5 biggest clinical trial developments of the past month
In the midst of the Covid-19 pandemic, the main focus lies in finding a cure and a vaccine to treat the virus. The potential discovery of a cure will, without a doubt, be one of the biggest scientific breakthroughs of the 21st century; and, as such, everyone has their eyes on the clinical trials taking place right now. COVID-19 has opened the eyes of many about how essential clinical studies are in fighting critical diseases. And not only this, without clinical research, new innovations cannot be discovered and tested for the medical sector, which could help millions of people living with underlying health conditions.
As the attention towards clinical trials has grown in the past couple of months, let’s take a look at the latest medical breakthroughs and developments outside of COVID-19; and consider what they mean for the future.
Alzheimer’s clinical trials
A team of scientists from the University of Alberta Distinguished University has found strings of amino acids (short peptides) that, when injected into mice with Alzheimer’s disease, significantly improved the mice’s memory and brain pathology in Alzheimer's disease were also greatly improved. Through the use of computer modeling and artificial intelligence, the team was able to discover a small-molecule drug, which is now awaiting human clinical trials. This discovery could mean significant improvements for Alzheimer’s patients and represents years of clinical research in the field.
Blood testing for Breast cancer
The University of Nottingham in England developed a blood test at the end of last year that could potentially detect early-stage breast cancer up to five years before there are any signs, such as lumps or other symptoms. The test looks at the ‘body’s immune response to substances produced by tumor cells’, according to new clinical research.
During the pilot study, blood samples from 90 breast cancer patients were conducted by the time of their diagnosis and matched with samples from patients without breast cancer. The 180 control samples were then examined for the presence of autoantibodies against cancer cells associated with breast cancer. Researcher Danizah Alfattani (PhD) mentioned during the study’s report that “the results are encouraging and indicate that it’s possible to detect a signal for early breast cancer” in the future. However, he also stressed that the test needed to be developed and validated more. Research sites are currently testing samples from 800 patients against a panel of 9 tumor-associated antigens (TAAs) in order to improve the accuracy of the test.
New drug to improve cystic fibrosis
In October of last year, the FDA approved the new drug Trikafta, which is the first triple combination therapy available to show significant improvements for patients with cystic fibrosis mutation. Cystic fibrosis is a rare, progressive and life-threatening disease where patients experience thick mucus build-up in the lungs, the digestive tract, and other parts of the body. The disease can lead to severe respiratory issues and can lead to other complications such as infections. Because of the severity of the disease, the fast-track approval of the FDA marks a big breakthrough for complex diseases such as cystic fibrosis “making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to additional effective therapy,“ said FDA Commissioner Ned Sharpless, M.D.
Promising developments with CRISPR gene editing
CRISPR is a gene-editing technology that allows scientists to modify genes and DNA. In a recent cancer study, CRISRP was proven to be well-tolerated by patients living with multiple myeloma and sarcoma. Researchers of the University of Pennsylvania in Philadelphia were able to collect immune cells (T cells) from cancer patients, giving them the ability to recognize a protein produced by some cancer cells through conventional genetic-engineering. CRISPR–Cas9 was used to block the T cells’ production of three proteins that might inhibit the cells’ ability to target tumors.
Gene therapy for sickle cell disease
In October, news broke that researchers of the National Institutes of Health (NIH) have developed a vector, a virus-based vehicle to deliver therapeutic genes that can be used in gene therapy to fight sickle cell disease. The vector has proven to be 10 times as efficient as currently used vectors in incorporating corrective genes into bone marrow stem cells. Sickle cell disease is a painful and life-threatening blood disorder, which affects around 100, 000 people in the United States. The successful development of the vector could improve the effectiveness of gene therapy and help millions of people worldwide.
With clinical trials standing their grounds and continuously working towards cures and new treatments for complex diseases, it is important to recognize the hard work and medical breakthroughs resulting from years of research that is making a difference for millions of people.
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