What's the most successful immunotherapy study so far?


This may just be a question for Elodie, since I’m not sure how many people have participated in or followed these studies. UCLA is talking to my son about participating in an immunotherapy study there. In the previous study of six, there was one complete remission, one stayed the same, and four participants got worse. I know there are other immunotherapy trials going on elsewhere but which has had the best outcome so far? Before he’s even accepted to this one, he will need to have the right kind of immune system (are there different kinds?) and express the right marker. Any thoughts or info on this would be greatly appreciated.


The most successful so far has been the engineered T-cells from Adaptimmune. The trial started several years ago at NIH in Bethesda, Maryland. It looks like the trial has been extended to different locations, including California:

Unfortunately, the inclusion criteria is rather restrictive. It is my impression that the majority of members who tried to join this trial were not eligible…
What kind of immunotherapy trial is being offered at UCLA? Is it an inhibitor of some type?


Thanks Elodie. I see they’re doing that study at City of Hope. We have to see if he fits the criteria but it definitely sounds intriguing. The UCLA trial is an NY-ESO-1-specific T-cell receptor and dendritic cell vaccination in combination with ipilimumab (Yervoy) in patients with advanced sarcoma.

My son’s cancer has returned for the second time in his neck, and is wrapped around his vertebral artery so it’s impossible to get margins. The other day his neurosurgeon implied that his disease is now chronic, in the sense that it will return and they will remove and treat it each time, awaiting new technologies or breakthroughs that can get rid of it. Does that sound plausible? Do you have any suggestions as we move forward to find the right treatment? He’s having surgery next week. Should we get a second opinion before choosing a therapy? Is it worth traveling to MD Anderson?

Thanks so much,


A second opinion does sound like a good idea, especially in a case like this. At least, you will know you looked at all options.

You may want to watch Dr. Mackall’s presentation on this video starting at 4:47:

She is presenting some results from the engineered T-cells trial at NIH…

Regarding the UCLA trial, I couldn’t find it on clinicaltrials.gov but I found this one which is similar but uses a PD1 inhibitor instead of CTLA-4:
The concept is interesting. The addition of a PD-1 or CTLA-4 inhibitor to the engineered T-cells treatment makes sense. The question is whether their T-cells are from Adaptimmune or of the same quality…


Hi Elodie:

We talked to my son’s doctor and it turned out he didn’t express the HLA-201, 204 or 206, but rather 2402 and 2601, so he wasn’t eligible for the trial. They did tell us about a trial Dr. Chawla was doing, so we went to see him and he told us about two possible trial that might work but the last one might be hard to get in.

They are:

Trial of CMB305 and Atezolizumab in Patients With Sarcoma (IMDZ-C232)

Basket Study of Entrectinib (RXDX-101) for the Treatment of Patients With Solid Tumors Harboring NTRK1/2/3, ROS1, or ALK Gene Rearrangements (Fusions) (STARTRK-2)

The second one was supposed to be very successful but difficult to qualify for.

My son was supposed to get surgery tomorrow to take out the tumor in his neck, but they’re pushing it back a couple of weeks while testing his tumor from 2014 to see if it expresses NY-ES0-1. If he does qualify for one of these trials, they will then debulk the tumor, leaving in 1 cm of it, so they can test whether the treatment is working or not. I hope we’re doing the right thing. I can’t help but wonder if we should just go ahead and take this one out, and test it to see which trials it might qualify for. That way, if it grows back and then we’d be armed with more information and options.

In the meantime, I’m researching other studies that are further along and seem to have a successful track record, so I can know which markers or criteria they’re looking for. Are there any others you are familiar with? I called MD Anderson regarding a second opinion and am waiting to hear back. Do you know if there are any trials going on similar to the one in the video you provided? My son is 25 so he could still fit in an AYA trial.



I haven’t heard of anything exciting for quite a while :frowning:
There is a study at Dana Farber in Boston that does tumor profiling in an attempt to individualize treatment. I don’t know if anybody has benefited so far:

Let us know how the testing goes!


Do you know of any CARs T-cell trials for sarcoma…like the one the woman talks about in the video?


The only CAR T-cell trials that I am aware of are for GD2 positive tumors:

By the way, there is a trial in Seattle for patients with HLA 2402: