 |
 |
The following article is about patients who participated in the Phase I GDNF trial in Bristol, UK. They are also appealing to Amgen to allow them to continue their treatment with GDNF. Source: This Is Bristol/ Evening Post http://www.thisisbristol.com/ US GIANT HALTS 'LIFE-CHANGING' PARKINSON'S DRUG TRIALS 11:00 - 16 November 2004 Pioneering research in Bristol into use of a drug hailed as "a cure for Parkinson's Disease" has ended because the company behind it has halted the international trial. Five male patients were part of the study at Frenchay Hospital and all reported significant improvement to their quality of life while taking the drug glial-derived neurotrophic factor (GDNF). But now the research, sponsored by US pharmaceutical giant Amgen, will not continue because of an "inconclusive" second trial. The Bristol patients now face the return of symptoms such as uncontrollable muscle tremors, loss of speech and facial expression, and lack of balance. Around 120,000 people in the UK suffer with the degenerative disease. Among the trial patients was Keynsham man Richard Hembrough. He was diagnosed when he was 40 and said he could not believe the difference in his quality of life after he started receiving GDNF three years ago. The 60-year-old, like the others in the study, underwent invasive surgery to insert two pumps behind his abdomen that pushes GDNF through tubes straight into the basal ganglia. The drug then helps to re-grow brain cells that produce dopamine, the natural chemical that controls our muscles - effectively reversing the condition. Mr Hembrough, a former teacher who has four children and one grandchild, said: "It feels like Amgen has just shut our treatment down without taking any notice of the massive benefits it has given us. "Before I had GDNF, I couldn't get out of my armchair or lift my head properly. I had a long, expressionless face - it was awful. "Now I can do housework, go to the shops, make dinner, go on holiday and all sorts of things that were just simply impossible before. "It's devastating that they won't keep giving myself and the other four people the drug when it was so obvious it was working. "I'm now faced with this situation, which is very scary. Thinking of going back to how it was seems like a cruel blow after all we've been through." The research project in Bristol was led by Steven Gill, a consultant neurosurgeon at Frenchay. It was his team's initial scientific breakthrough, using the internal pump technique, that persuaded Amgen to give Frenchay the expensive drug for the trial and then launch a second study of another 42 patients in the United States. Mr Gill has been in talks with Amgen for months to try to persuade them to continue their research, but they announced the closure of the trial six weeks ago. However, Mr Gill said he believes the US methods were "dramatically different" to those used in Bristol and therefore would not have worked as well. He said: "The trial did not use the smaller catheters we did to inject the GDNF straight into the tissue. "This means that the drug would have defused around the area, but not necessarily right into it, thereby not effecting re-growth of the neurons needed to reverse the condition in the way we saw in Bristol. "The stopping of the study on an individual level is terrible. "We've asked for the drug for them on a compassionate basis but unfortunately there are strict criteria to be met on this and these men do not meet them. " Andrea Rothschild, spokesperson for Amgen, said the research had stopped because of grave safety concerns and because the drug did not seem to have any real effects on patients. She said half the patients were given the GDNF and half were given a placebo - no drug at all. Patients from both groups experienced improvements but the company was unable to determine whether it was as a result of the drug or not. She said: "We discovered two problems - first, some patients developed an immunity to the drug, and second, we found that it could cause irreversible brain damage. "So, ethically, we really had to shut down the trial." For more on this issue see The Grassroots Connection http://www.grassrootsconnection.com/grcissue_GDNF_research.htm ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn