Thanks to funding and some strategic ‘match-making’ by the MND Association, a new drug may have taken one step closer to beginning clinical trials in MND after producing promising results in an animal model of the disease.
The drug, known as Cogane, was developed by the biotechnology company Phytopharm. It had already demonstrated in laboratory tests that it could help to protect neurones by promoting the production of natural, nerve nourishing substances called neurotrophic factors and early animal testing had hinted at its potential beneficial effects in MND. However, its journey towards clinical testing in MND had hit a road block because it hadn’t been extensively put through its paces in large numbers of the most widely used animal model of the disease, the SOD1 mouse. Without robust data from this model, there would have been little to encourage further investment in Cogane’s development.
So up stepped the Association to introduce Phytopharm to Professor Linda Greensmith at University College London, a leading MND researcher with considerable expertise in SOD1 mouse testing. With funding from the Association, Prof Greensmith and her team were able to conduct a rigorous study of the effects of Cogane, administered to the mice after they had developed MND-like symptoms.
The drug produced some significant improvements in muscle strength and motor neurone survival and managed to produce positive effects even in mice that had reached the later stages of the disease. To give more substance to these preliminary but very encouraging results, the research team will now go on to the painstaking work of examining more closely Cogane’s effects on the motor neurones and other key cells that play a critical role in the progression of MND.
After the disappointment of the Trophos trial results, it’s great to be able to share some positive news on the drug development front. We know from long experience that it’s wise to limit our excitement over positive results from the mouse model – after all, plenty of drugs have shown promise at this stage and have then gone on to fail in clinical trials. However, Prof Greensmith’s experience and expertise mean that Cogane will have been tested with the utmost rigor. As she herself commented, the results indicate that “Cogane has significant potential as a therapy for ALS and merits further evaluation”. We don’t yet know what Phytopharm’s next steps will be – these may become clearer once the more detailed data from Prof Greensmith’s work have been published, which could take the best part of a year. Let’s hope that we have a given Cogane enough of a boost to push it out of the drug development ‘doldrums’.
As an MND sufferer diagnosed March 2011. I would be willing to be put on human trials and think the option should be available to any rational human. By the time this trial has run through SOD1 many of us sufferers will be dead and the rest of us will be alot worse physically !!!
Hi Kate and Research Team,
Great news! As you stated definitely needed after recent disappointments. Just a shame, if successful, this could be up to a decade away before coming downstream to patients. Will it really be a year before we hear any more?
Not related but is there any chance we could have a research post about what things we can expect in 2012?
Matt, Maria and Robert,
We appreciate that the speed of drug development can seem incredibly slow, and often bureaucratic, but there really is good reason for this.
We understand that you want answers now, and a better treatment than riluzole, but the only way we can do this is by conducting rigorously controlled research that can tell us the ‘true’ answer, rather than what’s called a ‘false positive’ (when something seems positive, when the results are actually skewed and inaccurate). Although in its early stages of development, Cogane shows us a glimmer of hope for the future as Phytopharm make the next steps to put it through its paces.
We know that this particular treatment is a long way off, if indeed it does pass all the hurdles it’ll face in the next few years. It is however, reassuring to know that even though there are a number of promising trials currently underway across the world for MND, that researcher’s are not sat on their laurels, but instead are pushing their research efforts further forward to develop even more new treatments in the effort to find ‘the one’.
It’s understandable to feel that the bureaucracy of this process should be changed, as it’s frustrating to see potential treatments being put through their paces and not be able to take it in the hope that it’s beneficial. However, if unproven treatments were made readily available, researchers simply couldn’t make the leaps and bounds needed to progress treatments that could have a real potential.
One imaginary example would be if the trial drug olesoxime, which recently failed to prove its effectiveness in a Phase III trial, was made available on mass before the trial even began. Some people taking the drug would feel that by taking olesoxime that they had a sense of control over the disease, and think that the drug was effective. But we now know that olesoxime isn’t more effective than riluzole through the controlled study. This means that in our hypothetical scenario, that hundreds of people could have wasted their time, efforts and inevitably their hope on a drug that doesn’t work. In this time taking olesoxime, each person also wouldn’t be eligible to take part in new clinical trials due to taking an experimental treatment. This would mean that we would lose a lot of hope of being able to find a treatment as trials would be more difficult to recruit to, meaning slower trials with less significance and power.
Although clinical trials can seem time consuming, they really are necessary. As MND is a rare disease, clinical trials are notoriously difficult to design in order to ensure that they have meaningful results. Designing better and quicker clinical trials will aid us to find the answers as to whether a treatment is beneficial or not, without losing the significance of a study.
In answer to your questions Matt, for this particular study, it will take the research group until at least the end of the year to finish – when study results are published in scientific journals, it’s often the accumulation of years of work in a few short pages. This makes the project seem fairly straightforward but doesn’t explain the quandaries that were overcome or the time that the experiments took. It’s important that this study has the time to generate this rigorous evidence, so that Phytopharm (and its investors) can be assured that their drug stands the best chance of success in demonstrating its potential beneficial effects in clinical trials in the future.
At the beginning of the year we posted an update on what we acheived in 2011, but I’m afraid we simply don’t know what 2012 holds for us in terms of research news stories. One thing we do know that will happen this year is the 23rd International Symposium on ALS/MND, to be held in Chicago, USA in December, and a few other scientific meetings along the way. Hopefully we’ll be able to write some blog posts on these other meetings later this year as well as reporting from the symposium.
As always, as soon as we hear any news, we’ll keep you updated on the blog and our Twitter account.
Kind regards,
Research Team
I am a caregiver and son for a man that has suffered from ALS./MND for the past 7 years. He is at a point where he’ll sign any paperwork, assume any/all liability, in order to take a drug that may give him a few more months of life….A life he worked so hard to create and must now let go. Let me assure you,these experimental drugs are much safer than this disease and at the end of the day, there is nothing to lose. This bureaucracy appears to be less about the patient’s well being and more about the drug makers investors and market positioning. Hate to sound cynical, but ALS patients are in a truly unique position to test a wide array of potential therapies with little to no consequence if they don’t work. Why doesn’t the FDA acknowledge this?
I agree with Maria’s comment. I am also suffering from ALS and we should have the right to try anything in order to fight this disease. I am very disgusted with the slow pace the US FDA takes in approving clinical trials. It is time for all ALS patients to take action and demand that these bureaucratic regulations be changed. I want to have a chance to survive and not let some bureaucrat determine the course of my life.
I would try any drug to rid me of this nasty disease that is MND.
I am awaiting the final outcome of a blood test for Kennedy’s disease, which i understand only affects 1 in 40,000 people. I would try anything or be part of research to find a cure for this rare form of MND. Unfortunately I suspect that three of my younger brothers also have this disease. I have read the comments and whilst I am fit at 43 next week this surely is the ideal scenario not when its too late or the disease has progressed too far?
Surely the red tape and beurocratic hurdles that must be summounted to implement these new drugs in human trials must be sped up, even though as stated MND is rare, that is cold comfort for the people who are suffering the daily tortures that this insidious disease exacts upon them. The long term prognosis for an individual suffering from the onset of MND is grim to say the least. It makes a person think that the rarity of the disease renders it lower down in any serious medical research for a possible cure. A dear friend of mine has developed MND and I know for a fact that any opportunity in clinical trials for a possible cure or slowing of the horrendous symptoms until a cure is discovered would give literaly anything to be the first to test them. Professor Linda Greensmith, Research Team, please bare this heartfelt plea in mind if any trials become available.
Yours Hopefully
Derek Wilson