Leucovorin now approved by FDA--will providers prescribe to ASD kids?

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I would love to hear other people experiences with Leucovorin

Because we’re doing science by anecdote now I guess.

Ever hear of case studies?

Case studies are not the kind of evidence the FDA generally uses to determine the safety and efficacy of a medication.

It's been used for 70 years. We already know it's safe.

They put cocaine in cough syrup for 70 yesrs

Do you not realize that Leucovorin continues to be used?

… in cancer. Stop lying.

You compared the safety record of Leucovorin to the safety record cocaine. I'm not the one being disingenuous. You can argue we don't know how effective it is for ASD, but we already know it's safe.

That wasn’t me but for the record cocaine actually does have medical uses right now (as a topical anesthesia).

So do you think that makes the safety record of cocaine similar to the safety record of Leucovorin? Otherwise, what's your point?

The point is that saying a drug is safe and effective for one indication says literally nothing at all about whether it is safe and effective for a wholly different indication. If you cannot grasp this entirely basic and fundamental point then you probably shouldn’t be in this discussion.

[Anonymous]

Anonymous wrote:

Anonymous wrote:It did not go through the standard FDA approval process. I know someone who works at FDA. You may want to see how a newly approved drug like this works on others before you jump into it.

Why? The new drugs are a crapshoot on efficacy and have substantially worse side effects and safety profiles. Even if it ultimately doesn't work, it's worth a try just as much as the more dangerous drugs that are on-label.

Also, understand that the processes for updating labels are different and much less rigorous than the processes for approving a drug. This drug has already been approved for 70 years. Off-label prescriptions are both allowed and common, although given the litigious nature of modern society, some doctors are very reluctant to prescribe off-label in kids until it becomes common practice by others. Updating the label will hopefully facilitate more data, and if informal experiences and future clinical trials don't pan out, they can remove it from the label. Since the drug isn't dangerous in any meaningful way, there's no harm.

So you just think the FDA approval process and research study methods are inapplicable to autism medications. OK!

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I'm really reticent to jump into this thread because it has turned so political and hostile, but I am surprised no one has mentioned Dr. Rosario Trifiletti in NJ. We came across his name on this board for PANS/PANDAS but he has spoken on the use of leucovorin for autism.

Due to the political angle, the thread drew a lot of attention from people without non-verbal kids. The reaction would have been completely different if Trump and RFK hadn't been involved in the press event.

The political angle IS the angle. And of course this is being touted for all kids with autism not just non-verbal kids. It’s not like it was some accident that Trumps and RFK Jr were involved.

Your just as guilty of the binary thinking by taking the opposite extreme- that we should withhold it from kids impacted by profound disabilities.

Trump and RFK didn't make up the 20 years of data or the two double-blind RCTs that have already been done. We wouldn't be having this discussion if this was a cancer drug, but a lot people don't particularly care about kids with profound autism because they don't directly see the impact that it has.

We wouldn’t be having this discussion about a cancer drug because Trump and RFK jr have not decided to upend and politicize cancer patients they way they have decided to do for autism. I think leucovorin and Tylenol should be studied and assessed using the NORMAL procedures already developed to ensure drugs are safe and effective. I think our kids deserve BETTER research not politicized publicity stunts.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:It did not go through the standard FDA approval process. I know someone who works at FDA. You may want to see how a newly approved drug like this works on others before you jump into it.

Why? The new drugs are a crapshoot on efficacy and have substantially worse side effects and safety profiles. Even if it ultimately doesn't work, it's worth a try just as much as the more dangerous drugs that are on-label.

Also, understand that the processes for updating labels are different and much less rigorous than the processes for approving a drug. This drug has already been approved for 70 years. Off-label prescriptions are both allowed and common, although given the litigious nature of modern society, some doctors are very reluctant to prescribe off-label in kids until it becomes common practice by others. Updating the label will hopefully facilitate more data, and if informal experiences and future clinical trials don't pan out, they can remove it from the label. Since the drug isn't dangerous in any meaningful way, there's no harm.

So you just think the FDA approval process and research study methods are inapplicable to autism medications. OK!

No, I'm saying the process for updating a label is always different than the process for initially approving a drug, whether it is for autism or any other condition.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I would love to hear other people experiences with Leucovorin

Because we’re doing science by anecdote now I guess.

Ever hear of case studies?

Case studies are not the kind of evidence the FDA generally uses to determine the safety and efficacy of a medication.

It's been used for 70 years. We already know it's safe.

They put cocaine in cough syrup for 70 yesrs

Do you not realize that Leucovorin continues to be used?

… in cancer. Stop lying.

You compared the safety record of Leucovorin to the safety record cocaine. I'm not the one being disingenuous. You can argue we don't know how effective it is for ASD, but we already know it's safe.

That wasn’t me but for the record cocaine actually does have medical uses right now (as a topical anesthesia).

So do you think that makes the safety record of cocaine similar to the safety record of Leucovorin? Otherwise, what's your point?

The point is that saying a drug is safe and effective for one indication says literally nothing at all about whether it is safe and effective for a wholly different indication. If you cannot grasp this entirely basic and fundamental point then you probably shouldn’t be in this discussion.

Again, we don't know it is effective, but we do know it is safe. Safe doesn't necessarily mean there's absolutely no possibility of risk. We approve drugs and other substances while there is still small potential for risks, with after-market monitoring being used to catch exceptional events. Leucovorin has the substantial benefit over new drugs in that we already have a long safety history with the drug.

It's ridiculous to suggest it isn't safe. Particularly given the risks of other things that are available and generally accepted as safe- including prescription medications, supplements, and food ingredients.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:It did not go through the standard FDA approval process. I know someone who works at FDA. You may want to see how a newly approved drug like this works on others before you jump into it.

Why? The new drugs are a crapshoot on efficacy and have substantially worse side effects and safety profiles. Even if it ultimately doesn't work, it's worth a try just as much as the more dangerous drugs that are on-label.

Also, understand that the processes for updating labels are different and much less rigorous than the processes for approving a drug. This drug has already been approved for 70 years. Off-label prescriptions are both allowed and common, although given the litigious nature of modern society, some doctors are very reluctant to prescribe off-label in kids until it becomes common practice by others. Updating the label will hopefully facilitate more data, and if informal experiences and future clinical trials don't pan out, they can remove it from the label. Since the drug isn't dangerous in any meaningful way, there's no harm.

So you just think the FDA approval process and research study methods are inapplicable to autism medications. OK!

No, I'm saying the process for updating a label is always different than the process for initially approving a drug, whether it is for autism or any other condition.

Can you provide support for that assertion?

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I would love to hear other people experiences with Leucovorin

Because we’re doing science by anecdote now I guess.

Ever hear of case studies?

Case studies are not the kind of evidence the FDA generally uses to determine the safety and efficacy of a medication.

It's been used for 70 years. We already know it's safe.

They put cocaine in cough syrup for 70 yesrs

Do you not realize that Leucovorin continues to be used?

… in cancer. Stop lying.

You compared the safety record of Leucovorin to the safety record cocaine. I'm not the one being disingenuous. You can argue we don't know how effective it is for ASD, but we already know it's safe.

That wasn’t me but for the record cocaine actually does have medical uses right now (as a topical anesthesia).

So do you think that makes the safety record of cocaine similar to the safety record of Leucovorin? Otherwise, what's your point?

The point is that saying a drug is safe and effective for one indication says literally nothing at all about whether it is safe and effective for a wholly different indication. If you cannot grasp this entirely basic and fundamental point then you probably shouldn’t be in this discussion.

Again, we don't know it is effective, but we do know it is safe. Safe doesn't necessarily mean there's absolutely no possibility of risk. We approve drugs and other substances while there is still small potential for risks, with after-market monitoring being used to catch exceptional events. Leucovorin has the substantial benefit over new drugs in that we already have a long safety history with the drug.

It's ridiculous to suggest it isn't safe. Particularly given the risks of other things that are available and generally accepted as safe- including prescription medications, supplements, and food ingredients.

No we do NOT KNOW if it is safe in the population it is now being touted for. It has side effects that absolutely could be more unsafe for kids with autism vs adults with cancer. Specifically, seizures and aggression are known side effects and these are bigger concerns for autism. AND there is also research connecting too much prenatal folate to autism.

We.do.not.know.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I'm really reticent to jump into this thread because it has turned so political and hostile, but I am surprised no one has mentioned Dr. Rosario Trifiletti in NJ. We came across his name on this board for PANS/PANDAS but he has spoken on the use of leucovorin for autism.

Due to the political angle, the thread drew a lot of attention from people without non-verbal kids. The reaction would have been completely different if Trump and RFK hadn't been involved in the press event.

The political angle IS the angle. And of course this is being touted for all kids with autism not just non-verbal kids. It’s not like it was some accident that Trumps and RFK Jr were involved.

Your just as guilty of the binary thinking by taking the opposite extreme- that we should withhold it from kids impacted by profound disabilities.

Trump and RFK didn't make up the 20 years of data or the two double-blind RCTs that have already been done. We wouldn't be having this discussion if this was a cancer drug, but a lot people don't particularly care about kids with profound autism because they don't directly see the impact that it has.

We wouldn’t be having this discussion about a cancer drug because Trump and RFK jr have not decided to upend and politicize cancer patients they way they have decided to do for autism. I think leucovorin and Tylenol should be studied and assessed using the NORMAL procedures already developed to ensure drugs are safe and effective. I think our kids deserve BETTER research not politicized publicity stunts.

Other administrations likely wouldn't have the narcissistic personalities insisting on making it a political event, but there certainly would have been public and political pressure to facilitate use.

You see this in more extreme cases- people pressuring the FDA to open up drugs that *don't* have established safety records for compassionate use.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I'm really reticent to jump into this thread because it has turned so political and hostile, but I am surprised no one has mentioned Dr. Rosario Trifiletti in NJ. We came across his name on this board for PANS/PANDAS but he has spoken on the use of leucovorin for autism.

Due to the political angle, the thread drew a lot of attention from people without non-verbal kids. The reaction would have been completely different if Trump and RFK hadn't been involved in the press event.

The political angle IS the angle. And of course this is being touted for all kids with autism not just non-verbal kids. It’s not like it was some accident that Trumps and RFK Jr were involved.

Your just as guilty of the binary thinking by taking the opposite extreme- that we should withhold it from kids impacted by profound disabilities.

Trump and RFK didn't make up the 20 years of data or the two double-blind RCTs that have already been done. We wouldn't be having this discussion if this was a cancer drug, but a lot people don't particularly care about kids with profound autism because they don't directly see the impact that it has.

We wouldn’t be having this discussion about a cancer drug because Trump and RFK jr have not decided to upend and politicize cancer patients they way they have decided to do for autism. I think leucovorin and Tylenol should be studied and assessed using the NORMAL procedures already developed to ensure drugs are safe and effective. I think our kids deserve BETTER research not politicized publicity stunts.

Other administrations likely wouldn't have the narcissistic personalities insisting on making it a political event, but there certainly would have been public and political pressure to facilitate use.

You see this in more extreme cases- people pressuring the FDA to open up drugs that *don't* have established safety records for compassionate use.

They did a lot more here than compassionate use (which is generally on an individual level and a last ditch effort when the person is dying). Totally different from rushing a med for otherwise physically healthy *children*.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I would love to hear other people experiences with Leucovorin

Because we’re doing science by anecdote now I guess.

Ever hear of case studies?

Case studies are not the kind of evidence the FDA generally uses to determine the safety and efficacy of a medication.

It's been used for 70 years. We already know it's safe.

They put cocaine in cough syrup for 70 yesrs

Do you not realize that Leucovorin continues to be used?

… in cancer. Stop lying.

You compared the safety record of Leucovorin to the safety record cocaine. I'm not the one being disingenuous. You can argue we don't know how effective it is for ASD, but we already know it's safe.

That wasn’t me but for the record cocaine actually does have medical uses right now (as a topical anesthesia).

So do you think that makes the safety record of cocaine similar to the safety record of Leucovorin? Otherwise, what's your point?

The point is that saying a drug is safe and effective for one indication says literally nothing at all about whether it is safe and effective for a wholly different indication. If you cannot grasp this entirely basic and fundamental point then you probably shouldn’t be in this discussion.

Again, we don't know it is effective, but we do know it is safe. Safe doesn't necessarily mean there's absolutely no possibility of risk. We approve drugs and other substances while there is still small potential for risks, with after-market monitoring being used to catch exceptional events. Leucovorin has the substantial benefit over new drugs in that we already have a long safety history with the drug.

It's ridiculous to suggest it isn't safe. Particularly given the risks of other things that are available and generally accepted as safe- including prescription medications, supplements, and food ingredients.

No we do NOT KNOW if it is safe in the population it is now being touted for. It has side effects that absolutely could be more unsafe for kids with autism vs adults with cancer. Specifically, seizures and aggression are known side effects and these are bigger concerns for autism. AND there is also research connecting too much prenatal folate to autism.

We.do.not.know.

On that basis, almost nothing is "safe" for use in kids with autism because these kids often aren't part of clinical trial populations. Since you're saying a safety record for the general population shouldn't be used to infer safety in a subpopulation.

Again, you're being disingenuous. This is done all the time. Heck, look how often drugs get prescribed off-label because of how hard it is to run trials (particularly in kids).

Frankly, off-label use here would seem to be the right move, too. But clearly something has happened over the last 10 years, and particularly with this drug, to make some doctors lose their ability to weigh risks and potential benefits rationally and in the best interest of their patients. Why are they treating it so differently than something like guanfacine, another drug with a long safety history that is frequently prescribed for young kids with autism presenting with aggressive or self-injurious behaviors, despite those being off-label.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I would love to hear other people experiences with Leucovorin

Because we’re doing science by anecdote now I guess.

Ever hear of case studies?

Case studies are not the kind of evidence the FDA generally uses to determine the safety and efficacy of a medication.

It's been used for 70 years. We already know it's safe.

They put cocaine in cough syrup for 70 yesrs

Do you not realize that Leucovorin continues to be used?

… in cancer. Stop lying.

You compared the safety record of Leucovorin to the safety record cocaine. I'm not the one being disingenuous. You can argue we don't know how effective it is for ASD, but we already know it's safe.

That wasn’t me but for the record cocaine actually does have medical uses right now (as a topical anesthesia).

So do you think that makes the safety record of cocaine similar to the safety record of Leucovorin? Otherwise, what's your point?

The point is that saying a drug is safe and effective for one indication says literally nothing at all about whether it is safe and effective for a wholly different indication. If you cannot grasp this entirely basic and fundamental point then you probably shouldn’t be in this discussion.

Again, we don't know it is effective, but we do know it is safe. Safe doesn't necessarily mean there's absolutely no possibility of risk. We approve drugs and other substances while there is still small potential for risks, with after-market monitoring being used to catch exceptional events. Leucovorin has the substantial benefit over new drugs in that we already have a long safety history with the drug.

It's ridiculous to suggest it isn't safe. Particularly given the risks of other things that are available and generally accepted as safe- including prescription medications, supplements, and food ingredients.

No we do NOT KNOW if it is safe in the population it is now being touted for. It has side effects that absolutely could be more unsafe for kids with autism vs adults with cancer. Specifically, seizures and aggression are known side effects and these are bigger concerns for autism. AND there is also research connecting too much prenatal folate to autism.

We.do.not.know.

On that basis, almost nothing is "safe" for use in kids with autism because these kids often aren't part of clinical trial populations. Since you're saying a safety record for the general population shouldn't be used to infer safety in a subpopulation.

Again, you're being disingenuous. This is done all the time. Heck, look how often drugs get prescribed off-label because of how hard it is to run trials (particularly in kids).

Frankly, off-label use here would seem to be the right move, too. But clearly something has happened over the last 10 years, and particularly with this drug, to make some doctors lose their ability to weigh risks and potential benefits rationally and in the best interest of their patients. Why are they treating it so differently than something like guanfacine, another drug with a long safety history that is frequently prescribed for young kids with autism presenting with aggressive or self-injurious behaviors, despite those being off-label.

This is a drug being specific touted FOR kids with autism. So yes, it is relevant to ask how it specifically affects them. And of course the whole point is that this went far beyond off-label use, departing from how additional uses are added and making overblown claims not supported by the evidence.

More importantly while it may be an understandable stance for an individual parent with their doctor to use a med off-label under the background of slow or nonexistent goldstar research - that is far different from the people *literally in charge of the research* upending it. Trump and RFK jr are pretending like they aren’t the ones with the power to actually do the research our kids deserve. because they don’t actually care about our kids. They care about politics.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I'm really reticent to jump into this thread because it has turned so political and hostile, but I am surprised no one has mentioned Dr. Rosario Trifiletti in NJ. We came across his name on this board for PANS/PANDAS but he has spoken on the use of leucovorin for autism.

Due to the political angle, the thread drew a lot of attention from people without non-verbal kids. The reaction would have been completely different if Trump and RFK hadn't been involved in the press event.

The political angle IS the angle. And of course this is being touted for all kids with autism not just non-verbal kids. It’s not like it was some accident that Trumps and RFK Jr were involved.

Your just as guilty of the binary thinking by taking the opposite extreme- that we should withhold it from kids impacted by profound disabilities.

Trump and RFK didn't make up the 20 years of data or the two double-blind RCTs that have already been done. We wouldn't be having this discussion if this was a cancer drug, but a lot people don't particularly care about kids with profound autism because they don't directly see the impact that it has.

We wouldn’t be having this discussion about a cancer drug because Trump and RFK jr have not decided to upend and politicize cancer patients they way they have decided to do for autism. I think leucovorin and Tylenol should be studied and assessed using the NORMAL procedures already developed to ensure drugs are safe and effective. I think our kids deserve BETTER research not politicized publicity stunts.

Other administrations likely wouldn't have the narcissistic personalities insisting on making it a political event, but there certainly would have been public and political pressure to facilitate use.

You see this in more extreme cases- people pressuring the FDA to open up drugs that *don't* have established safety records for compassionate use.

They did a lot more here than compassionate use (which is generally on an individual level and a last ditch effort when the person is dying). Totally different from rushing a med for otherwise physically healthy *children*.

These aren't healthy children. They are children with a profound disabilities that have a short window of time during childhood to reach important developmental milestones.

Like "last ditch efforts" for dying cancer patients, this is worth substantial risk. And that's reflected by the side effects of the drugs approved and commonly prescribed for ASD, despite not being particularly effective.

In comparison, Leucovorin has a remarkable safety profile. Adjusting expectations/demands for efficacy accordingly, there's no rational cost-benefit analysis that wouldn't lead you to conclude it is worth a shot in kids profoundly impacted by autism.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I would love to hear other people experiences with Leucovorin

Because we’re doing science by anecdote now I guess.

Ever hear of case studies?

Case studies are not the kind of evidence the FDA generally uses to determine the safety and efficacy of a medication.

It's been used for 70 years. We already know it's safe.

They put cocaine in cough syrup for 70 yesrs

Do you not realize that Leucovorin continues to be used?

… in cancer. Stop lying.

You compared the safety record of Leucovorin to the safety record cocaine. I'm not the one being disingenuous. You can argue we don't know how effective it is for ASD, but we already know it's safe.

That wasn’t me but for the record cocaine actually does have medical uses right now (as a topical anesthesia).

So do you think that makes the safety record of cocaine similar to the safety record of Leucovorin? Otherwise, what's your point?

The point is that saying a drug is safe and effective for one indication says literally nothing at all about whether it is safe and effective for a wholly different indication. If you cannot grasp this entirely basic and fundamental point then you probably shouldn’t be in this discussion.

Again, we don't know it is effective, but we do know it is safe. Safe doesn't necessarily mean there's absolutely no possibility of risk. We approve drugs and other substances while there is still small potential for risks, with after-market monitoring being used to catch exceptional events. Leucovorin has the substantial benefit over new drugs in that we already have a long safety history with the drug.

It's ridiculous to suggest it isn't safe. Particularly given the risks of other things that are available and generally accepted as safe- including prescription medications, supplements, and food ingredients.

No we do NOT KNOW if it is safe in the population it is now being touted for. It has side effects that absolutely could be more unsafe for kids with autism vs adults with cancer. Specifically, seizures and aggression are known side effects and these are bigger concerns for autism. AND there is also research connecting too much prenatal folate to autism.

We.do.not.know.

On that basis, almost nothing is "safe" for use in kids with autism because these kids often aren't part of clinical trial populations. Since you're saying a safety record for the general population shouldn't be used to infer safety in a subpopulation.

Again, you're being disingenuous. This is done all the time. Heck, look how often drugs get prescribed off-label because of how hard it is to run trials (particularly in kids).

Frankly, off-label use here would seem to be the right move, too. But clearly something has happened over the last 10 years, and particularly with this drug, to make some doctors lose their ability to weigh risks and potential benefits rationally and in the best interest of their patients. Why are they treating it so differently than something like guanfacine, another drug with a long safety history that is frequently prescribed for young kids with autism presenting with aggressive or self-injurious behaviors, despite those being off-label.

This is a drug being specific touted FOR kids with autism. So yes, it is relevant to ask how it specifically affects them. And of course the whole point is that this went far beyond off-label use, departing from how additional uses are added and making overblown claims not supported by the evidence.

More importantly while it may be an understandable stance for an individual parent with their doctor to use a med off-label under the background of slow or nonexistent goldstar research - that is far different from the people *literally in charge of the research* upending it. Trump and RFK jr are pretending like they aren’t the ones with the power to actually do the research our kids deserve. because they don’t actually care about our kids. They care about politics.

The reality of modern medicine is that doctors now want top-cover for prescribing medications- particularly in kids. Guanfacine was able to become common before those pressures shifted.

The only way we can really provide that top-cover is adding it to the label. If a doctor isn't satisfied there's a good risk-benefit tradeoff based on the available data, then they obviously don't need to prescribe it. It's not like they're trying to make this available OTC without a prescription.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I'm really reticent to jump into this thread because it has turned so political and hostile, but I am surprised no one has mentioned Dr. Rosario Trifiletti in NJ. We came across his name on this board for PANS/PANDAS but he has spoken on the use of leucovorin for autism.

Due to the political angle, the thread drew a lot of attention from people without non-verbal kids. The reaction would have been completely different if Trump and RFK hadn't been involved in the press event.

The political angle IS the angle. And of course this is being touted for all kids with autism not just non-verbal kids. It’s not like it was some accident that Trumps and RFK Jr were involved.

Your just as guilty of the binary thinking by taking the opposite extreme- that we should withhold it from kids impacted by profound disabilities.

Trump and RFK didn't make up the 20 years of data or the two double-blind RCTs that have already been done. We wouldn't be having this discussion if this was a cancer drug, but a lot people don't particularly care about kids with profound autism because they don't directly see the impact that it has.

We wouldn’t be having this discussion about a cancer drug because Trump and RFK jr have not decided to upend and politicize cancer patients they way they have decided to do for autism. I think leucovorin and Tylenol should be studied and assessed using the NORMAL procedures already developed to ensure drugs are safe and effective. I think our kids deserve BETTER research not politicized publicity stunts.

Other administrations likely wouldn't have the narcissistic personalities insisting on making it a political event, but there certainly would have been public and political pressure to facilitate use.

You see this in more extreme cases- people pressuring the FDA to open up drugs that *don't* have established safety records for compassionate use.

They did a lot more here than compassionate use (which is generally on an individual level and a last ditch effort when the person is dying). Totally different from rushing a med for otherwise physically healthy *children*.

These aren't healthy children. They are children with a profound disabilities that have a short window of time during childhood to reach important developmental milestones.

Like "last ditch efforts" for dying cancer patients, this is worth substantial risk. And that's reflected by the side effects of the drugs approved and commonly prescribed for ASD, despite not being particularly effective.

In comparison, Leucovorin has a remarkable safety profile. Adjusting expectations/demands for efficacy accordingly, there's no rational cost-benefit analysis that wouldn't lead you to conclude it is worth a shot in kids profoundly impacted by autism.

I thought we were past the point where we disregarded any harms of autism therapy because autism is uniquely bad. Or are we back to bleach, chelation, shocks and Lovaas abuse? And the label is for autism not “severe autism.”

Also you need to stop comparing leucovorin to Abilify and Risperdal. Those meds are approved for specific severe autism symptoms (aggression) where the benefits may outweigh the risks. They are not for the core autism symptoms that Leucovorin is being touted for. They have nothing to do with each other.

Our kids deserve the best in research not incomplete research pushed by quacks.

[Anonymous]

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:

Anonymous wrote:I would love to hear other people experiences with Leucovorin

Because we’re doing science by anecdote now I guess.

Ever hear of case studies?

Case studies are not the kind of evidence the FDA generally uses to determine the safety and efficacy of a medication.

It's been used for 70 years. We already know it's safe.

They put cocaine in cough syrup for 70 yesrs

Do you not realize that Leucovorin continues to be used?

… in cancer. Stop lying.

You compared the safety record of Leucovorin to the safety record cocaine. I'm not the one being disingenuous. You can argue we don't know how effective it is for ASD, but we already know it's safe.

That wasn’t me but for the record cocaine actually does have medical uses right now (as a topical anesthesia).

So do you think that makes the safety record of cocaine similar to the safety record of Leucovorin? Otherwise, what's your point?

The point is that saying a drug is safe and effective for one indication says literally nothing at all about whether it is safe and effective for a wholly different indication. If you cannot grasp this entirely basic and fundamental point then you probably shouldn’t be in this discussion.

Again, we don't know it is effective, but we do know it is safe. Safe doesn't necessarily mean there's absolutely no possibility of risk. We approve drugs and other substances while there is still small potential for risks, with after-market monitoring being used to catch exceptional events. Leucovorin has the substantial benefit over new drugs in that we already have a long safety history with the drug.

It's ridiculous to suggest it isn't safe. Particularly given the risks of other things that are available and generally accepted as safe- including prescription medications, supplements, and food ingredients.

No we do NOT KNOW if it is safe in the population it is now being touted for. It has side effects that absolutely could be more unsafe for kids with autism vs adults with cancer. Specifically, seizures and aggression are known side effects and these are bigger concerns for autism. AND there is also research connecting too much prenatal folate to autism.

We.do.not.know.

On that basis, almost nothing is "safe" for use in kids with autism because these kids often aren't part of clinical trial populations. Since you're saying a safety record for the general population shouldn't be used to infer safety in a subpopulation.

Again, you're being disingenuous. This is done all the time. Heck, look how often drugs get prescribed off-label because of how hard it is to run trials (particularly in kids).

Frankly, off-label use here would seem to be the right move, too. But clearly something has happened over the last 10 years, and particularly with this drug, to make some doctors lose their ability to weigh risks and potential benefits rationally and in the best interest of their patients. Why are they treating it so differently than something like guanfacine, another drug with a long safety history that is frequently prescribed for young kids with autism presenting with aggressive or self-injurious behaviors, despite those being off-label.

This is a drug being specific touted FOR kids with autism. So yes, it is relevant to ask how it specifically affects them. And of course the whole point is that this went far beyond off-label use, departing from how additional uses are added and making overblown claims not supported by the evidence.

More importantly while it may be an understandable stance for an individual parent with their doctor to use a med off-label under the background of slow or nonexistent goldstar research - that is far different from the people *literally in charge of the research* upending it. Trump and RFK jr are pretending like they aren’t the ones with the power to actually do the research our kids deserve. because they don’t actually care about our kids. They care about politics.

The reality of modern medicine is that doctors now want top-cover for prescribing medications- particularly in kids. Guanfacine was able to become common before those pressures shifted.

The only way we can really provide that top-cover is adding it to the label. If a doctor isn't satisfied there's a good risk-benefit tradeoff based on the available data, then they obviously don't need to prescribe it. It's not like they're trying to make this available OTC without a prescription.

Forgive me if I expect more from the people and institutions literally in charge of making the determinations of what can be prescribed safely. That’s the insane part. You’re saying that because our research and prescribing systems are not ideal that the people in charge of our research and prescribing systems have no other choice but to shrug their shoulders and say “the system sucks, take whatever”?