that extra drive to advance medicines for unmet needs.
“When Matthew was born, I dedicated myself to helping advance treatment options for rare diseases,” Nicholls says. “Every face I see behind that group is Matthew’s.”