Findings from a Cleveland Clinic-led clinical trial showed that the use of bempedoic acid (a cholesterol-lowering drug) in statin-intolerant patients – who have not yet had a cardiovascular event, but do have risk factors, like diabetes – significantly reduced the chance of death from heart disease and other major adverse cardiovascular events such as heart attacks and strokes.

Findings were presented today during a Late Breaking Science Session at the American Diabetes Association’s 83rd Annual Scientific Session in San Diego and simultaneously published in the Journal of the American Medical Association.

Back in March, results from the CLEAR Outcomes trial showed that bempedoic acid reduced adverse cardiovascular outcomes in 14,000 statin intolerant patients. Within this CLEAR trial, there were 4200 patients enrolled with high risk for heart disease but no previous cardiovascular events (primary prevention). After six months of treatment, bempedoic acid, compared with placebo, reduced LDL cholesterol by 23.2% and reduced inflammation measured by C-reactive protein by 22.7%.  In this primary prevention subgroup, there was also a 30% reduction in major cardiovascular events, a 39% reduction in death from heart disease and a 39% decline in heart attacks.  

“These findings emphasize the large benefits from lipid-lowering therapy in patients with no prior cardiovascular event, but who are at risk for a first event,” said the study’s lead author Steven E. Nissen, M.D., Chief Academic Officer of the Heart Vascular & Thoracic Institute at Cleveland Clinic. “Approximately two-thirds of the participants had diabetes, which further supports recommendations that primary prevention patients with diabetes should be treated with cholesterol-lowering therapies.”

Primary prevention patients are currently under-treated in the United States, with more than half of patients at high risk for a cardiovascular event not currently receiving cholesterol lowering drugs. Few clinical trials in recent years have studied patients who have not yet had a cardiac event.

Elevated LDL cholesterol can accumulate in the walls of blood vessels, creating blockages and raising the risk of heart attack or stroke. Statins are the standard first-line treatment for the prevention of cardiovascular disease and work by lowering cholesterol levels in the blood.  However, some patients struggle with adverse side effects – particularly muscle pain or weakness – that prevent them from using statins at recommended doses. Bempedoic acid differs from statins by not activating until it reaches the liver which limits the drug’s potential to cause adverse muscle effects on muscle.

Adverse effects observed with bempedoic acid included a higher incidence of gout and gallstones.

The study was funded by Esperion Therapeutics, developer of bempedoic acid.

Dr. Nissen has served as a consultant for many pharmaceutical companies and has overseen clinical trials for AbbVie, Amgen, AstraZeneca, Bristol Myers Squibb, Eli Lilly, Novartis, Mineralys, Silence Therapeutics, and Pfizer. However, he does not accept honoraria, consulting fees or other compensation from commercial entities.

Findings from a Cleveland Clinic-led clinical trial showed that the use of bempedoic acid (a cholesterol-lowering drug) in statin-intolerant patients – who have not yet had a cardiovascular event, but do have risk factors, like diabetes – significantly reduced the chance of death from heart disease and other major adverse cardiovascular events such as heart attacks and strokes. Findings were presented today during a Late Breaking Science Session at the American Diabetes Association’s 83rd Annual Scientific Session in San Diego and simultaneously published in the Journal of the American Medical Association. Back in March, results from the CLEAR Outcomes trial showed that bempedoic acid reduced adverse cardiovascular outcomes in 14,000 statin intolerant patients. Within this CLEAR trial, there were 4200 patients enrolled with high risk for heart disease but no previous cardiovascular events (primary prevention). After six months of treatment, bempedoic acid, compared with placebo, reduced LDL cholesterol by 23.2% and reduced inflammation measured by C-reactive protein by 22.7%.  In this primary prevention subgroup, there was also a 30% reduction in major cardiovascular events, a 39% reduction in death from heart disease and a 39% decline in heart attacks.     “These findings emphasize the large benefits from lipid-lowering therapy in patients with no prior cardiovascular event, but who are at risk for a first event,” said the study’s lead author Steven E. Nissen, M.D., Chief Academic Officer of the Heart Vascular & Thoracic Institute at Cleveland Clinic. “Approximately two-thirds of the participants had diabetes, which further supports recommendations that primary prevention patients with diabetes should be treated with cholesterol-lowering therapies.” Primary prevention patients are currently under-treated in the United States, with more than half of patients at high risk for a cardiovascular event not currently receiving cholesterol lowering drugs. Few clinical trials in recent years have studied patients who have not yet had a cardiac event. Elevated LDL cholesterol can accumulate in the walls of blood vessels, creating blockages and raising the risk of heart attack or stroke. Statins are the standard first-line treatment for the prevention of cardiovascular disease and work by lowering cholesterol levels in the blood.  However, some patients struggle with adverse side effects – particularly muscle pain or weakness – that prevent them from using statins at recommended doses. Bempedoic acid differs from statins by not activating until it reaches the liver which limits the drug’s potential to cause adverse muscle effects on muscle. Adverse effects observed with bempedoic acid included a higher incidence of gout and gallstones. The study was funded by Esperion Therapeutics, developer of bempedoic acid. Dr. Nissen has served as a consultant for many pharmaceutical companies and has overseen clinical trials for AbbVie, Amgen, AstraZeneca, Bristol Myers Squibb, Eli Lilly, Novartis, Mineralys, Silence Therapeutics, and Pfizer. However, he does not accept honoraria, consulting fees or other compensation from commercial entities.

The Minister of State for the Public Service the Hon. Pia Glover said in this administration’s first 20 months as a government, after re-opening the economy, it strengthened the country’s overburdened healthcare system by upgrading numerous clinics and hired dozens of doctors, nurses, and other support staff.

“We increased nurses’ salaries and benefits and improved several wards in the Princess Margaret Hospital,” she added during her contribution to the 2023/24 National Budget Debate in the House of Assembly, Monday, June 12, 2023.

The Minister of State explained that the government addressed challenges in education by hiring over 200 teachers while increasing salaries and benefits packages.

“We got those who dropped out of school during the pandemic back in the classroom by the hundreds and are now getting them caught up through our learning loss interventions, ably led by the honourable members for Englerston & Yamacraw.”

She said for those who would have graduated or were near graduation, the Smart Start Programme is providing them with support in entering the job market.

The State Minister said many people are receiving hands-on experience and training through the PS-PEP programme launched in this government administration’s first year – this included the first ever cohort of differently abled people who are currently working with private sector partners, receiving salaries, gaining experience, and being respected, included, and treated with the dignity they deserve.

Labour Relations

The Minister of State explained that during the government’s first 20 months, the Labour Relations Unit of her ministry worked diligently from day one and as a result, the government signed 18 Union Agreements, after years of stagnancy and bad relations under the previous administration.

“That is about one new agreement per month. And in each one of those agreements, our people, the teachers, nurses, police officers, and clerical staff, all the people who make our country tick, received higher pay, better insurance packages, and improved benefits.”

She also noted that the government has decreased the promotions backlog and we initiated a digitization process for document management to speed up public service operations. “We increased public servant pensions and increased salaries for thousands of public servants.” 

The Minister of State for the Public Service the Hon. Pia Glover said in this administration’s first 20 months as a government, after re-opening the economy, it strengthened the country’s overburdened healthcare system by upgrading numerous clinics and hired dozens of doctors, nurses, and other support staff. “We increased nurses’ salaries and benefits and improved several wards in the Princess Margaret Hospital,” she added during her contribution to the 2023/24 National Budget Debate in the House of Assembly, Monday, June 12, 2023. The Minister of State explained that the government addressed challenges in education by hiring over 200 teachers while increasing salaries and benefits packages. “We got those who dropped out of school during the pandemic back in the classroom by the hundreds and are now getting them caught up through our learning loss interventions, ably led by the honourable members for Englerston & Yamacraw.” She said for those who would have graduated or were near graduation, the Smart Start Programme is providing them with support in entering the job market. The State Minister said many people are receiving hands-on experience and training through the PS-PEP programme launched in this government administration’s first year – this included the first ever cohort of differently abled people who are currently working with private sector partners, receiving salaries, gaining experience, and being respected, included, and treated with the dignity they deserve. Labour Relations The Minister of State explained that during the government’s first 20 months, the Labour Relations Unit of her ministry worked diligently from day one and as a result, the government signed 18 Union Agreements, after years of stagnancy and bad relations under the previous administration. “That is about one new agreement per month. And in each one of those agreements, our people, the teachers, nurses, police officers, and clerical staff, all the people who make our country tick, received higher pay, better insurance packages, and improved benefits.” She also noted that the government has decreased the promotions backlog and we initiated a digitization process for document management to speed up public service operations. “We increased public servant pensions and increased salaries for thousands of public servants.” 

Researchers presenting preliminary data from a clinical trial aimed at discovering a cure for sickle cell disease reveal positive results among its first patients. 

Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies.

Researchers involved in the multicenter RUBY Trial presented an update on the safety and effectiveness of a single dose of EDIT-301, an experimental one-time gene editing cell therapy that modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease. Results were presented today at the European Hematology Association Hybrid Congress in Frankfurt, Germany.

The first four patients, two of whom are participating in the RUBY trial at Cleveland Clinic Children’s, had their stem cells collected for gene editing. The patients then underwent chemotherapy treatment to destroy their remaining bone marrow, making room for the repaired cells that were later infused back into their body. 

This is the first time a novel type of CRISPR gene-editing technology – known as CRISPR/CA12 - is being used to edit human cells in a clinical trial. This technology is a highly precise tool to modify blood stem cells genomes that can potentially enable robust, healthy blood cell production.

The data showed new white blood cells in all four patients at about four weeks with no severe adverse effects. Patients also achieved a normal level of hemoglobin, which is the most important component of red blood cells that carry oxygen throughout the body. The patients also have been free of sickle cell disease’s associated pain attacks for a period of 11 months and seven months following therapy. 

“New treatments like this are critical for people who have sickle cell disease,” said Rabi Hanna, M.D., director of the pediatric blood and bone marrow transplant program at Cleveland Clinic Children’s and principal investigator at Cleveland Clinic Children’s. “These initial results provide hope that this new technology will continue to show progress as we work toward creating a possible functional cure for this devastating and life-threatening disease.”

While there are an estimated 1 million to 3 million people in the United States who have the sickle cell trait, there are only about 100,000 people with sickle cell disease.  Sickle cell trait and the disease are found more often in certain ethnic groups, including African Americans. In the United States, about one in 365 African American babies have sickle cell disease.

Sickle cell disease is an inherited blood disorder that leads to the production of abnormal hemoglobin, which is a red protein responsible for transporting oxygen in the blood. Normal red blood cells are round and can move through small blood vessels to deliver oxygen. However, in people with sickle cell disease, the genetic change in DNA causes a chemical alteration in hemoglobin and alters the shape of red blood cells into a sickle, blocking them from passing through narrow blood vessels. They can clog or break apart which also leads to decreased red blood cell life, and increased iron storage in the liver and heart. This can cause conditions such as liver fibrosis, liver failure, stroke, cardiomyopathy and heart failure along with severe pain.

For most people with the disease, medications can modify disease severity and treat symptoms. However, despite current therapies, the average life of a sickle cell patient, is in the mid 40s. A blood or marrow transplant can cure sickle cell disease, but the transplant often requires a sibling donor and has the potential for severe graft-versus-host disease, which is when donor bone marrow or stem cells attack the recipient.

The RUBY Trial aims to enroll 40 adult patients, ages 18 to 50, with severe sickle cell disease. Patients will be monitored closely after treatment for up to two years.

Researchers presenting preliminary data from a clinical trial aimed at discovering a cure for sickle cell disease reveal positive results among its first patients.  Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies. Researchers involved in the multicenter RUBY Trial presented an update on the safety and effectiveness of a single dose of EDIT-301, an experimental one-time gene editing cell therapy that modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease. Results were presented today at the European Hematology Association Hybrid Congress in Frankfurt, Germany. The first four patients, two of whom are participating in the RUBY trial at Cleveland Clinic Children’s, had their stem cells collected for gene editing. The patients then underwent chemotherapy treatment to destroy their remaining bone marrow, making room for the repaired cells that were later infused back into their body.  This is the first time a novel type of CRISPR gene-editing technology – known as CRISPR/CA12 - is being used to edit human cells in a clinical trial. This technology is a highly precise tool to modify blood stem cells genomes that can potentially enable robust, healthy blood cell production. The data showed new white blood cells in all four patients at about four weeks with no severe adverse effects. Patients also achieved a normal level of hemoglobin, which is the most important component of red blood cells that carry oxygen throughout the body. The patients also have been free of sickle cell disease’s associated pain attacks for a period of 11 months and seven months following therapy.  “New treatments like this are critical for people who have sickle cell disease,” said Rabi Hanna, M.D., director of the pediatric blood and bone marrow transplant program at Cleveland Clinic Children’s and principal investigator at Cleveland Clinic Children’s. “These initial results provide hope that this new technology will continue to show progress as we work toward creating a possible functional cure for this devastating and life-threatening disease.” While there are an estimated 1 million to 3 million people in the United States who have the sickle cell trait, there are only about 100,000 people with sickle cell disease.  Sickle cell trait and the disease are found more often in certain ethnic groups, including African Americans. In the United States, about one in 365 African American babies have sickle cell disease. Sickle cell disease is an inherited blood disorder that leads to the production of abnormal hemoglobin, which is a red protein responsible for transporting oxygen in the blood. Normal red blood cells are round and can move through small blood vessels to deliver oxygen. However, in people with sickle cell disease, the genetic change in DNA causes a chemical alteration in hemoglobin and alters the shape of red blood cells into a sickle, blocking them from passing through narrow blood vessels. They can clog or break apart which also leads to decreased red blood cell life, and increased iron storage in the liver and heart. This can cause conditions such as liver fibrosis, liver failure, stroke, cardiomyopathy and heart failure along with severe pain. For most people with the disease, medications can modify disease severity and treat symptoms. However, despite current therapies, the average life of a sickle cell patient, is in the mid 40s. A blood or marrow transplant can cure sickle cell disease, but the transplant often requires a sibling donor and has the potential for severe graft-versus-host disease, which is when donor bone marrow or stem cells attack the recipient. The RUBY Trial aims to enroll 40 adult patients, ages 18 to 50, with severe sickle cell disease. Patients will be monitored closely after treatment for up to two years.

 

In recognition of lupus awareness month in May, the Insurance Commission of The Bahamas recently made a donation to local non-profit support group Lupus 242. This is the third year that the Commission has partnered with the group to assist its members with the purchase of medication and pharmaceutical supplies. Vice President of Lupus 242 Shonalee Johnson, (second from right), thanked Superintendent of Insurance Michele Fields (center) and her team at the Insurance Commission for continuing to support persons in The Bahamas living with the chronic disease. Since its inception in 2012, Lupus 242 has focused on raising awareness and providing education and support to its members.

This month, the group held a series of events including a POP-UP Run and a paint n sip art fundraiser. Lupus 242 also encouraged Bahamians everywhere to P.O.P (Put On Purple) For Lupus every Friday in May. The month ended with a Health Talk on Saturday May 27th at UB with Rheumatologists Dr. K. Neil Parker and Dr. Anishka Rolle along with HR Specialist Lashanta Smith who spoke on Managing Lupus In The Workplace. Pictured from l to r: Yolande Rolle, Deputy Legal Counsel, Insurance Commission, Sharanda Humes, Legal Officer, Insurance Commission, Michele Fields, Superintendent of Insurance, Shonalee Johnson, Vice President of Lupus 242 Association and Jamell Bodie, Manager-Supervision at the Insurance Commission and a lupus fighter. To support the cause, follow @Lupus242 on Facebook or Instagram, visit www.lupus242.org or call 242-424-4279.