Category Archives: Hematology

Vaccine for Meningitis Shows Some Protection Against Gonorrhea

Scientists have not been able to develop a vaccine against the sexually transmitted disease gonorrhea, despite working toward one for more than 100 years.  However, they may have stumbled onto something that could provide clues to advance the development of such a vaccine.

Decades ago, in the late 1990s, a strain of meningitis B was reaching epidemic proportions in New Zealand. A vaccine, MeNZB, was developed to protect young people who were at the highest risk of getting this particular type. It did not provide protection against any other strain.

Between 2004 and 2006, MeNZB was given to anyone under the age of 20. Babies and preschoolers were routinely immunized until 2008. People with a high medical risk continued to get the vaccine until 2011. Once the epidemic was over, the vaccination program was stopped.

However, scientists noticed that the meningitis vaccine also seemed to offer some protection against gonorrhea. A study published in the Lancet last month showed that one-third of the people who had received MeNZB did not get gonorrhea, compared to a control group who was not inoculated. The lead author noted that the bacteria causing both diseases share between 80 and 90 percent of their primary genetic sequences.

Dr. Steven Black, an infectious disease expert at Cincinnati Children’s Hospital, noted, “This is the first time it’s been shown that you could have a vaccine that would protect against gonorrhea. And if these results are confirmed in another setting, that would mean that it would be very reasonable … to go forward with developing perhaps a more targeted vaccine.” Black’s comments were published in the current issue of JAMA, the Journal of the American Medical Association.  The JAMA article concludes that ultimately, a preventive vaccine could be the only sustainable solution to a fast-changing bug that has proven adept at developing resistance.

The World Health Organization reports that gonorrhea is becoming harder, and sometimes impossible, to treat, warning that it could become incurable in the not-too-distant future. At the moment, there no new antibiotics being developed to treat this disease.

The U.S. CDC reports that gonorrhea is the second most commonly reported notifiable disease in the United States. All known cases must be reported to the CDC, but officials there estimate that they are notified of fewer than half of the 800,000 new cases each year.

Displayed with permission from Voice of America via RePubHub FREE LIC; Image courtesy of FREEPIK by marioluengo CC0 License

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First FDA Approval for Sickle Cell Treatment 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.  The FDA granted the approval of Endari to Emmaus Medical Inc.

“Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” said Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent, or “sickle,” shape). This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. According to the National Institutes of Health, approximately 100,000 people in the United States have sickle cell disease. The disease occurs most often in African-Americans, Latinos and other minority groups. The average life expectancy for patients with sickle cell disease in the United States is approximately 40 to 60 years.

The safety and efficacy of Endari were studied in a randomized trial of patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days).  Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).

Endari received Orphan Drug designation for this use, which provides incentives to assist and encourage the development of drugs for rare diseases.  In addition, development of this drug was in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.

Displayed with permission from PRNewswire for Journalists; Illustration Courtesy of NIH

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Clinical Trials Monthly Case Study Analysis: Cost Reductions / Hematology

Each month, we investigate a different Clinical Trial Case Study that posed us with a challenging question – this time it’s a look at an orphan indication in HEMATOLOGY, and significant cost reductions for the sponsor:

We asked ourselves:

Scenario: A Phase IV study was required by the FDA as a condition of approval of an orphan drug. The sponsor was faced with monitoring, managing, and locking data for a 12-month study conducted at 100 sites.

•    Criterium offered an integrated technology-enhanced solution that allowed the sites to register ePRO Patient Diaries Input-Output Diagrampatients and allowed the patients to record their event data directly into our remote systems.
•    Criterium’s solution enabled the client to eliminate most of the scheduled monitoring visits. Criterium’s diligence and centralized system meant that monitors only had to visit sites that had problems.

What If:  If the client had used traditional methods, the cost would have soared for a product that has modest revenue and it would have taken much longer to provide the data to the FDA and the prescribing community.

Results:  Criterium’s centralized real-time data accession utilizing Automated CRFs and Interactive Voice Response (IVR) medical history and patient diary application saved the client $1 million to $1.5 million in clinical monitoring and data management costs. In addition, the data were satisfactory for rapid submission to the FDA for conditional approval, were provided to the medical community to support the indication for which the drug was approved and numerous publications resulted from this fairly large body of data for this orphan indication.

Read more of our successful case studies: http://www.criteriuminc.com/case_studies.php

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