pipeline

A board to discuss future MS therapies in early stage (Phase I or II) trials.

pipeline

Postby Brownsfan » Sun Nov 13, 2005 8:33 am

Any opinions and/or data on which of these drugs in the pipeline shows the most promise for becoming a viable and effective treatment in the next 2-3 years? Tovaxin, Neurovax, FTY720 seem to have the most "buzz" at this point. $12,000 per year for a possible 30% reduction in relapse rate just doesn't cut it.
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Postby dignan » Sun Nov 13, 2005 8:45 am

A quick reply -- if you want to know about the next 2-3 years, then Tovaxin and Neurovax aren't even under consideration because they are both going through phase II trials that won't be over until late 2006, or 2007, then the results have to analyzed and submitted to the regulator for approval to do a phase III trial, then the phase III has to be set up and run for 2 years then that data has to be analyzed...so Tovaxin and Neurovax are promising, but if all goes well, they might be on the market in 2010, but more likely later than that even.

On the bright side FTY720 is in Phase III so there is a real possibility that it could be approved within 3 years. The other possibilities that are in Phase III are Teriflunomide, Laquinimod and Mylinax. From what I've seen, FTY720 has the most impressive Phase II results of that bunch.

There's also a phase III trial of hormones that's for women only. Hormones have been discussed quite a bit on this site.
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Postby Brownsfan » Sun Nov 13, 2005 10:15 am

Thanks for the quick reply Dignan

What about drugs that have already been approved by the FDA such as statins and antibiotics? Might we see these drugs more widely prescribed in conjunction with the interferons or glatiramer if proven to be complementary? And why is there no formal trial for LDN?
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Postby bromley » Sun Nov 13, 2005 11:54 am

Brownsfan,

I have to agree with Dignan on timescales. Neurovax and Tovaxin look promising but there's some way to go. You could try and get on a trial but there is the risk that you might end up on the placebo!

Tysabri could come back sometime next year.

Copaxone and minocycline are being trialled so if good results are observed this might be an option in the not too distant future.

There are some trials in the UK for SP MS (cannaboids and lamitrigine (sp?)). But unlikely that the trials will be finished until 2008.

I have an appointment with my neuro to discuss starting on a DMD so will ask him what else is available / might be available soon.

We are, at the moment, in limbo, in terms of treatment options. There are the four DMDs which, as you noted, are expensive and not very effective. Tysabri looked promising but we all know what happened there. There are numerous drugs in trial but we are unlikely to see any available for at least three years.

What's annoying is the articles one sees on how good Neurovax is / Tovaxin is. Yet these treatments are several years away - if they are a good as the articles suggest!

By 2010 there should be a much wider range of treatments on the market. But the waiting is as cruel as it gets - we (or at least I) end up wishing our lives away to when effective treatments will be available. And time is not on our side given the degenerative nature of this disease. What also worries me is that most of the treaments being trialled are based on the theory that MS is an auto-immune disease (yet to be proven) and that the research was based on the mouse model of MS (a poor model to say the least).

But by 2010 they may have made headway with stem cells or with effective neuro-protective treatments (a New Zealand company Neuren has some interesting looking therapies in the pipeline). There's also the Promise 2010 (the NMSS major initiative) which is funding research to identify therapies for neuro-protection and repair.

What will be really sad is if we get to 2010 and (those of us who are still around) are still posting on this website about future treatments etc.


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Postby Brownsfan » Sun Nov 13, 2005 12:09 pm

Bromley, you couldn't have summed up my thoughts any better! I'm choosing to stay away from Tysabri when it's re-released so hopefully FTY720 will show continued promise and be available in 2008. I hope you will post on the outcome of neuro visit and his thoughts on the disease modifiers. I visited 2 docs, one recommended either copaxone or avonex, the other recommended either copaxone or rebif. I ended up choosing the copaxone - so far so good.

J
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Postby dignan » Sun Nov 13, 2005 8:43 pm

Good question about statins and antibiotics. I know the Phase II trials for zocor and lipitor started in 2003 or early 2004, so hopefully we'll be hearing about results some time in the next year. And hopefully not much after that for minocyline with copaxone. The thing I'm not clear on with substances already approved that could potentially be used off-label is, is there a certain standard they have to pass before doctors prescribe them? (ie a Phase II trial) Or do doctors prescribe based on their own opinion and/or patient need? Anybody know how something gets to be commonly prescribed off-label?
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Postby gibbledygook » Mon Nov 14, 2005 1:52 am

In the UK you can get anything prescribed privately. A doctor is free to prescribe absolutely anything she/he likes even if it isn't yet licensed! At least that's what my doctor says. :?
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Postby SarahLonglands » Mon Nov 14, 2005 2:29 am

True, but you still have to be able to persuade the doctor. Some are easier than others! :wink:
An Itinerary in Light and Shadow Completed Dr Charles Stratton / Dr David Wheldon abx regime for aggressive secondary progressive MS in June 2007, after four years. Still improving with no relapses since starting. Can't run but can paint all day.
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Postby CureOrBust » Mon Nov 14, 2005 11:15 pm

The medication also has to be available. for example I dont think tovaxin is available in most countries? or is it?

Here (in australia), you also have to get the doctor to sign a special form ("category a") which basically (by most peoples belief) means you are on your death bed without it. But I think that regulation can be read a little more relaxed.
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Postby gibbledygook » Tue Nov 15, 2005 5:19 am

One does need a good probably private doctor. My private quack claimed he could get me Goats Serum as some of his other patients were on it. I'll ask him to get Tovaxin and see what he can do! However I think you're probably right - the medication needs to be licensed somewhere before you can get it.
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Postby CureOrBust » Tue Nov 15, 2005 11:16 pm

gibbledygook wrote:However I think you're probably right - the medication needs to be licensed somewhere before you can get it.

In australia, the medication doesnt need to be licensed anywhere. I was just trying to say it needs to be physically available. For example, if Aimspro (ie Daval) did not have an office here in australia, I would not have been able to practically ship it from the US/UK.
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Postby viper498 » Mon Nov 21, 2005 8:34 am

Gibbledygook,

Tovaxin is not a drug in the conventional sense. You can't just get it. It is customized for each individual based on your own t-cells. They extract your own cells, and develop the end result, and inject it back in to you. This formula has to be injected with in 3 days of its creation. Tovaxin (pharmafrontiers) will require centers all of over the world that have the facilities and trained staff to actually formulate and disperse to all of the people who need it.

I think the U.S. should allow people, to choose drugs they want as long as they have made it through the Phase I trial. It is absolutely ridiculous that we are having to wait this long. It is our life, let us make our own decisions. Like it has been said. Time is absolutely not on our side. Let an FDA regulator come down with MS and see how he feels about the time frames. Unbelievable.
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Postby dignan » Mon Nov 21, 2005 9:34 am

Viper, I agree about making drugs available. Drugs should have to go through phases II and III, and it shouldn't be easy to get them if they've only gone through phase I, but if somebody needs a breakthrough drug to keep them hanging on, then I think it should be possible to prescribe things still in trials.
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Postby dignan » Mon Nov 21, 2005 7:41 pm

and here's an interesting article about people hoping to use experimental treatments to help patients with dementia...



Riskier Dementia Therapies Urged

Associated Press - November 21, 2005 - A fog is slowly creeping over Dr. William Deutsch's brain. He'd try a risky experiment, even one requiring brain surgery, in hopes of at least stalling his incurable dementia. But scientists have little to offer.

Families battling Alzheimer's disease and similar dementias increasingly are calling for a shot at riskier therapies that might bring bigger benefits than today's pretty safe but largely disappointing drugs.

It's a conundrum: Dementia robs its victims of the ability to fully consent to medical experiments. When loved ones can do so for them varies in part according to how much risk is involved.

Critics say that's one reason scientists and regulators have generally treated Alzheimer's more like a chronic disease than the killer it is, tolerating fewer side effects than for, say, cancer therapies. Further chilling was the 2002 halt of a study of a vaccine designed to attack Alzheimer's brain-clogging gunk that caused serious brain inflammation in a few patients.

Now the tide seems to be turning. Scientists are tentatively exploring a handful of bold, sometimes invasive, approaches — from another vaccine attempt to a gene therapy that requires brain injections — prompting an ethical debate about how to study a vulnerable population.

Patients and caregivers want a voice in those decisions, and often accept more risk than doctors predict, was the overarching message when the Alzheimer's Association arranged an unusual meeting between families, drug developers and the Food and Drug Administration.

"No one has ever survived this disease," Frank Broyles, the University of Arkansas' athletic director whose wife recently died of Alzheimer's, reminded the meeting.

"We would take any risk," even a drug with a 50 percent chance of death if there were an equal shot at benefit, he said of his own family.

Added Deutsch, a former New York podiatrist in the early stages of a similar dementia: "You're going to have to have some more invasive procedure done to get dramatic results. ... You should at least be given the choice."

The first formal study of family risk tolerance, published in this month's journal Neurology, supports those calls. University of Michigan researchers wondered: Is it OK for Grandma to undergo a spinal tap when she doesn't understand why? What if the research won't help her but might lead to future Alzheimer's treatments?

Using 10 research scenarios, they surveyed 229 elderly people at increased risk of developing Alzheimer's because a close relative had it. Most accepted even the greatest potential for side effects, such as gene therapy or a painful spinal tap, for themselves, and were only slightly less risk-tolerant when deciding for a loved one.

Yet until now, "the patient community's been conspicuous by its absence," Dr. Russell Katz, FDA's neurologic drugs chief, told the recent Alzheimer's Association meeting, to which a reporter was invited.

In contrast, a handful of cancer survivors helps the FDA privately deliberate what cancer drug research to allow, and the agency is starting a similar pilot project in Parkinson's disease — two patient groups that requested a role.

It's easy to say you'll accept more risk in hope of more gain. How to quantify that is the vexing problem of regulators like Katz. Does risk mean pain? Premature death? And what benefit justifies those risks, improved functioning or merely hope?

An experiment is just that: Nobody knows the full risks until participants have been treated, something difficult for desperate families to grasp, cautioned Dr. Steven DeKosky, the University of Pittsburgh's neurology chief.

Still, Katz said FDA ultimately allows most Alzheimer's proposals.

Among the most invasive: Scientists at Rush University in Chicago recently injected six patients' brains with a virus carrying a nerve growth factor that might reverse deterioration. A slightly different gene therapy approach, in California, produced promising preliminary results although two patients experienced dangerous bleeding in their brains. The Chicago researchers are tracking patient reactions, and a follow-up study could begin in a year.

But Broyles and others complain that such research moves too slowly. Consider Elan Corp.'s 2002 vaccine, ultimately found to slow memory decline somewhat and clear out some plaque, even though the experiment was halted before participants received a full dose. It took FDA more than three years to allow Elan to study a potentially safer version.

Caregivers differ on how much risk they'll tolerate; one called it gut-wrenching to watch a loved one suffer even a minor side effect.

But Deutsch, still able to measure how his abilities are melting away, boils the question down to family choice: "I don't think it's the scientists' job to pick who's going to get the treatment."

http://health.yahoo.com/news/128838
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