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Viral History from a Single Drop of Blood, NCI Launches Gene Therapy Trial, FDA Approves Eltrombopag for Pediatric ITP, and more

December 30, 2021

Going Viral: A New Test Detects Lifetime Virus Exposure with a Single Drop of Blood

VirScan, a recently developed, high-throughput blood test, can detect nearly every virus a person has been exposed to in his or her lifetime, according to a study reported in Science. The test may be useful for tracking disease patterns across age groups and geographic populations, as well as studying complex, chronic diseases, such as cancer, diabetes, and autoimmune diseases.

"In addition to directly causing acute or chronic illness, viral infections can alter host immunity in more subtle ways, leaving an indelible footprint on the immune system," the authors wrote. "VirScan may prove to be an important tool for uncovering the effect of host-virome interactions on human health and disease and could easily be expanded to include new viruses as they are discovered, as well as other human pathogens."

Current serologic methods of viral detection are limited to testing one pathogen at a time and are used primarily to address specific clinical hypotheses. VirScan, however, allows for a comprehensive analysis of antiviral antibodies in human sera using less than 1 μl of blood.

The researchers tested VirScan on 569 individuals living in the United States, South Africa, Thailand, and Peru, assaying antibody-peptide interactions for reactivity to 206 human viral species and more than 1,000 strains. Most study participants were exposed to approximately 10 species of virus, with two participants being exposed to as many as 84 viruses. In general, those living outside of the United States were exposed to more viruses.

In terms of performance, the new blood test's ability to detect known infections and distinguish between exposures to related viruses was comparable to that of classical serum antibody tests for single viruses. The researchers noted, however, that the test can only detect identified viruses and may overlook very small viruses or past infections that occurred when the immune response was not as robust.

The test, which can be performed for $25, currently takes two days to two months to complete, though the developers are looking to streamline the test's processes and produce results in as few as two to three days.

Source: Xu GJ, Kula T, Xu Q, et al. Comprehensive serological profiling of human populations using a synthetic human virome. Science. 2015 June 5. [Epub ahead of print]

NCI Launches MATCH Trial to Link Gene Mutations with Appropriate Cancer Treatments

The National Cancer Institute (NCI) announced the launch of a phase II, nationwide trial that will determine links between cancer treatment options and gene mutations. The trial, called NCI-MATCH (Molecular Analysis for Therapy Choice), began patient enrollment in July, screening individuals from 2,400 U.S. sites.

"NCI-MATCH is a unique, ground-breaking trial," said Doug Lowy, MD, the acting director at NCI, in a press release. Because many gene mutations in tumors are infrequent or unique, screening for individual mutations is not cost-effective or efficient in clinical trials. Instead, NCI-MATCH will use advanced gene sequencing techniques to screen for many molecular abnormalities at once.

"It is the first study in oncology that incorporates all of the tenets of precision medicine," Dr. Lowy said, "and holds the potential to transform cancer care."

The study was co-developed by the NCI, part of the National Institutes of Health, and the ECOG-ACRIN Cancer Research Group, part of the NCI-sponsored National Clinical Trials Network (NCTN).

NCI-MATCH will begin with a DNA analysis of tumor biopsies from approximately 3,000 patients. The samples will undergo DNA sequencing to detect genetic abnormalities that may be driving cancer growth and could be targeted by one of the drugs in the study. Patients will then be treated with the targeted regimen for as long as their tumor shrinks or remains stable. Patients are expected to have one or at most two treatable mutations in the tumors. Investigators hope to enroll about 1,000 patients in the various treatment arms. The study drugs that will be used in the trial are either already approved by the U.S. Food and Drug Administration (FDA) or are currently under development in clinical trials.

The study will include additional small arms for each treatment being investigated, starting with approximately 10 arms and potentially expanding to 20 or more. Patients will be assigned to treatment options based on their individual gene mutations – not the location of the cancer. Each arm will enroll up to 35 patients.

To be included in the trial, participants must be 18 years of age or older with solid tumors or lymphomas that have advanced following at least one line or standard systemic therapy, or those whose tumors have no standard treatment available.

The study's primary endpoint is the overall response rate, with a secondary endpoint of six-month progression-free survival.

To be deemed successful, the researchers are looking for a tumor reduction of at least 16 percent; response rates less than 5 percent will be deemed unsuccessful.

Source: National Cancer Institute press release

New Approach Uses Patients' Own Bone Marrow T-Cells to Treat Multiple Myeloma

According to results of a new proof-of-principle study recently reported in Science Translational Medicine, a patient's bone marrow T cells may provide a new form of treatment for multiple myeloma (MM).

"Successful adoptive T-cell therapy (ACT) requires the ability to activate tumor-specific T cells with the ability to traffic to the tumor site and effectively kill their target as well as persist over time," the authors wrote. They hypothesized that ACT using marrow-infiltrating lymphocytes (MILs) could impart greater antitumor immunity because they were obtained from the tumor microenvironment.

To test this hypothesis, the investigators selected 25 patients with newly diagnosed or relapsed forms of MM to receive ACT, which used MILs that were harvested, activated, and expanded, then infused on the third day following myeloablative therapy.

Patients were followed for a median of 6.4 years. At one year post-MILs therapy and chemotherapy, 54 percent (n=13) of the 22 evaluable patients had at least a partial response to therapy:

  • 27% had a complete response
  • 27% had a partial response
  • 23% had stable disease
  • 14% had progressive disease

For patients who achieved at least a 90-percent reduction in disease burden, progression-free survival was significantly longer than for patients who did not achieve a high reduction in disease burden (25.1 months vs. 11.8 months; p=0.01).

Although this is an early proof-of-principle study, the researchers hope that this ACT MILs method could be used in other hematologic malignancies infiltrating the bone marrow.

Source: Noonan KA, Huff CA, Davis J, et al. Adoptive transfer of activated marrow-infiltrating lymphocytes induces measurable antitumor immunity in the bone marrow in multiple myeloma. Sci Transl Med. 2015;7:288ra78.

Updated ABIM Score Reporting for MOC Examination

The American Board of Internal Medicine (ABIM) is updating the score reporting for its Maintenance of Certification (MOC) examinations. The changes are a result of input from the Internal Medicine community, according to the ABIM, and will provide more information on physician performance when delivering MOC scores.

Beginning with the spring 2015 MOC examinations, physicians will receive score reports in a new electronic format that includes a visual presentation of the results, detailed feedback on incorrect questions, and links to additional information.

"As the practice of medicine changes, ABIM must work closely with the physician community to continuously ensure that our exams reflect foundational knowledge that doctors in practice today need to serve their patients," said Nick Fitterman, MD, chair of the ABIM Internal Medicine Board Exam Committee, in a press release.

The ABIM has come under fire recently for its MOC requirements, and for not providing usable feedback to exam takers.

Source: American Board of Internal Medicine press release

FDA Approves Eltrombopag for Pediatric Chronic Immune Thrombocytopenia

The U.S. FDA has approved eltrombopag for children who are 6 years or older with chronic immune thrombocytopenia (ITP) who have not responded to treatment with corticosteroids, immunoglobulins, or splenectomy. The drug was previously approved by the U.S. FDA in 2008 for the treatment of ITP in adults.

Eltrombopag is a once-daily oral thrombopoietin receptor agonist that increases platelet production.

The recent approval for pediatric ITP was based on data from two double-blind, controlled trials, including the largest phase III trial in the pediatric ITP patient population. In each of these studies, eltrombopag was shown to significantly increase and sustain platelet counts, while also allowing for the safe discontinuation of some concomitant medications (including corticosteroids).

The most commonly reported adverse events associated with eltrombopag included upper respiratory tract infections, nasopharyngitis, rhinitis, abdominal pain, cough, inflammation of the throat or mouth, toothache, abnormal liver function tests, diarrhea, rash, and vitamin D deficiency.

Source: Novartis press release

AG-120 Receives Orphan Drug Designation for the Treatment of AML

The U.S. FDA granted orphan drug designation to AG-120 for the treatment of acute myeloid leukemia (AML). AG-120 is an oral, first-in-class IDH1 mutant inhibitor, which is currently being evaluated in a phase I clinical trial in patients with IDH1-mutant positive AML.

Preliminary reports from the ongoing phase I study found that AG-120 demonstrated clinical activity and a favorable safety profile in 57 patients. The overall treatment response rate was 31 percent and the complete remission rate was 15 percent. Patients who have responded to treatment have remained on AG-120 for up to 11 months, with 79 percent of responders receiving treatment for at least three months.

The most commonly reported adverse events associated with AG-120 in this trial included fatigue, diarrhea, pyrexia, and nausea. So far, 13 deaths have been reported among study participants, though the researchers did not attribute any mortalities as related to treatment.

An additional three expansion cohorts consisting of 175 AML patients are expected to be studied.

Source: Agios Pharmaceuticals news release

APTO-253 Receives FDA's Orphan Designation for the Treatment of AML

The U.S. FDA has granted orphan drug designation for APTO-253 for the treatment of patients with acute myeloid leukemia (AML). The drug is a first-in-class inducer of the Krüppel-like factor 4 (KLF4) gene and is currently being evaluated in a phase Ib trial in patients with AML, high-risk myelodysplastic syndromes, and other hematologic malignancies.

Epigenetic suppression of the KLF4 gene acts as a key transforming event in AML and, through its unique mechanism of action, APTO-253 exhibited strong activity to kill AML cells as both a single-agent and in combination with other agents, according to preclinical studies. Those reports also showed that APTO-253 had a favorable safety profile with no evidence of suppression of the normal bone marrow.

Source: Aptose Biosciences press release

FDA Introduces New Simplified REMS Tracking

On June 17, 2015, the U.S. FDA rolled out an updated website for its Risk Evaluation and Mitigation Strategies (REMS). The website aims to make REMS more accessible and the process of finding an REMS and determining when it had been updated to reflect new information easier for users.

Users can now find REMS guides containing a medication guide more simply, and the lists can be sorted by date of last update, as well as by brand name of the drug. Users can also download a complete list of REMS guides in addition to historical data via a structured data format.

Source: FDA news release

FDA Grants Orphan Drug Designation to New Hemophilia Drug

ATX-F8-117, a peptide therapy for the treatment of factor VIII intolerance, was recently granted the U.S. FDA's orphan drug designation for patients with hemophilia A who currently have inhibitors or who are at risk for developing factor VIII inhibitors. Currently, no drugs are approved to prevent or treat the development of inhibitors.

Hemophilia A patients are typically treated with factor VIII to help with blood clotting; however, because these patients' immune systems have had no or low exposure to factor VIII, they often do not tolerate the replacement factor VIII used to treat their condition. Consequently, up to 30 percent of these patients develop factor VIII inhibitor antibodies.

The development of these antibodies also significantly limits the treatment of this disorder, while also increasing the cost burden for hemophilia A patients. The two peptides in ATX-F8-117, derived from factor VIII, have the potential to treat and prevent inhibitor development in hemophilia A patients treated with factor VIII.

Source: Apitope press release


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