Unexpected use of former cancer drug, Zebularine...

Researchers at Lund University have unexpectedly discovered that an old cancer drug can be used to prevent rejection of transplanted tissue. The researchers now have high hopes that their discovery could lead to new treatments for both transplant patients and patients with autoimmune diseases.

"Our group were studying the effects of the old tumour drug Zebularine, (see structure) developed in the USA in the 1960s, and by chance we discovered that it had completely unexpected effects on the immune system," says Leif Salford, Senior Professor of Neurosurgery.

"It turned out that Zebularine has the ability to subdue the reaction of the body's immune system. This could be important in situations where tissue or organs are transplanted. We also think it could be used to curb the body's attacks on its own tissue in autoimmune diseases, for instance type 1 diabetes or rheumatoid arthritis," says Dr Nittby.

In studies on animals, the researchers used rats that were made diabetic. The researchers transplanted the islets of Langerhans  cell groups in the pancreas producing insulin -- from healthy rats from another kind of rat into those with diabetes. The diabetic rats were divided into two groups; one group were treated with Zebularine and the other, the control group, did not receive any treatment. The diabetic rats that were treated with Zebularine survived for a significantly longer period than the untreated rats.

"It is very interesting that we only treated them with Zebularine for two weeks, but the effects of the treatment could be observed throughout the 90-day follow-up period.

"The findings are very exciting and are a sign that the immune system was not just generally suppressed, but that the treatment was more targeted. Neither did we see any signs of side-effects," said Dr Nittby.

The researchers are now working intensively to further refine the treatment. The next step is to teach certain cells in the immune system -- the dendritic cells -- to accept certain specific proteins using the Zebularine treatment. This would mean that the treatment could be targeted even more.

Unexpected use of former cancer drug

Drug shrinks brain tumors in children with tuberous sclerosis complex, study suggests

In continuation of my update on Everolimus

 "Every patient in this study experienced a decrease in size of their tumors, and no patient required surgery for their tumors after treatment with everolimus," says Dr. Franz, co-director of the TSC Clinic at Cincinnati Children's and the study's main author. "Thirty-five percent of patients in this study on everolimus had at least a 50 percent reduction in tumor volume after an average of 42 weeks on medication."

The phase III study was conducted among 117 patients with TSC who were randomly assigned to either everolimus or a placebo. Patients were 9 ½ years old on average but ranged from infants to adults. No patient on placebo showed improvement in their tumors. Tumor volume was measured by MRI assessment of the brain.

Dr. Franz conducted an earlier, phase II study of everolimus published in The New England Journal of Medicine in 2010. Based on that data, the U.S. Food and Drug Administration granted accelerated approval of everolimus for patients with these tumors, known as subependymal giant cell astrocytomas, or SEGAs. The new, placebo controlled study was conducted to confirm these earlier results.

Prior to FDA approval, surgery was considered standard therapy for SEGAs, but everolimus is a potential alternative to surgery and the first targeted medical therapy for TSC, says Dr. Franz.

"Children and teens may not only avoid surgery but they also may see improvement in other aspects of this disease, including a reduction or even elimination of hydrocephalus  a buildup of fluid inside the skull leading to increased intracranial pressure. Hydrocephalus is commonly associated with these tumors because they are located deep within the brain in spinal fluid pathways, or ventricles."

In Dr. Franz's 2010 study, patients reported their quality of life, as measured by a validated quality of life and neuropsychological assessments, improved at three months and six months after treatment with everolimus...

Ref : http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(12)61134-9/fulltext

Drug shrinks brain tumors in children with tuberous sclerosis complex, study suggests