Scientists have hailed a ‘penicillin moment’ in cancer treatment following trials of a drug that uses genetic data to target the formation of specific tumours.
The study has raised hopes drug manufacturers will be able to tailor drugs to individual cancers that will halt them in their tracks and even reverse the growth of existing tumours.
The breakthrough is one of the most significant to use advances in our knowledge of DNA to tackle the root causes of disease.
As part of the latest research, scientists in California developed a drug to block the effects of a specific gene mutation, B-RAF, linked to malignant melanoma – one of the deadliest cancers.
Cancer cells
In one small clinical trial, tumours shrank by at least 30 per cent in 24 out of 32 patients with B-RAF mutations, and disappeared entirely in two other patients.
The drug cannot yet be declared a success: it comes with side-effects, can only treat the specific B-RAF mutation and there are no indications of its long-term usefulness.
However, a study of the chemical process behind the drug, detailed in the journal Nature, demonstrates the potential for speedy development of similar treatments targeting the particular genetic mutations that lie behind different types of tumour.
Professor Mark Stratton, Director of the Wellcome Trust Sanger Institute in Cambridge, which first linked B-RAF to malignant melanoma, said: “We’ve entered an end game in which we are going to complete our understanding of what causes cancer.”
‘We have entered an end game in which we will complete our understanding of what causes cancers.’
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Scientists have discovered a way to shrink tumours in certain cancers – a finding that provides hope for new treatments.
The cancers in question are those caused by a new class of genes known as ‘microRNAs’, produced by parts of the genome that, until recently, were dismissed as ‘junk DNA’.
The current finding identifies one particular microRNA (microRNA 380) that appears to disable the king of tumour suppressors, the P53 gene. In order for a cell to become cancerous, P53 must either be mutated or otherwise disabled.
For the research, Australian and American scientists studied neuroblastoma, a childhood cancer of the nervous system in which 99pc of patients do not have mutations of the P53 gene.
Dr Alex Swarbrick, from Sydney’s Garvan Institute of Medical Research, Dr Susan Woods from Brisbane’s Queensland Institute of Medical Research and Dr Andrei Goga from The University of California San Francisco found instead that neuroblastomas disable p53 by over-producing microRNA 380. When they blocked the microRNA, P53 production resumed, cancer cells died and tumours became much smaller.
“The revolutionary thing about this finding is that it’s the first time anyone has blocked the growth of a primary tumour by the simple delivery of a microRNA inhibitor,” said Alex Swarbrick, from Sydney’s Garvan Institute of Medical Research.
“By that, I mean we delivered the microRNA inhibitor in a way we might give it to a person – as a twice-weekly injection – not using some genetic trick. It’s the closest thing to a clinical result that’s yet been published. That, of course, makes this microRNA a potential therapeutic target for all cancers that depend on it,” he added.
The results are reported in the prestigious international journal Nature Medicine, online today.
A newly developed drug shows promise in treating the most aggressive form of thyroid cancer, according to a new study.
About 5 percent of thyroid cancer patients have a very aggressive form of the cancer which spreads to other organs, such as the lungs, and can be fatal. Because these tumors cannot usually be treated effectively with surgery or radioactive iodine, patients often have no choice but to be treated with chemotherapy, which is not very effective.
In the new study, 37 patients with aggressive thyroid cancer took a drug called pazopanib every day. In nearly half of the patients, the size of their tumor decreased by at least 30 percent, said study author Dr. Julian Molina, an oncologist at the Mayo Clinic in Rochester, Minn.
On average, patients were able to take pazopanib for about a year before their cancer started growing again.
“For a disease for which we did not have any effective treatment, in which the treatments were vey toxic, we were able to find that this drug pazopanib works in 50 percent of patients, allowing the tumor to shrink and the patient to have a good quality of life,” Molina said.
This is the best response reported yet for aggressive thyroid cancer, according to the researchers. The best existing treatment, a drug called axitinib, is effective in about 31 percent of patients, Molina said.
About 37,000 patients a year are diagnosed with thyroid cancer, making it more common than ovarian, esophageal and gastric cancer. Cases of thyroid cancer have doubled over the last decade, which researchers suspect is due to an increase in detection. For about 90 to 95 percent of patients, thyroid cancer is treatable and not fatal, Molina said.
http://www.msnbc.msn.com/id/39239797/ns/health-cancer/
A protein crucial for the immune response appears is a key player in the progression of a devastating form of childhood leukemia called T-cell acute lymphoblastic leukemia (T-ALL) has been identified by scientists.
Suppressing the activity of the protein kills the leukemic cells, the study shows, opening a potential avenue to new drugs that could prevent progression of the disease.
Led by Iannis Aifantis of New York University and colleagues at the Institute Municipal d’Investigacions Mediques in Barcelona, the new study discovered the protein by picking up on a bit of cross-talk, or conversation, between two unrelated genes.
“We are very excited about this discovery because small molecule drugs that block this protein are already in development.
http://www.medindia.net/news/Scientists-Find-Key-Pathway-Implicated-in-Progression-of-Childhood-Cancer-74277-1.htm
Gene therapy has been used for the first time to treat an inherited blood disorder in what doctors say is a major step forward.
A man given pioneering treatment to correct a faulty gene has made “remarkable” progress, a US and French team has revealed.
Gene therapy is an experimental technique that manipulates genes in order to treat disease.
It has seen some successes, but also setbacks, including a patient’s death.
Beta thalassaemia is an inherited blood disorder that affects the body’s ability to create red blood cells.
The first gene therapy trial was in an 18-year-old man with a severe form of the condition, who had been receiving regular blood transfusions since the age of three.
Stem cells from his bone marrow were treated with a gene to correct for the faulty one.
They were then transfused back into his body, where they gradually gave rise to healthy red blood cells.
Three years after the treatment, which took place in 2007, the man remains mildly anaemic, but no longer needs blood transfusions, doctors said.
The team, led by Philippe Leboulch, of Harvard Medical School in Boston, said: “At present, approximately three years post-transplantation, the biological and clinical evolution is remarkable and the patient’s quality of life is good.”
http://www.bbc.co.uk/news/health-11313273