LOGO
  • ,

    Ask Dr. Kevin: Understanding clinical trials and sickle cell disease

    Clinical trials are critical to bringing new medicines to people who need them, particularly those affected by conditions with limited treatment options, such as sickle cell disease (SCD). These studies, which determine the safety and effectiveness of new investigational treatments, rely on the participation of volunteers. Without enough volunteers, researchers are unable to complete the research required by the U.S. Food and Drug Administration (FDA) to evaluate if a new drug should be made available to patients. Therefore, the only way to get any drug approved by the FDA and accessible to patients in need is through clinical trials.

    Unfortunately, finding enough patient volunteers is often a challenge in conducting clinical trials, and this has been particularly true for trials exploring new treatment options for SCD, which occurs in one out of every 365 African American births. Although African Americans make up 12 percent of the U.S. population, they comprise only 5 percent of clinical trial participants overall.

    Fear and misunderstanding about clinical trials likely contribute to the difficulty of recruiting participants. Recently, Pfizer and the NNPA collaborated on a national poll consisting of 741 participants to learn more about perceptions of SCD and clinical trials in the Black community. A majority of respondents indicated a willingness to participate in future clinical trials for SCD, if given appropriate knowledge and recommendations from health care professionals. Of those who indicated that they would not participate in clinical trials, ‘fear of uncertainties’ was the most cited reason.

    To help address some of these uncertainties, I’m here to answer the most common questions about clinical trials. It is my hope that this information will help you make an informed decision should you or a loved one choose to participate in a clinical trial in the future.

    Are clinical trials safe?

    Strict guidelines and supervision are in place to protect the safety of people who take part in studies—from careful study design to periodic monitoring of study data by independent experts. That said, clinical trials are designed for research purposes, and because of this, there is some level of risk involved. However, before an investigational drug can be given to clinical trial volunteers, researchers must complete a rigorous screening and preclinical testing process (in the laboratory and in animals), which can take up to six years to complete.

    If I participate in a clinical trial, will I get a “sugar pill” or placebo instead of a real drug?

    Participants in a clinical trial using a placebo will always be informed if there is a possibility they could be receiving the placebo, which looks identical to the real drug but does not contain the active ingredient. However, the vast majority of studies are not placebo-controlled trials, and patients in the clinical trial who are not receiving the drug under study will receive the current standard of care available to the public. Patients who take part in clinical trials will never be asked to sacrifice quality of care.

    Will it be expensive to participate in a clinical trial?

    Patient care costs are generally covered by health insurance, as they are for tests and treatments you would receive even if you were not involved in the research. The majority of participants in clinical studies receive at least some reimbursement from their health insurance. Most often, the clinical trial sponsors will pay for the study therapy and insurance companies will pay for the routine care procedures, such as blood tests.

    Before participating in clinical trials, I recommend checking with the researchers regarding your financial responsibilities as a patient, as well as with your health insurance carrier regarding coverage.

    How do I find out about clinical trials?

    You can always talk to your doctor; however, he or she may not know about all available clinical trials that might apply to you. Only approximately 1 in 5 patients say that their health care professionals have talked to them about participating in a clinical trial.

    As for resources, the National Institutes of Health has an online database that is a great tool to search for appropriate trials: https://clinicaltrials.gov. Another great resource is “I’m In,” a campaign to build awareness about the importance of diversity in clinical trials, especially among African Americans, Asian Americans, and Hispanic populations. You can find more information on Pfizer’s Clinical Trial page, too.

     

    What’s the timeframe for clinical trials?

    I’m often asked, “Why does a new treatment take so long?” The short answer is that treatment takes as long as it does because the cardinal rule of medicine is “First, do no harm.” Thus, the development of a new therapy is a multi-stage, complex process that has to meet the highest standards of patient protection.

    Clinical trials, which generally take 5 to 10 years, are at the center of the rigorous science that demonstrates the safety and efficacy of a medicine and provides a thorough view of its benefits and risks, and is the only avenue to bring medications to patients in need.

    By Dr. Kevin Williams
    Chief Medical Officer, Pfizer Rare Disease Unit

    ONLINE: more information about sickle cell disease, the collaboration between the NNPA and Pfizer Rare Disease, and a new nationwide poll conducted by Howard University to deepen understanding and gauge perceptions around SCD and clinical trials among African Americans.

     

    Dr. Kevin Williams is the chief medical officer for Rare Disease at Pfizer. He pursued medicine after being inspired by his father’s work as a general practitioner in his hometown of Baton Rouge, Louisiana. Follow Pfizer on Facebook and Twitter.  This article is the third installment in the “Ask Dr. Kevin” series, brought to you by Pfizer Rare Disease in collaboration with the National Newspaper Publishers Association (NNPA) to increase understanding of sickle cell disease.

     

     

    Read more »
  • ,

    Cured of sickle cell

    Baton Rouge native confirmed as first person cured of disease

    image

    In the late 1940s and early 1950s, the nature of Sickle Cell began to become clearer and doctors and researchers proved that Sickle Cell comes from an inherited gene from both mother and father. To date, as many as 140 thousand Americans are living with Sickle Cell  with another 2 million people carrying a gene that could potentially be passed down to their children. But with so many Americans affected and all of the research done over the one hundred years since western discovery, there is no cure for the disease.

    However one Baton Rouge native subsequently had been cured through a marrow transplant meant to save her life from another disease.

    Here’s her story.

    In 1976, Kimberlin Wilson George was two years old and newly diagnosed with Sickle Cell Anemia. Beginning treatment at Earl K. Long under the care of Dr. Shelia Moore, the Wilson family learned more about the disease and became active participants with the Sickle Cell Foundation of Baton Rouge.

    “Throughout my childhood I would have a Sickle Cell crisis every other week,” George said. “I would remember my arms and legs being in excruciating unbearable pain. I would just lay there crying while parents and grandmother prayed and took turns rubbing my arms and legs. When the pain reached an intolerable level I would be on my way to the emergency room at our Lady of The Lake Hospital where they knew me well.”

    Because of the pain and extended hospital stays, George missed lots of school and activities that children her age would normally be involved.

    “Life as I saw it for me was just going to be filled with lots of pain and hospital stays,” she said.

    But by age 8, George said she experienced a Sickle Cell crisis she will never forget.

    “I was in lots of pain and had pneumonia,” she continued. “Of course I was admitted to the hospital and tests were run only to find more abnormalities. My parents were then put into contact with St. Jude Children’s Research Hospital in Memphis. Once there more tests were run and the only news I remember is that I had three months to live.”

    It was then that the Wilson family also learned that their daughter had AML Leukemia. She was admitted to St. Jude where doctors decided to experiment with one of their first bone marrow transplants to get rid of the cancer.

    George’s transplant – with marrow supplied by her younger brother Shongo – was done in 1983 in Birmingham at The University of Alabama Medical Center. She was then transported back to St Jude for one year.

    “The outcome of my transplant was miraculous,” George said. “I was not only cured of the Leukemia, but also of Sickle Cell. This stunned the doctors and was also proof that God answers prayers. After I was discharged and returned home, I was confined to the house for a while. This was ok with me because I knew that there would be no more pain and I could now live a much normal life.”

    George returned to school her ninth grade year and graduated in 1992. She went on to study at Xavier University of Louisiana, later transferring to Southern University A&M College where she graduated with a degree in child development.

    Finally healthy, George taught first grade for one year, then opened a childcare center that she operated for 11 years.

    “Throughout my adult life I ran into a few obstacles, the side effects from the medication, I thought that I would never have a family and I had a deteriorated hip bone, but I kept going strong,” George said.

    The only other medical issue George ever ran into again was a total hip replacement in 2002. She has since married and has three children.

    George’s results are extremely ill-typical and she is the first person ever documented to have been cured of the disease, which included chemotherapy.

    “I live a wonderful life, live it to the fullest and thank God for living it every day,” George said. “Because of my family, many other people, the bone marrow transplant and God, I stand before you today the first person in the world to be cured of Sickle Cell and the second person to have had two blood diseases still living.”

    One of the most important things that people can do is to get tested to see if they are carriers of the disease. The next step is to get informed. Sickle Cell Warriors is a fact-packed forum where patients can share information with each other (sicklecellwarriors.com), and the Sickle Cell Disease Association of America (sicklecelldisease.org) is a national advocacy group that caters to both patients and health care providers. Next, investigate clinical trials and get vaccinated because almost all sickle cell patients are immune-compromised, it’s all the more vital to keep on top of all recommended vaccinations.

    As a survivor, George would also suggest you get support, likewise many patients report getting tremendous benefit from support groups.

    “Understand you might not be the only one dealing with what you’re dealing with – always remain positive,” George said. “Just be as strong as you can, and always try to involve yourself with positive people.”

    To date, about 25 adults have received chemotherapy-free stem cell transplant for sickle cell disease in recent years. Approximately 85 percent have been cured, including Chicagoan Ieshea Thomas, who was the first Midwest patient to receive a successful stem cell transplant to cure her sickle cell disease without chemotherapy in preparation for the transplant, in 2012.

    By Leslie D. Rose
    The Drum

    Read more »
Back to Top
Facebook Auto Publish Powered By : XYZScripts.com