FSCDR Presents Positive Results from Case Study of Voxelotor (GBT440) in Sickle Cell Disease Patient

HOLLYWOOD, Fla., — Oct. 31, 2017 — On Oct. 28, Lanetta Bronté, M.D., M.P.H., M.S.P.H. presented a positive results case study for a sickle cell patient with severe and symptomatic anemia, after receiving voxelotor, at the 45th Annual National Convention hosted by the Sickle Cell Disease Association of America in Atlanta.

Dr Bronté, president and founder of the Foundation for Sickle Cell Disease Research (FSCDR), is thankful that FSCDR’s patient was able to receive voxelotor through single-patient compassionate access. She’s hopeful to see continued positive results as more patients enroll into Global Blood Therapeutics, Inc’s (GBT) Phase 3 HOPE study.

Dr Bronté presented on Juan E. Caballero, a 67-year old male with the HbSS sickle cell genotype with severe anemia refractory to transfusions due to red cell antibodies that developed after receiving multiple blood transfusions. He also had moderate chronic obstructive pulmonary disease (COPD) that required supplemental oxygen therapy, recurrent and frequent pain exacerbations, extreme fatigue and clinical depression. Caballero was ineligible to participate in GBT’s Phase 3 HOPE study of voxelotor because of these circumstances, but was able to receive the drug through compassionate access.


Juan E. Caballero in the 1970’s. Today, he’s 67 years old with HbSS sickle cell genotype. He’s receiving voxelotor, an investigational drug created by Global Blood Therapeutics, Inc., through single-patient compassionate access.


After receiving voxelotor 900 mg orally, once daily, Caballero rapidly showed improvement in pain, fatigue and mental health (after one to two weeks). His hemoglobin levels rose quickly to approximately 1.5g/dL above baseline with a sustained increase over 66 weeks in range of 1 to 1.5g/dL. Reductions occurred in reticulocyte count (indicated increased production to replace damaged red blood cells) and bilirubin (a measure of red blood cell destruction) both consistent with diminished hemolysis.


Juan Caballero, Sept. 26, 2015, at FSCDR’s headquarters. He had to use oxygen 24/7.


Caballero’s blood oxygen saturation level improved on a standard walk test, from 86mmHg at baseline to 96mmHg at 65 weeks, after which he discontinued continuous oxygen supplementation. No hospitalization prompted by sickle cell pain has occurred since voxelotor initiation. His only treatment-related side effect, Grade 2 diarrhea, occurred nine weeks after beginning voxelotor treatment when the dose was increased to 1,500 mg daily. He dose returned to 900 mg and has had no further treatment-related side effects.


Compassionate access is an option provided by the U.S. Food and Drug Administration (FDA) to make available, prior to regulatory approval, investigational medicines for the treatment of serious or life-threatening diseases or conditions for which there are no ongoing clinical trials and there is a lack of satisfactory therapeutic alternatives.


Caballero was presented with the opportunity to receive voxelotor through compassionate access by Dr Bronté and Gershwin Blyden, M.D., at FSCDR.


Dr Bronté founded FSCDR in 2012 after noticing the extreme lack of care for a disease that affects three million individuals nationwide. In 2015, FSCDR opened the nation’s first standalone (not connected to hospitals or academic centers) outpatient center solely devoted to sickle cell care and services. This is historically significant to South Florida, where FSCDR’s headquarters site is located, as this community has one of the highest numbers in the U.S. of individuals affected by sickle cell, but did not have a center specifically made for sickle cell treatment.

The Foundation for Sickle Cell Disease Research’s flagship site in Hollywood, FL


FSCDR is opening two new sites: in Homestead, Fla., target date Jan. 2018 and in Liberty City, Miami, target date June 2018. FSCDR’s headquarters address is 3858 Sheridan Street, Suite S, Hollywood, FL 33021. To learn more about its innovate care and services, please visit www.fscdr.org, email info@fscdr.org or call 954-397-3251. Also, follow FSCDR on Facebook @fscdr, Twitter at @Fundsicklecell and Instagram @fscdr.


Juan Caballero in the 1970s. Today, he is thankful for voxelotor. As his conditioned worsened, he said he felt like a shell of a person. Now, he feels closer to “his old self.”

About Voxelotor in Sickle Cell Disease

Voxelotor (previously called GBT440) is being developed as an oral, once-daily therapy for patients with SCD. Voxelotor works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes voxelotor blocks polymerization and the resultant sickling of red blood cells. With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT believes that voxelotor may potentially modify the course of SCD. In recognition of the critical need for new SCD treatments, the U.S. Food and Drug Administration (FDA) has granted voxelotor Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. The European Medicines Agency (EMA) has included voxelotor in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.


GBT is currently evaluating voxelotor in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a Phase 3 clinical trial in patients age 12 and older with SCD. Additionally, voxelotor is being studied in the ongoing Phase 1/2 GBT440-001 trial and in the ongoing Phase 2a HOPE-KIDS 1 Study, an open-label, single- and multiple-dose study in pediatric patients (and 6-17) with SCD. HOPE-KIDS 1 is assessing the safety, tolerability, pharmacokinetics and exploratory treatment effect of voxelotor.


Global Blood Therapeutics, Inc.

Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. GBT is developing its late-stage product candidate, voxelotor, as an oral, once-daily therapy for sickle cell disease.


Statements made in this press release are based off data collected and provided by the Foundation for Sickle Cell Disease Research, or, are from Global Blood Therapeutics Inc.’s press release published Oct. 28, 2017. They are objective and intended to consciously inform the sickle cell community of possible treatment options in the future.


Contact Information:

Kyla Thorpe

Foundation for Sickle Cell Disease Research


Foundation for Sickle Cell Disease Research Joins New Sickle Cell Disease Coalition, Issues Call to Action to Improve Treatment and Care

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September 12, 2016

Sickle Cell Disease Coalition launches a call to action on disease that has been neglected for far too long

(WASHINGTON, September 12, 2016) — Today, FSCDR joins other leading patient advocacy groups, researchers, clinicians, policymakers, industry, and foundations in a collective effort to improve sickle cell disease (SCD) care, early diagnosis, treatment, and research, both in the United States and globally. The newly formed Sickle Cell Disease Coalition’s first order of business is an international call to action to change the status quo of SCD.

SCD is an inherited, lifelong disorder characterized by red blood cells that become rigid and sickle-shaped, which causes them to stick together and block the flow of oxygen to the body, leading to intense pain and other serious issues such as stroke, organ failure, and even death. SCD affects nearly 100,000 Americans. It is a growing global health problem, and it is estimated that by 2050 the number of people with SCD will increase by 30 percent globally[i]. Over the last century, several important discoveries have led to improved diagnosis and treatments, but people with this disease still have severe complications and shorter life expectancy. Many are unable to access the care they need, and they live with pain and disability due to a lack of effective treatment options.

Tuesday, at a press conference and formal launch of the Coalition at the Knight Conference Center of the Newseum in Washington, D.C., the Coalition announced a call to action on SCD.  The American Society of Hematology and several members of the Coalition issued the State of Sickle Cell Disease: 2016 Report, which outlines unmet needs in four priority areas: Access to care, training and professional education, research and clinical trials, and global health. The Report not only illustrates that significant improvements are needed across all areas, it also highlights strategies for change.

“Around the world, people with sickle cell disease face unique challenges. In countries without early screening programs, patients are at risk for early death and childhood stroke.  Due to the lack of access to appropriate care in the United States, people with sickle cell disease still face a lifetime of mental and physical disability.  We can no longer accept that this is the status quo,” said Charles S. Abrams, MD, President of the American Society of Hematology, the organization spearheading the Coalition. “The Sickle Cell Disease Coalition is bringing together a number of groups that care about this disease and are committed to harness opportunities to effectuate change.”

The State of Sickle Cell Disease: 2016 Report includes several goals to help improve the state of SCD treatment and care. These goals include:

  • Shifting from acute care of complications to a chronic care model that focuses on prevention of crises, early identification, and intervention for common complications;
  • Increasing the availability of providers with SCD expertise through trainings of primary care providers and hematologists;
  • Linking research and care more closely through robust clinical research involving a wide range of partners to find new therapies, recruit more individuals with SCD, and achieve clear outcomes faster;
  • And designing, testing and implementing sustainable care and pain management approaches in countries with limited resources.

FSCDR is uniting with other organizations in the Coalition to address these areas of need.   In particular, FSCDR continues to support innovative research and implement effective treatment plans for sickle cell patients.

The Coalition will provide a unique platform to encourage stakeholders to work together to develop and implement important projects and activities that will ultimately help the SCD community, and improve outcomes for individuals with this disease. For more information on the Coalition and its mission, visit www.scdcoalition.org.

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About Foundation for Sickle Cell Disease Research

The Foundation for Sickle Cell Disease Research is a nonprofit organization in Hollywood, Florida, committed to supporting innovative research and improving the lives of patients with sickle cell. FSCDR collects data for scientific assessments of medical outcomes, completed through analyzing our patient registry and participating in clinical trials. In 2015, FSCDR opened the Sickle Cell Care and Research Network, where patients can receive outpatient care from a primary doctor, Monday through Friday, 9 a.m. to 5:30 p.m.

About the Sickle Cell Disease Coalition

The mission of the Sickle Cell Disease Coalition (SCDC) (www.scdcoalition.org) is to help amplify the voice of the SCD community, promote awareness, and improve outcomes for individuals with SCD. The Coalition is focused on promoting research, clinical care, education, training, and advocacy as well as providing a platform to encourage stakeholders to work together to develop and implement important projects and activities that will ultimately help the SCD community and improve outcomes for individuals with this disease. The Coalition is comprised of leading patient advocacy groups, people with SCD and their families, researchers, clinicians, policymakers, industry, and foundations with an interest in SCD.

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