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November 16, 2020 // -- A new study from the University of Virginia School of Medicine suggests that a drug promising to treat multiple sclerosis could actually worsen the disease.
the drug has not yet been used in human trials, UVA scientists have warned its researchers to study it with extreme caution.
report that in addition to worsening the disease in mouse models, the drug has an unexpected off-target effect.
, an MS researcher at the UVA Department of Neuroscience and its Brain Immunology and Neuroglia Research Center (BIG), said: "This is not what we expected at all.
the message of this study is that we should be very careful and do more basic research before we recommend it for clinical trials.
( Photo: www.pixabay.com) Multiple sclerosis is a debilitating autoimmune disease that affects an estimated one million Americans.
the disease causes the body's immune system to destroy myelin, the insulation that surrounds and protects our nerve fibers.
this prevents nerves from transmitting signals to the brain.
can cause a variety of symptoms, including muscle spasms, fatigue, difficulty in movement, numbness and pain.
these symptoms may vary from patient to patient.
that existing MS drugs can have adverse side effects, such as weakening the body's ability to fight infection, doctors are eager to develop better alternatives.
promising candidate is a small molecule drug called TEPP-46.
TEPP-46 was originally developed to fight cancer, with the goal of so-called "metabolic adaptability" (changes in cell energy production) that occur in both cancer and MS.
, however, in Gaultier's MS model, TEPP-46 causes the disease to worsen, shifting inflammation from the spinal cord to the brain.
and his team determined that the drug causes harmful changes in immune cells called T-cells, although he and his team don't fully understand why.
also have unexpected "off-target" effects, which means that the drug affects cellular processes other than expected.
Gaultier noted that his findings were inconsistent with other studies, saying more research was needed before scientists could use the drug in clinical trials in MS patients.
benefit of this new study is that it suggests that TEPP-46 could be used to create better models of MS mice, helping scientists try to understand and treat the disease.
results were published recently in the journal Science Signaling.
() Source: Forward multiple sclerosis drug may worsen disease, research source: Scott M. Seki et al, Modulation of PKM activity affects the source of TH17 cells, Science Signaling (2020). DOI: 10.1126/scisignal.aay9217