September 22, 2023

Advancements in clinical trials can pave the way to innovative treatment

Clinical trials and research aim to study new tests and treatments and evaluate the outcome of a study on human health. Innovative treatment is a therapy either newly introduced or modified and common in medical science.

The importance of clinical trials are as follows:

  1. To discover new treatments for diseases
  2. To provide a scientific basis to advise patients
  3. To detect new ways of diagnosis
  4. To evaluate new therapies, devices, and safety of existing therapies
  5. To provide information on the cost-effectiveness of treatment.

Current research and clinical trials provided access to new approaches.

Chronic dry eye disease

Dry eye occurs when eyes cannot produce enough tears as the tears evaporate too quickly. Sometimes, dry eyes can become a chronic condition. Burning, itching, redness, blurry vision, etc. are some common symptoms of chronic dry eye disease. Lubricating eye drops can reduce chronic dryness.

OK-101 to treat dry eye disease:

Dry eye disease is a multifactorial disease, which causes discomfort in the eyes and instability of the tear film and in turn may damage the ocular surface. It can become chronic especially in the older population. With the use of digital screens and the age of individuals, the prevalence of dry eye disease may increase. Despite the development of current drugs, dry eye diseases still remains a significant medical need and the leading cause of patients’ visit to ophthalmologists. OK-101 is OKYO Pharma Limited’s, a biotechnology company, lead pre-clinical compound. It is a long-acting drug. It is found to be a GPCR-based anti-inflammatory drug. OK-101 can treat patients with dry eye disease. “Methods of Lowering Blink Reflex for the Treatment of Dry Eye Disease” as US Patent No. 11,197,906 is issued by the United States Patent and Trademark Office. In dry eye disease patients in Q4, 2022 in phase 2 clinical trial (open human studies), the drug is planned to be administered topically. OKYO Pharma believes that OK-101 can be effective in treating dry eye disease, which is currently not well treated by approved drugs present now. The ChemR23 G-protein coupled receptor is found on immunological cells of the eye and produces an inflammatory response. OK-101 acts as an antagonist of the ChemR23 G-protein coupled receptor. A membrane-anchored-peptide (MAP) Technology was used to develop OK-101. OK-101 can be beneficial to treat dry eye disease.

Hemophilia disease

Hemophilia is a genetic bleeding disorder. The blood does not clot in hemophilia patients in the typical way. This is because blood lacks enough blood-clotting proteins called “clotting factors”. Patients suffering from this condition experience prolonged bleeding. Replacing the missing blood clotting factor is the best way to treat hemophilia. The missing protein is FVIII (8), hence the focus is to replace the missing protein so that a person can form a clot.

Fitusiran in the treatment of hemophilia

Fitusiran can cut bleeding by 90% compared to currently used standard treatment options, revealed in a late-stage clinical trial. Fitusiran is an RNA-modulating drug. For both hemophilia A and hemophilia B patients who have not developed inhibitors (a type of immune system response, which decreases the effectiveness of currently used standard treatments), fitusiran can be used. It was found that 40 of the 79 patients given fitusiran did not have bleeds and there were no blood clots found in patients given fitusiran. Patients given fitusiran also had an increase in liver enzymes may be by targeting a protein called anti-thrombin produced in the liver. However, none of the patients discontinued treatment and elevations were modest. A subcutaneous shot given monthly once might be helpful to patients, who in the case of factor replacement therapies, currently must receive intravenous infusions multiple times a week. By frequent infusions of proteins missing from the bloodstreams of hemophilia patients, Fitusiran can help patients with hemophilia to reduce their dependency on factor replacement therapies.

Alzheimer’s disease

Alzheimer’s disease is a disorder of neuron. This neurologic disorder slowly robs memory and other cognitive abilities. The brain shrinks and the brain cells die. Currently, there is no cure; however, medication and management strategies may temporarily improve symptoms. It can be chronic or continue for whole life.

Senescent cells in the treatment of Alzheimer’s disease

Scientists found that in human brains, toxic senescent cells can be used as a target for new Alzheimer’s disease treatment. The sick and old senescent cells that cannot repair themselves and do not die function abnormally and release toxic substances that can kill the nearby healthy cells, causing inflammation. These cells can build up in tissues in the whole body, leading to aging processes, memory loss and cognitive decline, and cancer. When scientists analyzed 10,000 cells from the postmortem brains of people who died of Alzheimer’s, approximately 2% of the brain cells were found to be senescent. Scientists also found that senescent cells were neurons, the basic unit of the brain, that process information and store memory. These cells are the primary ones that are found to be lost in Alzheimer’s disease. Also, the scientists found that senescent neurons had tangles; tangles are abnormal accumulations of tau protein that can collect inside neurons in Alzheimer’s disease patients. The tangles overlapped in such a way that hardly it was possible to differentiate between them. The more the tangles, the high the severity of Alzheimer’s disease. The effects of clearing senescent cells in older adults with mild cognitive impairment or early-stage Alzheimer’s were tested by administering a repurposed drug approved by U.S. Food and Drug Administration, which was designed to clear cancer cells in combination with a plant-derived antioxidant, a flavonoid. The therapy was found to work well in mouse models with Alzheimer’s and also has been proved to be safe in humans with other conditions. The drug can act against several biological processes that contribute to Alzheimer’s disease; for e.g., buildup of toxic senescent cells.

Innovative research and clinical trials stand out as an exciting new way for potential treatments. Medical research industries have taken an important step toward creating a treatment that can protect the lives of people.

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