Showing posts with label clinical trials. Show all posts
Showing posts with label clinical trials. Show all posts

Tuesday, November 26, 2024

The Fight Against Disease: A Strike Against Malaria

Malaria is an Epidemic

Malaria has been a growing problem in many third world countries, with mosquitos infecting an estimated 682,191 people a day in 2022. That is an increase of 5 million year over year from 2021. With the growing concern of how to stop this epidemic, researchers are looking at using a second-generation genetically attenuated parasite to create a "favorable immune induction profile and protective efficacy" response in humans infected with the virus. The trials show favorable results and it appears that the research is moving forward. This is great news, and to subdue any concerns, although the parasite does invade hepatocytes, it never reaches a blood stage infection due to the limitations placed on it via genetic modification. 


This is a great example of how genetic research can impact medicine and help save lives where other methods fall short. I'm excited to see where this goes, and if we can apply this to other parasite originated diseases. The next problem to work on in this case is a solution for those who have been previously infected, as this performs best in first infection cases, and sub optimally otherwise. Below is an important infographic relating to the study, easily digestible by those unfamiliar with the subject matter

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Links:

https://www.nejm.org/doi/full/10.1056/NEJMoa2313892

https://www.nature.com/articles/s41541-024-00975-0

Tuesday, April 24, 2018

Childhood Food Allergies Caused by a Combination of Genetic and Environmental Factors



Researches have found a link to child food allergies and genetic factors along with environmental factors. A specific combination of environmental factors and genetic factors can trigger infant and childhood food allergies. In order to trigger these allergies a child would have to have genetics that change the skin absorbency, have been exposed to infant wipes that leave soap on the skin, and have been exposed to allergens such as dust or food allergens. Joan Cook-Mills is a professor of allergy-immunology at Northwestern University Feinberg School of Medicine and was the lead researcher on this study. She explained ways to prevent these environmental factors, which when combine with the genetic factors can cause childhood allergies. She said to, “Reduce baby’s skin exposure to the food allergens by washing your hands before handling the baby. Limit use of infant wipes that leave soap on the skin. Rinse soap off with water like we used to do years ago.” The soap in the wipes disrupts the skin barrier. This link between environmental and genetic factors that causes food allergies was discovered through clinical evidence of food allergies in humans and tests were done using neonatal mice models. The clinical evidence showed 35% of the children that were looked at who were looked at with food allergies, also had dermatitis. Dermatitis is caused by three different gene mutations that reduce the skin barrier. This study is helping to understand food allergies and how they develop which is important to know for developing more responsive medicine. Cook-Mills’ goal is to discover interventions that will more effectively respond to signals and block the development of food allergies.


Tuesday, November 22, 2016

The Race for a Zika Vaccine

A vaccine for the Zika virus is much closer than anyone would anticipate. Scientists and researchers everywhere are working tirelessly to develop a vaccine from scratch that could stop Zika and its birth defects. Typically, vaccines take ten years to make, but a Zika vaccine could be available as soon as 2018, which would be a remarkable two years compared to the typical amount of time it takes to develop a vaccine. There are more than a dozen pharmaceutical and government companies working on this vaccine. Some companies are working on using DNA splicing, which has never before been approved for use in human vaccines. The motives of saving lives, along with the opportunity to make huge profits as the virus is in countries like Brazil and the US - places which can pay for public vaccination campaigns - are what is keeping this hunt for a vaccine going. Timing is crucial for the development of the vaccine. Clinical trials in South America will be done within the next few months as infection rates are expected to be high. However, if the window is missed there may be a year or longer delay as vaccines are preventative and thus will only perform as expected during a certain time frame after an outbreak.



Immunologist Dr. Fauci has led the National Institute of Allergy and Infectious Diseases for over 30 years and has played a role in every infectious outbreak since then. He is confident that a vaccine will be created for a number of reasons. The economic motivation is clear, being that the first team to create an effective vaccine will likely reap billions in profit from governments trying to eliminate the virus. Zika also belongs to a family of viruses called flaviviruses, which scientists having been fighting for years and years, and of which many vaccines already exist. Some teams went with the basic killed vaccine approach, the same one that is used for polio and the flu. It is reliable but can take decades to develop. Dr. Fauci and his team took a different approach which involves inserting a harmless part of the virus's DNA into humans which will then form Zika proteins in the body and lead the body to creating antibodies against the Zika virus in case it arrives. However, this technology has only been approved for animals, not humans. Clinical trials are starting throughout Central and South America, along with at a clinic in the University of Maryland School of Medicine. Dozens of people around the world have received potential vaccines for the virus thus far, so hopefully one will be successful. I believe that there is a very high amount of motivation around the world to form a successful vaccination for Zika, especially money. With all the new technology and techniques being tested by several different companies, at least one of them is bound to be effective very soon. This vaccine will be a huge breakthrough, considering how many people the Zika virus has already afflicted.

Wednesday, November 16, 2016

First Clinical Trial in Humans Using CRISPR Gene-Editing

In recent years, many treatments for cancer have involved using patients' own immune systems to fight cancer. A substantial part of these treatments have involved editing the genes of immune cells. Gene-editing has been very successful in fighting not only cancer, but other diseases as well. CRISPR-Cas9 is the most efficient and economical of all gene-editing tools currently being utilized. CRISPR-Cas9 contains a piece of RNA and an enzyme called Cas9, that together can "edit" or change DNA. The first clinical trial for humans, using the CRISPR gene-editing technique, was approved by a hospital ethics board in China this past summer. A team of scientists under the leadership of Lu You, an oncologist at Sichuan University, injected modified immune cells into a patient suffering from lung cancer. CRISPR-Cas9 was used to modify the immune cells of the patients blood, so that the cells attack the cancer by disabling a certain gene that codes for the protein PD-1.


These clinical studies are extremely beneficial because if successful, they could provide us with insight as to how well modified immune cells can attack different kinds of cancers, while using a gene-editing technique that is more simple and efficient than others. This article states that the first clinical trial in China could initiate a sort of "biomedical duel" between China and the United States, as they often compete with each other in the advancement of all types of technology. The prospect of this "biomedical duel" is exciting because this type of competition may facilitate quicker progress in cancer treatment research. In the United States the first CRISPR clinical trial is expected to start in the beginning of 2017, as well as more clinical trials in China. I think that this type of progression gives hope to many cancer patients and other people suffering from diseases that could benefit from gene modification treatments.


http://www.yourgenome.org/facts/what-is-crispr-cas9

http://www.nature.com/news/crispr-gene-editing-tested-in-a-person-for-the-first-time-1.20988?WT.mc_id=FBK_NA_1611_FHNEWSCRISPRTESTED_PORTFOLIO

Thursday, October 22, 2015

Making Cancer Patients Stronger: Tissue by Tissue

Many patients that have cancer begin to fade away as their cancer progresses. The malignant tumor(s) strengthen and eat at tissues bringing patients to their last breaths. The eating away of muscle and mass tissue is known as cachexia.  Cachexia affects 80% of cancer patients. Scientists, Amelia Johnson and Nick Hoogenraad set out to try and understand how a tumor becomes aggressive proposed by an idea from Harold Dvorak about tumors in 1986.  Dvorak proposed that tumors halt the body's healing responses and create a blood supply of it's own; growing and spreading rapidly.  Hoogenraad, in hopes of progressing with aggressiveness of tumors, came across a protein called Fn14. She came up with the idea of "turning off" this protein to see if growth of the tumor would continue. Unknowingly in her research, she had come across information that was relevant to tissue deterioration (cachexia).

Realizing this, Amelia Johnson and Nick Hoogenraad furthered this research and began a trial in mice.  Within this trial, cancer cells lacking the Fn14 protein were injected in some  mice and cancer cells with active Fn14 protein  were injected in others.  Observations concluded that the tumor in the mice lacking the protein had tumor growth, but no signs of emaciation or tissue loss.  The mice injected with the cancer cells with the Fn14 protein, had the same tumor growth and appeared to have tissue damage and was sickened within days.  What this meant for Johnson and Hoogenraad was that the Fn14 protein was the cause of the tissue destruction and cachexia.  With the suppressant of the protein, the cachexia was not evident and the mice had normal muscle and tissue mass.

The trial lasted for 27 days with the mice.  After the month-long experimentation and observations, conclusions were that the mice that had tumor growth with the Fn14 protein blocked were physically healthy, whereas the mice with the Fn14 protein not blocked became so sick that they had to be euthanized. The question remains if this same protein blocker could be used in humans that have diseases such as cancer, HIV, or TB. Antibodies containing the protein blocker are being made for human trials that undergo in a few years.  It is estimated that beyond these trials, if they are successful, doctors can incorporate this into treatment in about six to eight years.

I think if this is successful in human trials this will be a breakthrough in treatment for patients with cancer, HIV, or TB because it will be able to keep their bodies physically healthier to undergo more treatments.  Many people will not have to wither away as their tumors consume their bodies.  They can fight off the diseases and doctors can get a better understanding of the diseases as they progress into late and dangerous stages.

Monday, March 30, 2015

Possible Cure for Early Alzheimer's



Researchers at John Hopkins University found that a drug used to treat patients with epilepsy can actually calm down the brain of patients that have early signs of Alzheimer's. Recent research backs up previously published works by the John Hopkins' team that the drug has advantages. Animal studies have been done and a plan for long-term clinical trials is in place to see how the drug will affect human brains. 
Certain studies have shown that small amounts of atypical antiepileptic levetiracetam lowers the affects of Alzheimer's dementia. 84 people were studied and out of them 17 were healthy and the rest had pre-dementia. All were over the age of 55. In a double-blind trial some patients were given a placebo and some were given small doses of the drug. Low doses showed positive results in lessening memory loss. 

This article is so interesting because Alzheimer's affects many people. Finding a possible cure is amazing because then people would not have to suffer through the disease. Just knowing that grandparents and even parents would not lose their memory and not forget the ones they love is a great thing. With the way this research is going I think many positive outcomes will become available in the near future. 

Monday, October 13, 2014

New Gene Variants Identified for Prostate Cancer

Recently, an international team of scientists has identified a significant number of new genetic variants that are linked to an increased risk of getting prostate cancer. With these 23 newfound gene variants, the total number of common gene variants linked to prostate cancer is now at 100. Ros Eeles, a professor or oncogenetics at The Institute of Cancer Research in London, elaborated by announcing "We know that there are a few major genes that are rare and significantly affect prostate cancer risk, but what we are now learning is that there are many other common genetic variants that individually have only a small effect on risk, but collectively can be very important."


Now, a clinical trial to find out whether testing for genetic variants in men can successfully pick up the disease early and help direct targeted interventions for patients will shortly be conducted. After studying over 87,000 men of European, African, Japanese and Latino heritage, the team of scientists found 16 new genetic indicators associated with prostate cancer risk in European men. Additionally, they identified 7 genetic indicators in men of mixed heritage. To this end, the researchers feel that they can now explain 33 percent of the inherited origins in European men.
http://www.secretsofthefed.com/wp-content/uploads/2013/05/prostate-cancer-symptoms.jpg 

Looking ahead, the researchers plan to investigate if these findings can improve on prostate cancer screening test, particularly if genetic testing can help diagnose more men at risk for aggressive forms of the cancer. Dr. Matthew Hobbs, deputy director of research at Prostate Cancer UK said in a news release “The results of this study could take us a step closer to targeted screening by allowing us to identify those most at risk of the disease based on the genes that they possess.”

Identifying these new genetic variants is a huge step forward in finding a treatment or even a cure for prostate cancer. Hopefully, with this newfound more researchers and scientists will get involved and use the information to further their knowledge of this cancer. Overall, it is great to see that we are advancing in areas that can allow people with this cancer to identify it earlier on and try to treat it before it becomes too aggressive.

Article Link: http://health.usnews.com/health-news/articles/2014/09/15/new-gene-variants-for-prostate-cancer-identified



Wednesday, November 27, 2013

Genetic Tests do not appear to Improve Control of Blood Thinners

When administering the blood thinner warfarin, patients need to be administered the proper dose. If too much is given there is a risk of internal bleeding and on the other hand too little and there is a risk of developing blood clots. There is a delicate balance when using blood thinners therefore patients are monitored closely when taking them. A genetic test is done to test two genes. One of the genes influences how the liver metabolizes warfarin, and the other is about how the body responds to the blood thinner. A recent study, done by the Clarification of Optimal Anticoagulation through Genetics, looked at patients on warfarin from 2009-2013. This study was done dividing patients into two groups. The first group’s warfarin dosage was determined by clinical information such as weight, age and smoking status. The second group was given a dosage based on genetic testing and this clinical information. They found that genetic testing played no role in determining proper dosage. Furthermore it was found that in African Americans in the study had conditions worsen six months after original diagnosis when their dosage was determined by genetic testing. Previously, no clinical trials were conducted before having this genetic test widely used. This new finding has doctors bringing up the question “Should genetic tests be required before clinical use?” 


Links:
http://www.medicalnewstoday.com/articles/269141.php

Related Links:
http://www.technologyreview.com/view/521861/genetic-testing-falls-flat-in-large-patient-drug-trials/

http://news.uic.edu/genetic-test-will-help-dose-blood-thinner

Tuesday, November 26, 2013

200 Genes Linked to Chron's Disease

More than two hundred gene locations have now been identified for the chronic bowel condition Crohn's Disease, in a study that analyzed the entire human genome.This study shows how studying smaller but better defined groups can lead to a better understanding of how complex diseases are inherited, and paving the way for personalized treatment.

 
Crohn's Disease, a type of Inflammatory Bowel Disease, is a chronic illness of complex origins affecting approximately 100 to 150 people per 100,000. Understanding the genetic component of such complex diseases is central to explaining patients' symptoms and improving treatment.
Despite Crohn's having a large genetic component, this has been hard to dissect. This is partly due to the large number of genes involved, their complex interactions with environment and the spectrum of clinical presentations. As a result, many scientists have been focusing on ever larger cohorts of patients under the impression that larger data sets data will give better results.

http://www.sciencedaily.com/releases/2012/12/121213121805.htm
http://health.rush.edu/HealthInformation/In-Depth%20Reports/10/000103.ASPX
 

Sunday, April 15, 2012

One step closer to treating lymphoma

Lymphoma has most commonly been treated through radiation therapy and chemotherapy. Recently there was a new discovery which may lead to developing a  treatment using a compound called TL32711. This compound blocks a process in cells that helps it to avoid death. It makes it so cells die much easier.

This new compound had made different types of lymphoma vulnerable to these effects, but when added with another compound known as trail its effects were much greater. The way this was proved was basically because when cells die they go through a process called apoptosis. When they go through this they activate a protein called caspases. During the treatment of lymphoma with these two compounds the level of this protein were greatly increased, proving that cells were dying.



This TL32711 is not available just yet but clinical trials are starting to begin on more and more animals in order to get a better sense of how to treat humans.

Thursday, November 17, 2011

Spider Silk to the rescue?



Since 1989 there has been 1,500 clinical trails for gene therapies that have not been approved by the FDA. But hopefully this new study will change everything, Spider Silk. Spider Silk has been researched for years and have been found to be bio-compatible with everyday medicine. Scientists have modified spider silk to attached to diseased cells such as, human breast cancer, rather than healthy cells. The silk was also then engineered with a protein that will illuminate like fireflies do. By having the silk illuminate it allows researchers/doctors to get a clearer picture of where the diseased cells are located.

Article link: http://www.sciencedaily.com/releases/2011/08/110810101559.htm