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    I haven’t seen too much mention of this (might have missed it, I’m new here), so wanted to put it out there in case it’s useful. In researching melanoma treatments, it seems like Ipi is the current standard of care, and outside of that there are the PD-1 blockers that are coming but still in trial. But I found one study that found that using GM-CSF (Leukine) in combination with Ipi seemed to increase the efficacy of the Ipi, while reducing the side effects. GM-CSF has already been approved for quite a while, and is typically used for patients after chemo has beat down their immune system… my understanding is it promotes white blood cell growth.

    Here’s one link. Look under the “Immunotherapy” section here, about halfway down the page:


    In the CTLA-4 section talking about Ipi, it says this:

    “A recent early study found that combining ipilimumab with another immunotherapy drug known as GM-CSF helped patients with advanced melanoma live longer than those who got just ipilimumab alone. The people who got the combination also seemed to have fewer serious side effects.”

    One study result:


    Couple more links:



    Since GM-CSF is already approved, docs can use it at their discretion. I mentioned the above to my dad’s oncologist, and she was willing to give it a try. The other question is insurance coverage. Since it’s not “standard of care” for melanoma, some insurance may balk. Fortunately my dad’s insurance covered it. If it’s not covered, fortunately it’s still at least a lot cheaper than Ipi, and could probably be found online. I didn’t look too hard but found this:


    My dad is getting 250 mcg injections daily for 14 days starting with each Ipi infusion.

    Hope this helpful to someone… and hopefully my dad.


    My wife used the same combo of drugs. 😥

    Catherine Poole

    This is interesting. Leukine was once thought to be a great therapy for melanoma until phase III trials found it ineffective. However, in combination, it might be reincarnated. I know the Northern Ca. Melanoma Center was very interested in Leukine and had studies on it. There was a few instances where they thought it could cause leukemia as an adverse event. I will try to find that. But let us know how it works for your father.


    Yes this IS interesting.

    There were early trials by, I believe, the NIH where IPI and IL2 were sequenced together as well since IL2 also stimulates, in part, the production of white cells. However, as I recall, the ORRs were similar to or less than the response rates of the two therapies combined and there was no further investigation.

    Subsequent to those trials there was some talk that it may actually be better to apply IL2 a month or so after the last IPI dose, instead of concurrently. The thinking was that at that point the production of T regulatory cells would be well surpressed by the IPI and that a greater portion of the IL2 would be used by the body to produce more Killer cells, thus super-charging the immune system even further over that of IPI alone. However, I can’t find any body of data where that was actually tried in a controlled manner. This was indeed our back up plan with Rachel if she could not get into the sequenced IPI/PD1 trial at MSK, which she did not due to brain mets. However, by that time the disease had compromised her lung function so much that IL2 was taken off of the table.

    I wonder if the same thinking concerning the sequencing, if correct to begin with, could be applied to the use of Leukine as well.

    Maybe someone on the science board could comment on that?



    Dr. Weber recently said similar, that GM-CSF had not been proven to do much alone. However, T-VEC he said was giving good results, which is a virus modified such that it secretes GM-CSF inside cancer cells.



    How are you doing? I know you probably get asked that a lot but really, how are you doing?


    Catherine Poole

    Our scientific board can only speculate on something that hasn’t been through the trials, so I can’t really ask them about this. I think the road to interesting therapies should be lined with drugs that don’t cause ill effects. Leukine is a perfect example as well as PD1 of drugs that don’t take a toll on the body’s organs usually. IL2 on the other hand really can cause substantial damage to body organs so I tend to lean towards the others. I imagine we will see a lot of combinations in the works, but we won’t be able to truly trust them until they’ve been through the long and arduous clinical trial process of which, you, the patients are crucial! PS I would look to more use of PD1 in combination than IPI because of the side effect profile too.



    I did GM-CSF injections in 2005/2006 after two years of dealing with melanoma mets in different organs (lung, spine, brain and small bowels). At that time I was stage IV NED and did not qualify for any of the “few” trials for stage IV NED.

    My onco suggested GM-CSF and I decided to give it a try. I believe that GM-CSF was the kick that my immune system needed, I have been clear of melanoma since I started the Leukine.

    Best of luck to your dad, I hope it also works for him.

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