FSCDR receives land parcel to build first sickle cell hospital in Miami-Dade County

FOR IMMEDIATE RELEASE

July 25, 2018

 

MIAMI — On Tuesday the Miami-Dade County Board of County Commissioners Meeting passed Resolution 14A2, declaring surplus county-owned land and approving a lease for Florida Sickle Inc. (d/b/a/ Foundation for Sickle Cell Disease Research). The Resolution is sponsored by Commissioner Audrey M. Edmonson and co-sponsored by Commissioners Daniella Levine Cava and Barbara J. Jordan. FSCDR is to build a hospital for sickle cell disease, focused on medical care and clinical research, the first of its kind in the United States of America.

The Foundation for Sickle Cell Disease Research was founded by Dr. Lanetta Bronté in December 2012 after working closely with the sickle cell disease community and noticing the extreme lack of care for a disease and trait that affects three million people nationwide. In February 2015, FSCDR opened the nation’s first standalone outpatient center solely dedicated to sickle cell disease care and services. This is historically locally significant, as the South Florida community has one of the nation’s highest numbers of individuals affected by sickle cell.

The sickle cell hospital is to expand on the services offered at FSCDR’s flagship center in Hollywood, Fla. This includes wraparound services to improve the overall quality of life for an individual with sickle cell disease. At the flagship center, patients are currently provided with: treatment by a hematologist-oncologist, care by a sickle cell-trained RN, port access for blood drawing and flushing, a program to reduce visits to the emergency room, a program to reduce inpatient hospitalization, patient-tailored pain management, disability evaluation, school IEP, social resource needs assessment, neurocognitive evaluation with a neuropsychologist, preventative health services, flu vaccinations, care coordination, access to their electronic health record, opportunities to enroll into clinical trials and a MedicAlert Foundation ID.

 

For all press inquiries, please email kthorpe@fscdr.org.

FSCDR’s Experience with Voxelotor (GBT440) Treatment in Patients with Severe Sickle Cell Disease

FOR IMMEDIATE RELEASE

December 14, 2017

ATLANTA — Lanetta Bronté, M.D., M.P.H., M.S.P.H., founder and chief health officer of the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Fla., presented results from seven adult sickle cell disease patients with severe anemia and multiple co-morbidities who were not eligible to participate in Global Blood Therapeutics’s (GBT) Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study (abstract #3545), at the 59th American Society of Hematology Meeting & Exposition on Monday evening.

“Patients with severe SCD, who often suffer life-threatening complications, are refractory to conventional therapy and, thus, are very difficult to manage because treatment options are very limited,” said Dr. Bronté. The patients, four women and three men, age 22 to 67, received voxelotor for six to 17 months through single-patient compassionate access and monitored over 24 weeks at Dr. Bronté’s outpatient center. Results showed:

  • Improvements in multiple clinically important endpoints, including hemoglobin levels, daily pain, hospitalization for vaso-occlusive crisis (VOC), transfusions, depression and overall well-being.
  • Hemoglobin values rose in all patients, with an increase exceeding 1 g/dL in 5 of 7 patients.
  • Hospitalizations for VOC, the conventional measure of SCD pain, fell by 67 percent during 24 weeks of voxelotor treatment, compared with the 24-week period immediately before treatment was initiated (9 vs 28, respectfully), corroborating patient reports of improved well-being and quality of life.
  • The number of transfusions decreased by 60 percent during 24 weeks of voxelotor treatment compared with the 24-week period immediately before treatment initiation (13 vs 33, respectively). Of the six patients who received transfusions prior to voxelotor, two received no transfusions after treatment.
  • Improvements in blood oxygenation were observed in all four patients that had low room air oxygen saturations at baseline, and the two patients who were oxygen-dependent prior to treatment initiation no longer required continuous supplemental oxygen during 24 weeks of voxelotor treatment.
  • Two patients died while receiving voxelotor under compassionate access. Both had multiple co-morbidities and advanced organ injury prior to initiation of treatment (e.g., chronic kidney disease, hepatic dysfunction, iron overload), and the treating physicians concluded that voxelotor did not contribute to either death.
  • Voxelotor was well tolerated for up to 17 months at a dose of 9— mg with no discontinuations, and no voxelotor-related serious adverse events occured.

“We are grateful that these seven patients at our center were able to receive voxelotor through compassionate access and are so pleased with the clinical improvements we saw on meaningful endpoints,” said Dr. Bronté.

FSCDR is a leading innovator in sickle cell disease. Dr. Bronté opened the center in Nov. 2014 to address the growing trend of fragmented care and an increase in emergency room visits and inpatient utilization in adolescents and adults with SCD. FSCDR runs the U.S.’s only outpatient center solely devoted to sickle cell care and services and is independent from any hospital system or academic institution.

Compassionate access is an option facilitated 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 disease or conditions for which there are no ongoing clinical trials and there is a lack of satisfactory therapeutic alternatives. Voxelotor is an investigational treatment and controlled clinical trials are needed to confirm the clinical benefits and safety seen in the case study of seven patients.

You can read the full poster, here.

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

kthorpe@fscdr.org

FDA approves new treatment for sickle cell disease: Endari

FDA approves new treatment for sickle cell disease: Endari

This is the first drug approval for sickle cell disease in nearly 20 years

The U.S. Food and Drug Administration approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. This comes after nearly 20 years without a new drug approval.

Sickle cell disease, an inherited blood disorder in which the red blood cells are abnormally shaped, restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. Approximately 100,000 people in the United States have sickle cell disease, most commonly in African-American and Latinos.

Endari was studied in a randomized trial of patients ages five to 58 years old with sickle cell disease and two or more painful crises within the last year, prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo and evaluated over 48 weeks.

Patients treated with Endari experienced:

  • Fewer hospital visits for pain
  • Fewer days in the hospital
  • Fewer occurrences of acute chest syndrome

Common side effects include: constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.

The FDA granted the approval of Endari to Emmaus Medical Inc.

Information from www.fda.gov.

Sickle cell anemia, 3D illustration showing blood vessel with normal and crescent shaped cells. Adobe Stock.

 

New Research Suggests SANGUINATE™ Reduces the Number of Sickled Red Blood Cells in Patients with Vaso-Occlusive Crisis

vials

New Research Suggests SANGUINATE™ Reduces the Number of Sickled Red Blood Cells in Patients with Vaso-Occlusive Crisis

Restoration of Normal Morphology May Reduce Opiate Use and Prevent Hospitalization

SOUTH PLAINFIELD, N.J., May 1, 2017 – SANGUINATE™, the only investigational biopharmaceutical product currently in clinical development for the treatment of multiple comorbidities of sickle cell disease (SCD), is effective in returning red blood cells to a more normal morphology (shape) in patients experiencing vaso-occlusive crisis (VOC) related to SCD, according to research presented at the 11th Annual Symposium of the Foundation for Sickle Cell Disease Research (FSCDR) in Fort Lauderdale, Fla.

Sickle cell disease is a group of chronic genetic disorders that affects an estimated 70,000 to 80,000 Americans.1 Vaso-occlusive crisis, the most common complication of SCD, is an extremely painful, potentially deadly condition characterized by abnormal hemoglobin, a protein in red blood cells that transports oxygen from the lungs to tissues and organs in the body and carries carbon dioxide back to the lungs. 2,3 Cells affected by VOC become sickle- or crescentshaped, a deformity that prevents them from flowing freely through blood vessels, potentially causing excruciating pain and other symptoms that may lead to death. 4 VOC results in approximately 197,000 emergency department visits each year in the U.S. and costs an estimated $356 million annually for pain management alone.5

SANGUINATE is a dual-gas transfer agent that has been shown to revert sickled red blood cells to a more normal shape.
“SANGUINATE is a dual-gas transfer agent that has been shown to revert sickled red blood cells to a more normal shape. These latest findings are consistent with prior in vitro study results showing that SANGUINATE may offer a prolonged therapeutic effect on cell morphology,” said Ronald Jubin, Ph.D., vice president of research and development at Prolong Pharmaceuticals. “We look forward to advancing our clinical program to further evaluate SANGUINATE’s potential benefits for people suffering from sickle cell disease.” At the FSCDR symposium, researchers presented preliminary findings from an ongoing Phase II clinical trial evaluating the safety and efficacy of SANGUINATE in the treatment of VOC. The trial seeks to determine whether infusing SANGUINATE during an acute VOC episode can reduce the need for intravenous (IV) opiates and prevent hospitalization for patients experiencing VOC. The investigators collected whole blood samples from 10 patients prior to SANGUINATE infusion, at the time of patient discharge, and 72 hours post-infusion. The samples treated with SANGUINATE showed a reduction in the number of abnormally shaped cells. The beneficial effect of SANGUINATE was evident within hours of infusion, and was sustained at the 72-hour sampling, indicative of a prolonged duration of effect. “Vaso-occlusive crisis is a debilitating, painful condition caused by sickling of the red blood cells,” states Gershwin Blyden, M.D., Ph.D., principal investigator at the FSCDR. “These findings add to the mounting clinical evidence supporting the effects of SANGUINATE to ameliorate a painful crisis in the acute phase.” 2 “Clinical research underway to evaluate SANGUINATE is just one example of the cutting-edge research advanced and discussed at the annual symposium of the Foundation for Sickle Cell Disease Research,” noted Lanetta Bronté, M.D., MPH, MSPH, president and founder of the FSCDR. “It’s a privilege to bring this innovative research to eligible and interested patients with sickle cell disease at our innovative outpatient center.”

About the FSCDR

The Foundation for Sickle Cell Disease Research (FSCDR) is a comprehensive, non-profit organization that provides a platform for researchers, healthcare providers, and those living with sickle cell disease. The FSCDR works collaboratively with academia, pharmaceutical, biotechnology and community organizations to identify best practices to help with the management and future care for sickle cell patients. In 2015, the FSCDR opened the Sickle Cell Care and Research Network in South Florida, the nation’s first standalone outpatient center completely devoted to sickle cell care and services.

About SANGUINATE™

SANGUINATE is the only biological product currently in clinical development for the multiple comorbidities of SCD, and has received an Orphan Drug Designation from the U.S. Food and Drug Administration. SANGUINATE facilitates the transfer of oxygen to oxygen-deprived cells and tissues and has also been shown to down-regulate the inflammatory response in blood samples of patients with SCD. Many of the comorbidities of SCD are caused by a spiraling cycle of sickling, hemolysis and blood vessel inflammation. These comorbidities include VOC, acute chest syndrome, leg ulcers and pediatric and adult stroke. By correcting oxygen levels and down-regulating inflammation, SANGUINATE may be effective in treating many of the debilitating, acute comorbidities associated with SCD. Phase I studies in healthy volunteers and patients with stable SCD have been completed. Phase II trials of SANGUINATE are also planned for VOC and leg ulcers secondary to SCD as well as for ischemia.

About Prolong Pharmaceuticals

Headquartered in South Plainfield, New Jersey, Prolong Pharmaceuticals, LLC, is developing products to treat several diseases and their debilitating comorbidities associated with reduced quality of life, increased medical cost and significant mortality. Prolong’s senior management team includes inventors of the most successful drug delivery technology in pharmaceutical history, PEGylation, now responsible for the development of a dozen drugs improving the quality of life for sufferers of hepatitis, kidney disease, and a number of other life-threatening diseases.

References

1Genetics Home Reference. Sickle cell disease. 2016. https://ghr.nlm.nih.gov/condition/sickle-cell-disease#diagnosis.

2National Institutes of Health, National Heart, Lung, and Blood Institute. Evidence-based management of sickle cell disease. Expert panel report. 2014. http://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines.

3National Institutes of Health, National Cancer Institute. NCI Dictionary of Cancer Terms. 2017. https://www.cancer.gov/publications/dictionaries/cancer-terms?cdrid=45108.

4National Institutes of Health, National Heart, Lung, and Blood Institute. What is sickle cell disease? 2016. http://www.nhlbi.nih.gov/health/health-topics/topics/sca.

5Wilson BH, Nelson J. Sickle cell disease pain management in adolescents: a literature review. Pain Manag Nurs. 2015;16(2):146–151.

Contact

Robert Murphy
SmithSolve, LLC
973-442-1555 ext. 116
robert.murphy@smithsolve.com

Your bank statement will have a charge from Integrative Educational Solutions, Inc. the Business Solutions Platform for the Foundation for Sickle Cell Disease Research. Dismiss