This week, new research is presented in treatments for sickle cell disease, Alzheimer’s, asthma, and type 1 diabetes. Here’s a look at that and more.
This week, new research was presented in treatments for sickle cell disease, Alzheimer’s, asthma and Type 1 Diabetes. Here’s a look at that and more.
Fetal Hemoglobin and Sickle Cell Treatment
Study summary:
A new study finds a potential way to treat blood disorders by increasing a specific type of red blood cell (RBC).
Findings:
This study, conducted by researchers at St. Jude Children’s Research Hospital, found a link between the protein that adapts the body to hypoxia, or low oxygen, and the expression of a special kind of red blood cell in adults.
Background:
RBCs change throughout a person’s life, going from fetal red blood cells, or fetal hemoglobin (HbF), to adult red blood cells, or adult hemoglobin (HbA). The difference between the two types of cells lies in the mutations they can have. For example, it is a mutation of beta-globin, which is the hemoglobin linked to adults, that causes sickle-cell disease. This mutation does not, however, occur with gamma-globin, the hemoglobin associated with HbF.
The idea is to create an influx of HbF in the blood, leading to fewer cells that can have these mutations, effectively treating or getting rid of the disease. The researchers were able, using HIF1 (hypoxia-inducible factor 1), to promote production of HbF.
Relevance:
The researchers, led by Ruopeng Feng, Ph.D., have concluded that, while there needs to be further research into the use of HIF1 to treat blood diseases, they are hopeful that it could be useful in the treatment of sickle cell disease.
NeuroRestore ACD856 from AlzeCure
Summary:
New preclinical data released by AlzeCure on NeuroRestore ACD856 shows potential Alzheimer’s Disease (AD) modifying results.
Findings:
The researchers found the drug to have a neuroprotective effect when tested in a cell model of nerve injury. The hypothesized reasoning is that the drug causes improved mitochondrial function as well as better cellular protection on a structural level.
Background:
ACD856 works by stimulating two specific pathways of the brain, leading to the activation of Nerve Growth Factor (NGF) and BDNF (brain derived neurotrophic factor), which are both growth factors that lead to the growth of neurons and damage repair. Both growth factors have been linked to AD in that they are necessary for proper nerve function, but with the decline in cognition and brain function overall experienced in AD, it is thought this could be due to a decline in NGF and BDNF.
Relevance:
ACD856 has demonstrated the ability to improve cognition and memory in the preclinical trials and is set to move on to phase II trials, where it can be further tested as a treatment for AD.
Treating Allergic Asthma without Weakening Defenses for Flu
Summary:
Researchers have found a way to suppress the most common type of asthma without compromising defenses against the flu virus, which has previously not been done.
Findings:
In a new study led by researchers from NYU Grossman School of Medicine, data suggest that by removing a gene that is responsible for a specific calcium channel, asthmatic inflammation is reduced. The drug they tested, called a CRAC channel inhibitor, had a similar effect as the genetic manipulation.
Background:
In previous research, manipulation of the calcium channels prevented T-cells from being able to fight effectively against viruses like the flu, as the channels are the way these cells communicate. In this study, led by Stefan Feske, MD, the Jeffrey Bergstein Professor of Medicine in the Department of Pathology at NYU Langone Health, the research team was able to find a way to systematically deliver the drug, so that the rest of the body remained unaffected and immunologically capable.
Relevance:
The drug, and use of CRAC pharmacological, in general, need further research, but these results are promising in the treatment of asthma while not putting those patients into immunocompromised positions for diseases, like the flu, that may pose a greater threat to them anyway.
New Therapeutics for Type 1 Diabetes Possible
Summary:
Researchers have developed antigen-specific engineered regulatory T-cells that show potential as a targeted Type 1 Diabetes (T1D) prevention therapy.
Background:
Transfer of regulatory T-cells has shown to be an effective therapy in mouse models of T1D. In humans, however, the frequency of these antigen-specific regulatory T-cells occurring naturally is low, making this a more challenging option in humans.
Findings:
To combat the low natural occurrence of the specific regulatory T-cells needed, the researchers made their own. These engineered regulatory T-cells suppress effector T-cells, which is often a trigger for T1D in the pancreas. By engineering these cells, the researchers created a cell that worked to prevent T1D in mice by blocking the effector T-cells in the mice’s pancreas.
Significance:
The cells need further testing but show promise as a potential therapy in the prevention of T1D. The next step for the research team is to develop these cells into a usable treatment and begin preclinical trials.
