Mesenchymal stem cells (MSCs) are multi-functional stem cells that are present in multiple human tissues and can be found in the spinal cord, umbilical cord blood, umbilical cord tissue, placenta tissue, adipose tissue, etc.. With low immunogenicity, multi-directional diferentiation ability, in particular homing ability, MSCs have signifcant research potential in cardiovascular diseases, nervous diseases, and
Treatment with mesenchymal stem cells (MSCs) has failed to significantly reduce inflammation in the brain of adults with multiple sclerosis (MS) in a phase 2 clinical trial. The into-the-vein treatment also failed to improve other clinical aspects of the condition. The results were shared in a study titled “Safety, tolerability, and activity of mesenchymal stem cells versus placebo
The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Bespoke
After numerous setbacks at the turn of the century, gene therapy is treating diseases ranging from neuromuscular disorders to cancer to blindness. The success is often qualified, however. Some of these therapies have proved effective at alleviating disease but come with a high price tag and other accessibility issues: Even when people know that a
The new therapy was generally well-tolerated and prevented key signs of the condition in animal models. Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities. Angelman
This review aims to outline the particular approaches in the different published clinical trials for cell-based therapies for retinal diseases. The retinal pigment epithelium (RPE) is implicated in the pathophysiology of many retinal degenerative diseases. This cell layer is also an ideal target for cell-based therapies. Several early phase clinical trials evaluatingcell therapy approaches for
Is there enough evidence to show Biogen’s new Alzheimer’s drug Aduhelm provides a benefit to patients, physicians and society beyond the current standard care? The answer from a group of ICER experts, perhaps unsurprisingly, was “no.” During a daylong meeting Thursday that featured debate over the drug’s cost and more, experts voted 15 to 0 that
EMA’s safety committee, PRAC, has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known as acute myeloid leukemia (AML). The European Medicines Agency’s (EMA’s) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known
Sickle cell disease is the most common deadly genetic disorder. It results from a mutation that causes normally pliable red blood cells to become brittle and sickle shaped. This leads to pain, organ damage and early death. A team of scientists led by Mitchell J. Weiss, MD, PhD, and Jonathan Yen, PhD, at St. Jude, and David Liu,
St. Jude Children’s Research Hospital provides the first gene therapy manufacturing and distribution cost report for hemophilia B. A St. Jude Children’s Research Hospital analysis found a major gap between the cost to manufacture and distribute hemophilia B gene therapy and the $2 million-plus price reportedly under consideration for hemophilia gene therapy now in development. The study appeared
