Genetically modified T-cells to treat resistant leukemia


Leukemia is a form of cancer which affects the blood. Acute lymphoblastic leukemia is the most common form of childhood leukemia and occurs when there is uncontrolled proliferation of abnormal white blood cells. White blood cells form the ‘infection fighting’ part of the blood, protecting us from foreign or abnormal substances. The huge production of abnormal white blood cells, prevents the body producing normal white cells, red cells and platelets. Symptoms then result from anaemia, impaired blood clotting and susceptibility to infection caused by the lack of healthy blood components.

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Breakthrough success has been reported by a study team from the University College London Institute of Neurology in early diagnosis of Huntington’s disease. The team has developed a blood test that can accurately detect the earliest biomarkers of Huntington’s disease. If these results are validated in large scale clinical trials, broad avenues of hope would open to those at high risk of Huntington’s disease as timely diagnosis is half the battle won. This revolutionary study was published on September 13, 2018, in the journal Science Translational Medicine. This simple blood test is capable of detecting the earliest signs of Huntington’s disease, performing better than brain scans at the diagnostic ability. Early detection holds substantial value in the development of effective therapeutic strategies to fight disease progression.

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Food and Drug Administration (FDA) has granted Orphan Drug designation to A4250 for the treatment of Alagille syndrome, a life-threatening and rare disease that primarily targets the liver. This genetic disorder affects approximately 1 in 70,000 newborns and is characterized by liver damage due to bile stasis (cholestasis) with signs and symptoms including jaundice, poor weight gain, loss of appetite, poor growth, and pruritus. Alagille (AGS) syndrome is variably characterized by cholestasis, peripheral pulmonary artery stenosis, characteristic facies, anterior segment abnormalities, pigmentary retinopathy, and dysplastic kidneys.

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Multiple Sclerosis and Sleep Disorders


Multiple sclerosis is an autoimmune inflammatory disease of the central nervous system. Around 400,000 people in the US are diagnosed with MS [1]; it is commonly diagnosed in early adulthood and is twice as common in women as men [2]. In MS, inflammation damages the protective coat around nerves (the myelin sheath), which reduces the efficiency of message transmission down those nerves.

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An intricate case of a family with 2 children with a rare skeletal disorder has been reported in October 2018. The disorder has not been reported before, and clinically, it appears similar to Jansen metaphyseal chondrodysplasia (JMC). JMC is an exceedingly rare disorder causing abnormal development of portions of the arm and leg bones. Unusual cartilage formations and subsequent abnormal formation of the long bones results in short arms and legs. Short-limbed dwarfism is a cardinal sign which becomes apparent during childhood.

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On 9 October 2018, U.S. Food and Drug Administration granted 'Orphan Drug' Designation for AG10, a product of Eidos Therapeutics, Inc. (Eidos), for the treatment of Amyloid Transthyretin amyloidosis (ATTR). Jonathan Fox, M.D., Ph.D., president and chief medical officer of Eidos, expressed his deep-dwelled belief that AG10 holds great clinical promise as a disease-modifying drug for these patients.

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In September 2018, the U.S. Food and Drug Administration granted orphan drug designation to Q-Cells for the treatment of transverse myelitis, a rare neurological condition in which the spinal cord is inflamed. Steven Borst, MBA, president and CEO of Q Therapeutics Inc., applauded the move and considered it to be a stellar step toward their goal of advancing glial cell therapy for different types of neurodegenerative diseases.

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Microbiome, the Hidden Treasure of Heath Information


Health is the most treasured commodity ad infinitum. Evolutionary view of human health and disease is inevitable in the face of evidence and time. It is no more a hidden knowledge that out of about 23,000 human gene population, around 37% have homologs in bacteria and 28% originated in unicellular organisms. We are evolved versions of our earlier, pre-evolution existence, which was a unicellular form of life if traced to the origin.

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Male Menopause and Testosterone Crisis


When trying to define roles and personalities of women, Freud wrote that 'Biology is destiny'. A believer in the non-negotiating power of the hormones, Freud perfectly summed up the rationale behind all the wars and fracas that have sabotaged ill-privileged nations and communities and people. Testosterone is the hormone that gives men their masculinity. All the mighty kings and warriors had ample of testosterone in their blood pool to make them dynamic, aggressive and controlling.

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T-Cell Lymphoma: Stories of hope and courage


The tyranny of cancers has slaughtered more minds than bodies. Even the chance mention of a diagnostic possibility clouds the consciousness in a jiffy, demolishing the kernel of life. The mutated molecules of cancer have militated against the body in the most vilifying and varied manners, earning it the reputation of the meanest man-eater disease. The earliest known description of cancer dates back to 3000-28 BC in an ancient Egyptian textbook, Edwin Smith Papyrus, and the document coldly stated of the disease: "There is no treatment1". Even Dinosaurs suffered from cancers and their tumors resembled those of human patients, proving the perpetual existence of cancer. It would not be a hyperbole to state that cancer in humans is as old as the mankind or older even.

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