Researchers report on encouraging trial results of KNS-760704
09 December 2009
At the second day of the 20th International Symposium on ALS/MND, researchers have presented the encouraging results of a Phase 2 clinical trial looking at the safety and tolerability of a drug called KNS-760704 in ALS patients. ALS (Amyotrophic Lateral Sclerosis) is the most common form of motor neurone disease (MND).
The results were presented by Dr Merit Cudkowicz, an associate professor of neurology at the Massachusetts General Hospital of Harvard Medical School. Dr Cudkowicz is an expert in co-ordinating MND clinical trials in the USA.
The two-part Phase 2 trial found that KNS-760704 was safe and well-tolerated in ALS patients for up to nine months. The trial results also showed trends suggesting the potential for reducing the rate of decline in the functional capability of patients, for example, how far people can walk unaided, lifting objects, dexterity in eating; and survival.
The researchers emphasised that KNS-760704 is still in the early days of development for patients and that further testing in a large, longer-term, carefully-monitored Phase 3 trial is needed to establish the necessary evidence that the drug is both safe and effective for ALS patients.
“We are very encouraged by the results presented today in Berlin and, at the same time, we’re acutely aware of the work that remains to be done,” said Dr Michael Bozik, president and CEO of Knopp Neurosciences Inc, the pharmaceutical company behind the development of KNS-760704.
In Part 1 of the trial, 102 patients received the treatment or the placebo every day for 12 weeks. Patients taking KNS-760704 received the drug in one of three doses - low, medium or high. KNS-760704 showed a dose-dependent trend in slowing the rate of disease progression with the greatest benefit observed in the high dose group.
In Part 2 of the trial, 92 patients were randomly assigned to receive low or high doses of KNS-760704 for 24 weeks. In addition to results again suggesting a dose-dependent trend in slowing the rate of disease progression there was also a trend toward a survival benefit in the high group compared with the low dose group.
Dr Belinda Cupid, research manager at the MND Association who attended the announcement, explains: “Phase 2 clinical trials determine the size of the dose, the timing of the dose and how the drug is to be taken for the next phase of testing.
“Although Phase 2 testing provides some indication of a drug’s ability to treat a disease, the number of patients involved at this stage is much too small for the findings to be relied upon.
“Phase 3 clinical trials are important as they aim to show whether or not the drug actually has a beneficial effect on patients. This stage of testing will usually involve hundreds of patients which is enough to allow a reliable assessment of the drug’s effectiveness. Phase 3 results will determine whether or not a drug is to be approved to treat a disease."
She continues: “Researchers know that MND is caused by motor neurones dying but what we are now trying to find out is what goes on in these cells before they die.
“We are looking inside these cells as we believe that the compartments which supply energy in the motor neurones are one of the main culprits. Evidence is building to show that when the energy supply goes wrong, MND develops. KNS-760704 acts by improving the energy supply within motor neurones.
“It’s promising news to hear that KNS-760704 will now be tested for its beneficial effects in people with MND."
Phase 3 testing of KNS-760704 in ALS may begin in mid-2010 at sites in Europe and North America under a protocol under development by Knopp Neurosciences Inc., in consultation with global regulatory authorities.
If you are affected by MND and would like to know more about the development of KNS-760704, please contact Knopp Neurosciences Inc. at tom@knoppneurosciences.com.
Contact:
For media enquiries please contact Louise Coxon Communications Manager
01604 611843 / 07760 765142
louise.coxon@mndassociation.org
Notes to editors
The 20th International Symposium on ALS/MND is taking place in Berlin, Germany from 8 to 10 December.
This is the world’s largest medical conference on MND and is regarded as the premier event in the MND research calendar.
The event is organised every year by the UK-based MND Association and hosted this year by the Deutsche Gesellschaft für Muskelkranke e.V. (DGM) (German Neuromuscular Diseases Association).
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Scientists and clinicians gather at the 20th International Symposium on ALS/MND
08 December 2009
Eva Luise Köhler, patron of the German Alliance for Chronic Rare Diseases and wife of the German President and First Lady of Germany, has formally opened the 20th International Symposium on ALS/MND in Berlin.
Mrs Köhler welcomed over 850 delegates to what has become the world’s largest medical conference on MND. The International Symposium, organised by the MND Association, is now regarded as the premier event in the MND research calendar.
Mrs Köhler said: “I would like to welcome all of you to this year’s symposium in our capital city, Berlin. I am so glad that so many of you are here expressing your interest in MND.
“I hope there will be interesting and fruitful discussions which will help to provide further inspiration and impetus for advancing research and care for MND patients.”
Baroness Ilora Finlay of Llandaff, a patron of the MND Association and palliative care consultant and champion, gave an uplifting lecture on the difficult subject of end of life decisions. Her presentation highlighted how essential good palliative care is for people with MND and their families.
Baroness Finlay stressed that good communication and good listening is essential if health and social care professionals are to help patients who are feeling helpless and hopeless.
She said: “Life has been very cruel to our patients and there are some things that sadly we cannot change. But we can make a difference by asking: ‘What can we do, however trivial, to improve today?’.
"Our role is to bring some light into the darkness that patients are in and we can do this by listening and then we know exactly what we can do to help. We all need hope and by listening to our patients we are making sure that we do not abandon the hope they hold.”
So far, 870 delegates from 35 countries have walked through the doors to this year’s conference, signifying just how valued the International Symposium is within the MND clinical and research communities. It is the place where researchers, clinicians, and health and social care professionals, want to discuss their work, their progress and their achievements on the journey to a world free of MND.
This year there will be 100 speakers from 18 different countries as well as 340 scientific and clinical summaries which will be displayed around the symposium on individual 1m by 1m poster boards. The programme for the event contains a varied platform of debate, discussion and information-sharing, which represents the energy and dynamism of the MND clinical and research communities.
Dr Brian Dickie, director of research development at the MND Association who plays a key role in organising the symposium every year, said: “Twenty years ago the MND Association held the first small, international meeting on the disease. Very little was known about the disease, research was limited and multi-disciplinary care was in its infancy.
“Times have changed significantly. Now we are starting to see an explosion of new knowledge. It’s an exciting position to be in, but how we harness and communicate this information between our research labs and clinics around the world will be a major challenge. This will be one of the main areas of discussion at this year’s symposium. It’s a challenge however that we are ready for as we enter a new era of MND research.”
You can keep up to date with the latest from this year’s symposium by reading our symposium blog on our My Symposium pages.
Contact:
Louise Coxon Communications Manager
01604 611843
louise.coxon@mndassociation.org
Notes to editors
The 20th International Symposium on ALS/MND is taking place in Berlin, Germany from 8 to 10 December. The event is organised by the UK-based MND Association and hosted this year by the Deutsche Gesellschaft für Muskelkranke e.V. (DGM) (German Neuromuscular Diseases Association).
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