Sep
01
2007

Gene Identification Helps To Screen for Diabetes Risk Of Juvenile Diabetes (type 1 diabetes)

Gene therapy is the new buzzword in a hope for cure of disease, and genetic tests also assist in predicting the risk for disease.

These predictions are reaffirmed by research that comes from McGill University in Montreal under Dr. Constantin Polychronakos, a professor of pediatrics and human genetics. A new DNA chip has been used for testing the genetic makeup that produces diabetes type 1 (also called Juvenile Diabetes). So far the analysis has confirmed four previously identified genetic locations that contribute to the development of diabetes 1, and the test identified a new location occupied by the gene.

With the research team working on the discovery of all the remaining genes, they are confident to develop a test which will predict the risk of developing diabetes. According to Dr. Polychronakos the findings will influence future treatment options. The most promising treatment for those already diagnosed with diabetes1 are stem cell regenerative treatments, such as islet transplantation, since the Langerhans islets are responsible for the production of insulin in the pancreas.

Gene Identification Helps To Screen for Diabetes Risk Of Juvenile Diabetes (type 1 diabetes)

Family tree for inheritance of type 1 diabetes

These therapies will only work if the immune system is also treated, so the patient does not suffer a relapse of the disease. Once all the diabetes genes are identified, there will not only be fast screening but also therapies that can be targeted more effectively.

Reference: The Medical Post, August 7, 2007, page 1 and 60 of the Medical Post

More on genetics and diabertes: http://www.who.int/genomics/about/Diabetis-fin.pdf

Last edited December 5, 2012

Oct
01
2006

Successful Gene Therapy For Melanoma

Scientists at the National Cancer Institute in Bethesda, Md. have developed a modality of gene therapy to treat patients with melanoma.
The therapy alters the patients’ own lymphocytes to recognize and attack cancer cells. Dr. Steven Rosenberg and his team developed the new technique by drawing a blood sample containing normal lymphocytes from an advanced melanoma patient. Next the cells are infected with a retrovirus, which delivers genes that target specific receptors on T cells. Once the cells are infused back into the patient, these receptors attach themselves to the molecules on tumor cells and activate the lymphocytes to destroy the cancer cells. This method is called “adoptive cell transfer”. In early experiments there seemed to be little or no benefit, but researchers refined the methods and found valuable and promising aspects regarding the treatment of melanoma patients. It is crucial to administer the cells in their most active growth phase. This was shown in a group of 14 patients, where 2 terminal melanoma patients experienced cancer regression. Dr. Jeffrey Medin, head of the clinical research program in gene therapy at the Ontario Cancer Institute in Toronto agrees that this new therapy is a breakthrough as it has virtually brought terminal patients “back from the brink”. It is also exciting to see that the researchers could refine the techniques in order to create a more successful therapy. As a result the method will be more applicable to other cancers and broader populations.

Successful Gene Therapy For Melanoma

Successful Gene Therapy For Melanoma

Although adoptive cell transfer has only been used in melanoma patients so far, the researchers have shown ways to engineer cells of the immune system in a similar way to attack breast, liver cancer or lung cancers.

More information about treatment of melanoma: http://nethealthbook.com/cancer-overview/skin-cancer/melanoma/treatment-melanoma/

Reference: The Medical Post, September 19, 2006, page 1 and 61

Last edited November 1, 2014

Jul
01
2004

Gene Therapy Can Repair Blood Vessels

When the first attempt of gene therapy were made, a concoction of LDL- receptor genes was infused into the portal vein of patients with a family trait of high cholesterol levels. This was back in 1989, and the results at that point were not encouraging.
In the meantime advances have been made in genetic research. Dr. Duncan Stewart of the cardiology department of St. Michael’s Hospital in Toronto who is considered by many as the guru of cardiac gene therapy, reports that we are now “at a crucial stage of the field”. He cautions, that having the gene on hand is only part of the answer. For good results it is also important to understand how the delivery system to the diseased organ works.

Gene therapy stands out from other treatment options, because instead of drugs, DNA is provided. DNA itself is not the therapeutic agent. It penetrates the cells where it sets up shop and produces proteins, which are needed for therapy. In the case of heart disease, where heart vessels are blocked or have been damaged, the idea is to send specific DNA sequences to the heart cells and stimulate the production of growth factors. These growth factors would trigger new heart vessels to grow and take over for damaged or blocked vessels. This is not just a dream of a research team: a gene therapy trial – the only one of its kind in Canada – is on its way under the leadership of Dr. Stewart. This ambitious project was launched in 2002. In seven sites across Canada 110 patients with heart disease are receiving the vascular endothelial growth factor gene (VEGF for short). The gene is being directly injected into the areas of the heart where blood vessels have become diseased or blocked. The placement is measured with a mapping system known as NOGA. At the end of the year the study enrolment will be finished. Patients will be followed up for results 6 months later. This therapy promises long lasting effects, but it  still has a long way to go.

Gene Therapy Can Repair Blood Vessels

Gene Therapy Can Repair Blood Vessels

Dr. Robert Hegele from the Blackburn Cardiovascular Genetic Laboratory at the University of Western Ontario is credited with uncovering eight genes for human disease (four of them are related to cardiovascular illness) and 80 mutations in other genes that are contributing to premature heart disease and diabetes. Dr. Hegel’s interest is to discover the villains that predispose people to illness, but at the same time he cautions that genes are not everything. Being genetically susceptible does not necessarily foretell one’s destiny when it comes to developing heart disease. Dr. Hegele emphasizes that the longer he works in the genetic field, the more he respects environment and lifestyle and finds that most people can even overturn genetic susceptibility and he leaves us with the final remark: “Tell your patients to stay away from tobacco, eat wisely and get that needed exercise.”

More info on arteriosclerosis (hardening of the arteries): http://nethealthbook.com/cardiovascular-disease/heart-disease/atherosclerosis-the-missing-link-between-strokes-and-heart-attacks/

Reference: The Medical Post June 29, 2004 (Vol. 40, No.26): page 35

Comment (July 9, 2012): Note that this trial failed to show effectiveness as indicated in the paper below. So, eat wisely, exercise and don’t smoke.

VEGF gene therapy fails to improve perfusion of ischemic myocardium in patients with advanced coronary disease: results of the NORTHERN trial. Stewart DJ, Kutryk MJ, Fitchett D, Freeman M, Camack N, Su Y, Della Siega A, Bilodeau L, Burton JR, Proulx G, Radhakrishnan S; NORTHERN Trial Investigators. Mol Ther. 2009 Jun;17(6):1109-15. Epub 2009 Apr 7.

Last edited Oct. 26, 2014

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