New Meds

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neglect

neglect

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There have been several new meds in neurology over the past few years. While many are horrible 'me too,' or the ultimate 'me too' meds: XR versions, poly pills like Aricept+memantine, there has been very real innovation that's helped patients a great deal. I'm thinking of all the new meds in MS: pills, biologics, including ocrelizumab for PPMS. In sleep: orexin antagonist, Belsomra, which I've found very helpful if you set expectations low (it is not a knock-out drug). Seizure management is a different landscape now both medically AND surgically (watching my mentors program VNS highlighted the art >>> science of medicine), although for my purposes things remain the same: lamictal/trileptal/keppra/ZON/TOP. And recently a new med for PD psychosis, which I have used both on and off label with excellent tolerance and effect. We've discussed endovascular for stroke, and while very limited on a population basis, it provides great benefit in suitable patients.

I feel that I'm finally living the promise of neurology: great trials in the worst diseases are being done to show the effectiveness of a new wave of meds. Next up: DMTs in AD and new migraine meds (and yet another way of delivering sumatriptan and l-dopa). Exciting times.

So any experience, rants or raves with any new meds and interventions?
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Great thread for two reasons. Confirms my suspicion that neurology will be a very different field (in a good way) mid career and this is one of the reasons I'm interested. This is also a good thread for the occasional passerby to read to help dispel the "no good treatments" myth.

For context... Are you in general neurology and do you use all of the drugs you listed?

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NWwildcat2013 said:
Great thread for two reasons. Confirms my suspicion that neurology will be a very different field (in a good way) mid career and this is one of the reasons I'm interested. This is also a good thread for the occasional passerby to read to help dispel the "no good treatments" myth.

For context... Are you in general neurology and do you use all of the drugs you listed?

Sent from my iPhone using SDN mobile
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Fair. I'm gen neuro with sub-specialty in ad, but my role in private practice is managing clinical trials: amyloid, ms, pd, stroke. I use every lever. But I hate indiscrimate use, sloppy use, and use without a metric.
 
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Much less activity than I'd have thought. Oh well.

For you lurkers out there, I'm pretty dismal about technology and medicine (Theranos is first and foremost, but I also dislike most tech inventions that claim to help biological processes. Patientslikeme doesn't get people into trials. And do we really need a new technology to get Imitrex into humans?)

But there are exciting exceptions. 1. Telemedicine. This is amazing. Giving tPA over a video conference line is just great work. So much neurology can be done easily over a video line. If only regulations allowed us to bill. 2. Apps. Here's a bright spot in medicine. I can now do a computerized cognitive test, bill 96120, and make 40-50 bucks for a procedure. And the cognitive tests are solid. 3. Medical information is much more available. Books really are dead.
 
Sadly one new medication that won't be coming out is sola. Lilly's drug failed in mild AD with MMSE 20 and over and a positive amyloid scan. Merck will read out next year and I don't think anyone expects it to be positive in AD. Their MCI (like Sola, enriched with a + PET scan) trial is done recruiting. This is not the end of the road for all monoclonals, more monoclonals are in the pipeline: Crenuzumab and Ganterumab from Roche, Adu from Biogen, and another one from Eisai will exit a large phase II trial. Prevention trials will prove to be very slow moving.
 
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SMA now has a disease modifying drug. Weirdly biogen stock is down: I guess the finance folks aren't happy that the price is only a bit more than the pediatric neurologist's annual salary.
 
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cool, so we can finally give ritux on label and pay 10x for it.
 
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Thama said:
cool, so we can finally give ritux on label and pay 10x for it.
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I have a PPMS patient who's insurance denied them ritux two weeks ago, despite being on it for about 2 years (yep, I took a risk on + Ocrelizumab data. It's one of the best calls I've ever made.). So now they get to pay more for Ocre when it comes out in a few weeks.

Rituxan is good, but apparently there are differences in epitope binding, people give it differently (wait for CD20 cells to repopulate or not?), and it penetrates lymphatic tissue differently as well. There are also non-trivial allergic reactions.

I was shocked that Rebif cost as much as it does. For being such a poorly tolerated med, from a company with no MS or neuro pipeline.

Anyway, this represents true innovation. Roche took a risk on this medication when B-cells were not thought to play a role in MS. They did the trials, went after a PPMS indication, and set the price for a drug that will save lives and prevent disability. This is as far away from objectionable, Shkrelian practices as one can imagine.

Now the field will look towards remyelinating strategies.
 
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New med for early ALS. Weirdly I went to an ALS lecture (for CME) and It wasn't mentioned. I just got bored to death listening to all the genetics.
 
neglect said:
Just read this infuriating story. As I said before, the data on the DMD drug is poor and now the story comes out, it sounds very fishy. The kicker here is that insurance companies refuse to cover the med.
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Couldn't read the paper because I don't have a WSJ subscription but I've heard there are lots of problems with this Drug and it's approval.
 
Amygdarya said:
Couldn't read the paper because I don't have a WSJ subscription but I've heard there are lots of problems with this Drug and it's approval.
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If you google "How the FDA Approved a $300,000-a-Year Drug Its Own Experts Didn’t Believe Worked," you'll get it. Sorry for the formatting. For some reason, the WSJ allows you to access a few of its articles directly from google.
 
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Not really a new med, but a new finding about an older med. NEJM - Error

Looks like asa + plavix for 3-4 weeks after TIA and minor stroke wins. Longer = bleeding. Thoughts? And get ready to load that plavix.
 
neglect said:
Not really a new med, but a new finding about an older med. NEJM - Error

Looks like asa + plavix for 3-4 weeks after TIA and minor stroke wins. Longer = bleeding. Thoughts? And get ready to load that plavix.
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I was uneasy with the broad uptake in this practice after the CHANCE trial in China given the uncertain generalizability of their results, so I was happy to see when POINT came on line. We recruited for it and it was a well-run trial. Overall event rates were on the lower side of expectations, but the benefit was clear. It will be interesting to see how the community applies the results, either as a shorter term of therapy (as you mention) given the early ischemic gains and relatively static rate of hemorrhagic complications, or as a more direct application of the trial's 90-day window.
 
typhoonegator said:
I was uneasy with the broad uptake in this practice after the CHANCE trial in China given the uncertain generalizability of their results, so I was happy to see when POINT came on line. We recruited for it and it was a well-run trial. Overall event rates were on the lower side of expectations, but the benefit was clear. It will be interesting to see how the community applies the results, either as a shorter term of therapy (as you mention) given the early ischemic gains and relatively static rate of hemorrhagic complications, or as a more direct application of the trial's 90-day window.
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Interesting. All I had to hear was that Chinese platelets were not so different than other platelets and I ran with CHANCE, applied it to folks who didn’t get into POINT. I was one of those who did the broad uptake thing. Now that POINT is over, I think the jury has rendered a firm verdict: short term dual anti-plts are good. But timing? Loading? Ending?

Here’s Grotta’s take: NEJM - Error

Meanwhile, I come back to a dodge on the anti-platelet question: we’re overlooking the importance of BP and exercise. We allow people to go off statins. We’re not pushing these patients to really change their lifestyles.
 
So sad that new meds in neurology is >10 months old and I need to attest that my reply is discouraged.

Anyway, over last 10 months more new migraine meds have perhaps made more people stop complaining they have a headache. And the MS market gets a bit more crowded with fingolimod. Another MS Drug Wins FDA Approval

Sad to see two major AD trial fail as well.
 
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neglect said:
So sad that new meds in neurology is >10 months old and I need to attest that my reply is discouraged.

Anyway, over last 10 months more new migraine meds have perhaps made more people stop complaining they have a headache. And the MS market gets a bit more crowded with fingolimod. Another MS Drug Wins FDA Approval

Sad to see two major AD trial fail as well.
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Are you referring to CGRP meds?
 
The CGRP studies basically look like expensive Topamax. Help some people, don't help others, reduce HA by a similar amount to other first line agents. They're another thing to throw at patients, but anyone thinking they are the magic bullet and using them before trying other established first line meds is probably deep in a drug companies pocket whether they are aware of this or not.
 
Thama said:
The CGRP studies basically look like expensive Topamax. Help some people, don't help others, reduce HA by a similar amount to other first line agents. They're another thing to throw at patients, but anyone thinking they are the magic bullet and using them before trying other established first line meds is probably deep in a drug companies pocket whether they are aware of this or not.
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Nothing wrong with pharma. But for those responders to the CGRPs who didn't respond to others, I actually think these can be life changing - like Topamax was!

COI: Did some of the trials, but do no ad boards or marketing/speaking for these.
 
Nuplazid, or pimvanserin, a serotonin subtype antagonist and NOT a dopaminergic med looks like it just broadened it's indication from PD with hallucinations/delusions to dementia with psychosis. Looks like a big win to me.

This drug is a bit of a sleeper, kinda got ignored for the last few years, beaten up by stupid CNN article, which was nearly journalistic malpractice. It nearly missed significance for schizophrenia (not my deal), but they ended a trial early for success. The trial enrolled all folks with dementia, so it'll be interesting to see how many of them had AD vs. DLB. It was also a withdrawal study, which is a bit weird, but consistent with efficacy. Further safety data will be interesting - the FDA gave them a black box warning as an anti-psychotic, but the mechanism of action is different than the neuroleptics and dopaminergics.

Big win in dementia, even if it is only symptomatic and psychosis doesn't affect everyone with dementia. Huge win for DLB.
 
Ive been reading about the light/electromagnetic therapy for Dementia; and was very skeptical. But it could be real. Theres a lot of data now-

www.prnewswire.com

Alzheimer's Memory Loss Reversed by Easy-to-Wear Head Device from NeuroEM Therapeutics

/PRNewswire/ -- NeuroEM Therapeutics, a clinical stage medical device company focused on neurodegenerative diseases, today announced findings from an early...
www.prnewswire.com www.prnewswire.com

medicalxpress.com

Alzheimer's memory loss reversed by new head device using electromagnetic waves

There is finally some encouraging news for the millions of Americans suffering from Alzheimer's Disease. NeuroEM Therapeutics today announced findings from an open label clinical trial showing reversal of cognitive impairment in Alzheimer's Disease patients after just two months of treatment...
medicalxpress.com medicalxpress.com
 
deathmerchant said:
Ive been reading about the light/electromagnetic therapy for Dementia; and was very skeptical. But it could be real. Theres a lot of data now-

www.prnewswire.com

Alzheimer's Memory Loss Reversed by Easy-to-Wear Head Device from NeuroEM Therapeutics

/PRNewswire/ -- NeuroEM Therapeutics, a clinical stage medical device company focused on neurodegenerative diseases, today announced findings from an early...
www.prnewswire.com www.prnewswire.com

medicalxpress.com

Alzheimer's memory loss reversed by new head device using electromagnetic waves

There is finally some encouraging news for the millions of Americans suffering from Alzheimer's Disease. NeuroEM Therapeutics today announced findings from an open label clinical trial showing reversal of cognitive impairment in Alzheimer's Disease patients after just two months of treatment...
medicalxpress.com medicalxpress.com
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Oh boy, I'm also VERY skeptical about these approaches. These articles did nothing to offset this, although I"m encouraged that they are doing proper clinical trials to prove safety and efficacy.

The transcranial electromagnetic treatment was a phase 1b trial with no control. While safe, they essentially showed a test-retest phenomenon in their 8 subjects. Best luck to them, but this is a LONG way from FDA submission.

The light therapy is a hard sell. Although these lights, at various wavelengths, seem to help neurons in culture and in mice brains, I'm not sure they get INTO the human brain, and certainly not sure they are going to help much.
 
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neglect said:
Oh boy, I'm also VERY skeptical about these approaches. These articles did nothing to offset this, although I"m encouraged that they are doing proper clinical trials to prove safety and efficacy.

The transcranial electromagnetic treatment was a phase 1b trial with no control. While safe, they essentially showed a test-retest phenomenon in their 8 subjects. Best luck to them, but this is a LONG way from FDA submission.

The light therapy is a hard sell. Although these lights, at various wavelengths, seem to help neurons in culture and in mice brains, I'm not sure they get INTO the human brain, and certainly not sure they are going to help much.
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Yea agreed. I read about these first 3 years ago and completely dismissed them.

But actually I was just researching, there is already evidence of CNS penetration and reduction or amyloid and some other proteins. There is also an oscillations theory, that in principle works for DBS.( I haven’t researched that much yet)

The article says they are starting a proper trial to get FDA approval. There’s no shortage of patients. In fact, looks like atleast 2 long term trials are completing early next year!! ( different companies)
All they need is FDA clearance or something, millions of people with dementia are gonna ask for it.

Also, with the government planning to Approve “ right to try” act, I don’t think it’s too far along when we will be recommending patients to join a trial or just start using this thing!!
 
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deathmerchant said:
Yea agreed. I read about these first 3 years ago and completely dismissed them.

But actually I was just researching, there is already evidence of CNS penetration and reduction or amyloid and some other proteins. There is also an oscillations theory, that in principle works for DBS.( I haven’t researched that much yet)

The article says they are starting a proper trial to get FDA approval. There’s no shortage of patients. In fact, looks like atleast 2 long term trials are completing early next year!! ( different companies)
All they need is FDA clearance or something, millions of people with dementia are gonna ask for it.

Also, with the government planning to Approve “ right to try” act, I don’t think it’s too far along when we will be recommending patients to join a trial or just start using this thing!!
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Having been through this over the years, I REALLY doubt that lights, magnets, AC/DC currents, or retinal flashes will work in humans. I'll do the trials though.

The "Right to Try" act actually IS the law of the land. The fact that you thought it was under approval is consistent with the fact that it hasn't affected many people. And the reason for that is because it is a sham. The linked article goes into why pharma isn't eager for the public to get their hands on meds before any safety data is available. They face risk, and with no approval process, no benefit.

But that said, I think this right to try HAS had some negative consequences, because one of the companies, BrainStorm, was going to permit some sort of stem cell therapy (for 300K, and it isn't like insurance is going to pay for that), then backed out. Now they are going with a conventional trial. But this has already opened up the door to the charlatans who make Oz look like honest Abe Lincoln. I've seriously had a recent case of a poor family falling victim to the liars using the right to try to try to backdoor BrainStorm, selling false promises of stem cells in ALS. Trying to explain that injecting random fat cells into your spine or CSF (for a fraction of 300K, but more than a car costs) is not the same as a high quality clinical trial with a placebo control was like trying to explain Celtic history to me.

Do you have a link on any later trials completing in dementia? Thanks.
 
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neglect said:
Having been through this over the years, I REALLY doubt that lights, magnets, AC/DC currents, or retinal flashes will work in humans. I'll do the trials though.

The "Right to Try" act actually IS the law of the land. The fact that you thought it was under approval is consistent with the fact that it hasn't affected many people. And the reason for that is because it is a sham. The linked article goes into why pharma isn't eager for the public to get their hands on meds before any safety data is available. They face risk, and with no approval process, no benefit.

But that said, I think this right to try HAS had some negative consequences, because one of the companies, BrainStorm, was going to permit some sort of stem cell therapy (for 300K, and it isn't like insurance is going to pay for that), then backed out. Now they are going with a conventional trial. But this has already opened up the door to the charlatans who make Oz look like honest Abe Lincoln. I've seriously had a recent case of a poor family falling victim to the liars using the right to try to try to backdoor BrainStorm, selling false promises of stem cells in ALS. Trying to explain that injecting random fat cells into your spine or CSF (for a fraction of 300K, but more than a car costs) is not the same as a high quality clinical trial with a placebo control was like trying to explain Celtic history to me.

Do you have a link on any later trials completing in dementia? Thanks.
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You are right, it seems right to try is already there- but it was approved last year only( March 2018). I agree it has many negative consequences.

www.fda.gov

Right to Try

Information for patients about Right to Try for requesting use of unapproved drugs.
www.fda.gov www.fda.gov

These are some recent trials, I think only 1 of these is actively recruiting at the moment.


ClinicalTrials.gov

www.clinicaltrials.gov www.clinicaltrials.gov

ClinicalTrials.gov

www.clinicaltrials.gov www.clinicaltrials.gov

ClinicalTrials.gov

www.clinicaltrials.gov www.clinicaltrials.gov

There is one basic science research in Nature that I saw, that shows light therapy reduces amyloid in mice. Obviously it means nothing for humans. I don't know if they have are taking it forward. I know there are few more for TMS.

www.nature.com

Gamma frequency entrainment attenuates amyloid load and modifies microglia - Nature

Mouse models of Alzheimer’s disease show reduced, behaviourally driven gamma oscillations before the onset of plaque formation or cognitive decline; driving neurons to oscillate at gamma frequency (40 Hz) reduces levels of amyloid-β peptides.
www.nature.com www.nature.com
 
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neglect said:
Wow. Just wow. Victory from the jaws of defeat. Those on the DSMC should seek employment elsewhere.

www.statnews.com

In shocking reversal, Biogen to submit experimental Alzheimer's drug for approval

In a shocking reversal, Biogen said that it would resurrect an Alzheimer’s drug that the company previously said had failed and will ask the Food and Drug Administration to approve it.
www.statnews.com www.statnews.com
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Not gonna lie - this one looks sketchy as hell. Small benefits that are statistically signifcant due to brute force numbers only after a post-hoc second crack... I want to believe, but think the public is likely to get carried away here for a very marginally effective drug.
 
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Thama said:
Not gonna lie - this one looks sketchy as hell. Small benefits that are statistically signifcant due to brute force numbers only after a post-hoc second crack... I want to believe, but think the public is likely to get carried away here for a very marginally effective drug.
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One trial was +, the other was neg. THey are submitting.

This is one of those situations where the more I get into it, the more I’m confused. Why did they limit the doses? Why stratify for APOE and limit their ability to get high dose (amendment)? Why do a middle dose at all? Why didn’t they allow ARIA-E to be redosed right away (waited for an amendment)? Why didn’t the DSMC have ALL the data (apparently they bundled the trials together)? Still, doesn’t this mean that in aggregate it was not useful? Why are they submitting with one positive trial? What acccounted for one +, one neg trial - they were identical?

One thing I’m sure about: the rumors of the death of amyloid were greatly exaggerated. Not sure Biogen proved it, or made the case it is worth the SE profile. But this is amazing news.

I agree with you 100% on the fears of data massage. I’m generally sympathetic to the pharma folks and their biostats people - they operate in a world of 100% transparency and high levels of scrutiny. But when they use words like reanalysis and different samples, I hear data manipulation.

I disagree this is a marginally effective drug IF it reduces progression on CDR by 23%. CDR is a stubborn metric, hard to move, preserved in early disease. So 23% reduction in progression on THAT is probably a 40% reduction on cognitive loss. That’s actually amazing. And remember, a trial that lasts a year or two meets a disease that lasts a decade or two. So over time, a 20% reduction in progression will create a huge swing. The question, of course, is: does the drug DO that? One trial showed it did, the other showed it didn’t.
 
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1. New fish oil drug for high triglycerides after cardiac event: Amarin’s fish-oil pill now available to millions more patients. But it’s still no Lipitor If the patients have elevated triglyceride levels, then they "must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease." Basically a good fit for stroke risk reduction.

Can we please stop talking about anti-plts now?

2. Another shocking approval in DMD: FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation
 
Good rundown on the new migraine options: New Hope for Migraine Sufferers

Not sure about that device though. Seems hard to blind and if it stings or tingles, it’ll give a huge placebo response.
 
Amygdarya said:
Hot off the press: the first drug for DMD just got approved - and look at the novel mechanism of action
FDA grants accelerated approval to first drug for Duchenne muscular dystrophy
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Update on Sarepta gene therapy. This isn't my field, but they just did another trial which was negative, with the younger subjects doing a bit better. This is not great for the current (very expensive) meds.

This may turn out to be a huge stain on Janet Woodcock and the FDA. It is really sad.
 
deathmerchant said:
Have you guys heard anything about this drug. looks promising.

Cassava Sciences’ Simufilam Improves Cognition and Behavior in Alzheimer’s Disease in Interim Analysis of Open-label Study | Cassava Sciences, Inc.

The Investor Relations website contains information about Cassava Sciences, Inc.'s business for stockholders, potential investors, and financial analysts.
www.cassavasciences.com www.cassavasciences.com
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Lol their stock is up 500% this week. Must be pretty solid data.
 
DrSatan said:
Lol their stock is up 500% this week. Must be pretty solid data.
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Yea I had been following their data, but I was looking at it with a grain of salt. Especially since we have had so many promising drugs in early phases that didn't work out in the end.
I think my skepticism prevented me from buying the stock. It's a shame, I couldn't make money on the stock despite being a neurologist lol.
 
This was open label, I don't understand why it is up 500%. We've seen this before.
 
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