Cure Alveolar Soft Part Sarcoma International (iCureASPS)

We are in need of some advice. TJ found another lump almost directly behind the first one on the top of his head. I emailed the Doctors this morning and they said to watch it for 2 weeks and see what happens. I asked if this could be swelling due to the gamma knife or could it be another tumor. They said swelling was a possibility but they do not treat it unless Tom is having symptoms. I do not know how we are suppose to wait 2 weeks, fearing that their could be another tumor growing. Can anyone put our mind at ease or let us know if there is anything else we could be doing. He has not had any unusual headaches or other issues regarding the brain. He has had an increased amount of leg pain due to the spot that was treated on the l4 again and we continue to hope and pray that that starts to lessen as it is can be unbearable.

hugs and much love to you all,
Kathy

Kathy - the ultrasound scan is probably easy to get prescr. by the family dr on a ground that you suspect that there is some post treatment complication. It works well to see if the mass is a fluid or it is a vascular formation (which all the ASPS tumors are). What dr did you e-mail to?

I emailed the neurosurgeon that was the head of the team that treated this most recent met. So I will call his local Doctor and see about an ultrasound. I just hate the idea of sitting and waiting...Thank you so much for you fast response!

Dear Kathy,
I agree with Olga that some type of scan such as an ultrasound needs to be done as soon as possible to determine the nature of the new lump. I would definitely NOT wait for the lump to become symptomatic and I think that it is irresponsible of the neurosurgeon to advise you to wait 2 weeks. Are you able to determine if the new lump feels the same as the Gamma Knifed tumor and if it is soft or hard? I am so very sorry for this new concern and Hope that you can obtain an ultrasound and some definitive answers very soon. I am holding very tight to Hope that it is just post Gamma Knife swelling that will dissipate and disappear, and will be anxiously awaiting an update when your time and the situation allows. Take care dear Kathy.
Sharing your concern with deepest caring, healing wishes for dear Tom, love, and continued Hope,
Bonni

They are telling us that an ultrasound cannot be done on the brain. I don't understand why they do mot seem more concerned!

Kathy,
They're right, ultrasound cannot detect the brain mets. But the lump is in scalp, right? Then the ultrasound is applicable.

Lynette

Lynette, that is what I thought. I will be calling again in the morning!

Dear Kathy,
I agree with Lynette, and strongly encourage you to insist that the doctor(s) schedule some kind of other scan if they continue to be negative/uncooperative about the ultrasound. It seems very irresponsible for them to be so nonchalant and resistant about addressing a diagnostic evaluation of the new lump, and I am so sorry that they are putting you and Tom through so much additional and unnecessary stress!! If you don't receive a positive response and action tomorrow, I think that you should find a different more responsive, compassionate, and caring doctor as soon as possible.
Sharing your frustration and reaching out across the miles with special hugs, caring thoughts, healing wishes for Tom, much love, and continued Hope,
Bonni

So the "new spot" we felt is no longer there and the spot they treated feels and looks smaller - we are hopeful! We are meeting a new oncologist on December 6th at UPMC. His name is Dr. Tawbi. Dr. Choudry (he did the bowel surgery) and Dr. Gerzten, (radio surgery), highly recommend him. We met him once years ago. It didn't feel like a fit at the time so we are hoping it will be now. The pain Tom is experiencing ever since the L-4 was treated again has been very intense and hard to manage. We have another appointment with Pain management on Monday.

I have a question for all of you. What do you know about/think about the immunotherapy. I found a clinical trial at MD Anderson. I lean on you all so much for advice. I often feel like I a swimming upstream and that I have do idea what I am doing. We are terrified at the rate this has progressed for Tom and we are not sure what to do next. I want to head into this appointment with the new oncologist with as much current information as possible. Oh how I wish I could take you all with us.

Here is the information I found:

***Trial Summary***
Short Title
iC9-GD2-CAR-VZV-CTLs/Refractory or Metastatic GD2-positive Sarcoma/VEGAS

Patient Abstract
The purpose of this study is to find the largest safe dose of GD2-T cells (also called iC9-GD2-CAR-VZV-CTLs), and additionally to evaluate if a VZV vaccine can improve the expansion and persistence of infused T cells, to learn what the side effects are, and to see whether this therapy might help patients with advanced sarcomas. Because there is no standard treatment for recurrent/refractory sarcomas at this time or because the currently used treatments do not work fully in all cases, patients are being asked to volunteer to take part in a gene transfer research study using special immune cells.

The body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells. Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise, but have not been strong enough to cure most patients.

Investigators have found from previous research that a new gene can be put into T cells that will make them recognize cancer cells and kill them. Investigators now want to see if a new gene can be put in these cells that will let the T cells recognize and kill sarcoma cells. The new gene is called a chimeric antigen receptor (CAR) and consists of an antibody called 14g2a that recognizes GD2, a protein that is found on sarcoma cells (GD2-CAR). In addition, it contains parts of the CD28 and OX40 genes which can stimulate T cells to make them live longer.

Investigators have found that CAR-T cells can kill some of the tumor, but they don't last very long in the body and so the tumor eventually comes back. T cells that recognize the virus that causes chicken pox, varicella zoster virus (VZV), remain in the bloodstream for many years especially if they are stimulated or boosted by the VZV vaccine. Investigators will therefore insert the GD2-CAR gene into T cells that recognize VZV. These cells are called iC9-GD2-CAR-VZV-specific T cells but are referred to as GD2-T cells for simplicity.

This study consists of two groups. The first group of patients will receive GD2-T cells followed six weeks later by vaccination with the VZV vaccine. The second group will receive GD2-T cells, the vaccine, and a second dose of GD2-T cells two days after the vaccine. We want to see whether giving a second dose of GD2-T cells right after the vaccine helps them to be more effective.

Terms
This is an abstract courtesy of ClinicalTrials.gov, a service of the US National Institute of Health Developed by the National Library of Medicine. ClinicalTrials.gov provides regularly updated information about federally and privately supported clinical research in human volunteers.

***Health Professional Summary***

Full Title
Vaccination to Enhance the Anti-Tumor Activity of GD2 Chimeric Antigen Receptor-Expressing, VZV-Specific T Cells in Subjects With Advanced Sarcomas (VEGAS)

Objectives
Primary Outcome Measures:
Number of subjects with a dose limiting toxicity [Time Frame: 6-weeks] [Designated as safety issue: Yes]

Secondary Outcome Measures:
Number of patients with a response to the T cells [Time Frame: 14 weeks] [Designated as safety issue: No]
Amount of T cells in the blood after the infusions [Time Frame: 15 years] [Designated as safety issue: No]


Protocol Entry Criteria
Inclusion Criteria:

Procurement:

- Diagnosis of refractory or metastatic GD2-positive sarcoma not responsive to standard treatment.

- Either previously infected with varicella zoster virus(VZV; chicken pox) or previously vaccinated with VZV vaccine

- Karnofsky/Lansky score of greater than or equal to 50

- Informed consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent

Treatment:

- Diagnosis of refractory or metastatic GD2-positive sarcoma not responsive to standard treatment

- VZV seropositive

- Recovered from the acute toxic effects of all prior chemotherapy (at least 4 weeks from start of last chemotherapy) before entering this study

- Karnofsky/Lansky score of greater than or equal to 50

- Bilirubin less than or equal to 3x upper limit of normal, AST less than or equal to 5x upper limit of normal, Serum creatinine less than or equal to 2x upper limit of normal, Hgb greater than or equal to 9.0 g/dl, ANC>500/uL, platelets > 50,000/uL

- Pulse oximetry of greater than or equal to 90% on room air

- Sexually active patients must be willing to utilize one of the more effective birth control methods for 6 months after the CTL infusion. Male partner should use a condom.

- Available autologous transduced cytotoxic T lymphocytes with greater than or equal to 20% expression of GD2 CAR determined by flow-cytometry and killing of GD2-positive targets greater than or equal to 20% in cytotoxicity assay

- Informed consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent

Exclusion Criteria:

Procurement:

• Known primary immune deficiency or HIV positivity

Treatment:

- Severe intercurrent infection

- Known primary immune deficiency or HIV positivity

- Pregnant or lactating

- History of hypersensitivity reactions to murine protein-containing products

- Known allergy to VZV vaccine

Special Study Parameters
Patients give blood to make GD2-T cells that are grown and frozen. To get the GD2-CAR to attach to the surface of the T-cell, a gene is inserted into the T-cell. As described in the Brief Summary, the gene contains the GD2-CAR. This is done using part of a virus (known as a retrovirus) that has been put into a vector made for this study and that will carry the antibody gene into the T cell. This retrovirus vector also helps identify the T cells in the patient's blood after they have been injected. Because the patients have received cells with a new gene in them they will be followed for a total of 15 years to see if there are any long term side effects of gene transfer.

When enrolled on this study, patients will be assigned to one of 6 groups of different doses of GD2-T cells. At the beginning, patients will be started on the lowest dose of GD2-T cells. Once that dose schedule proves safe, the next group of patients will be started at the next higher dose. If the patient is assigned to dose level 1, 2 or 3 they will receive one infusion of GD2-T cells followed by a dose of the VZV vaccine. If the patient is assigned to dose level 4, 5 or 6 they will receive an infusion of GD2-T cells followed by a dose of the VZV vaccine followed by another infusion of GD2-T cells.

An injection of cells will be given into the vein through an IV line at the assigned dose. Before the injection is received, a dose of Benadryl and Tylenol will be given. The injection will take between 1 and 25 minutes. After the injection the patient will be followed in the clinic for 1 to 4 hours. The treatment will be given by the Center for Cell and Gene Therapy at Texas Children's Hospital or Houston Methodist Hospital. After the GD2-T-cell injection the patient will be followed in the clinic or through communication with their primary doctor.

Six weeks later, the patient will receive a dose of the VZV vaccine. This will be given as a subcutaneous (just under the skin) injection and takes less than a minute. The vaccine will also be given by the Center for Cell and Gene Therapy at Texas Children's Hospital or Houston Methodist Hospital.

Depending on when the patient enters the study, they may also receive a second dose of GD2-T cells two days after the vaccine. This second dose of GD2-T cells will be given the same way as the first dose. Approximately half the patients enrolled in the study will receive one dose and half will receive two doses of GD2-T cells.

Medical tests before treatment--

Before being treated, patients will receive a series of standard medical tests:

- Physical exam

- Blood tests to measure blood cells, kidney and liver function

- Measurements of their tumor by routine imaging studies. We will use the imaging study that was used before to follow the patient's tumor (Computer Tomogram (CT), Magnetic Resonance Imaging (MRI), or Positron Emission Tomography(PET/CT)

Medical tests during and after treatment--

Patients will receive standard medical tests when they are getting the infusions and afterwards:

- Physical exams

- Blood tests to measure blood cells, kidney and liver function

- Measurements of their tumor by routine imaging studies 6 and 12 weeks after the infusion

To learn more about the way the GD2-T cells are working and how long they last in the body, an extra amount of blood, based on the patient's weight, up to a maximum of 60 mL (12 teaspoons) of blood will be taken on the day of the GD2-T- cell infusion(s), (before and at t

Estimated Enrollment
26

Terms
This is an abstract courtesy of ClinicalTrials.gov, a service of the US National Institute of Health Developed by the National Library of Medicine. ClinicalTrials.gov provides regularly updated information about federally and privately supported clinical research in human volunteers.

***Trial Contact Info***

Please note, EmergingMed has not independently confirmed the accuracy of this contact information or the status of the trial at this site.

Site Name: Houston Methodist Hospital
City: Houston
State/Province: TX
Country: USA
Protocol ID: H-32335 VEGAS...NCT01953900
Institutional ID:

Contacts:

Name: Lisa L Wang, MD
Role:
phone: 832-824-4822
fax:
email: llwang@texaschildrens.org
Note:

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Thanks for always being there for us when we feel so incredibly alone,
Kathy






























Kathleen Coursen <kathycoursen@gmail.com>


6:10 AM (3 hours ago)








to Thomas

Dear Kathy,
I am so very grateful and relieved that the second suspicious lump seems to have disappeared and that the treated one is now shrinking Hopefully the second lump was just an area of post treatment swelling which has now disippated and resolved.
I am so sorry for Tom's continued post L-4 treatment pain and Hope that the pain team can provide some pain relief meds and treatment options for him. Do Tom's doctor's have any idea what may be causing the pain since the L-4 met is Hopefully now gone or at least smaller? In Brittany's situation with the radical resection of her large cervical and thoracic spinal tumor in which they removed one third of each of seven vertebrae, she suffers severe and chronic spinal pain attributed to lack of spinal support for her neck and head from the missing portion of her seven vertebrae as well as the post-op surgical trauma and scar tissue. Heartbreakingly, her surgeon and the pain team are unable to provide her with any significant pain relief as they have told us they have done and given her everything possible including high dose opiate pain meds, physical therapy, and hypnotherapy in addition to the accupuncture treatment (provided short term relief) and botox injections (a complete failure!!) which we pursued on our own. We continue to search and Hope that an effective pain treatment and remedy can be found, but in the meantime, Brittany Lives with intense ongoing debilitating pain. She finds some relief from a heating pad and marijuana which is now legal in Washington does seem to help her to relax through the pain. We had considered implantation of a pain pump, but dear ASPS patient Jordanne Gerbing had a very negative and unsuccessful experience with her implanted pain pump so we decided not to pursue it.
The immunotherapy trial at MD Anderson sounds very interesting and promising, but I am personally not familiar with this particular one. Immunotherapy vacccines are being widely researched for cancer treatment and the concept on which they are based seems to be a very valid approach. Brittany participated in the GVAX Immunotherapy Vaccine Clinical Trial at Dana Farber in Boston in 2005-2006, but unfortunately it did not prove to be successful and all four of the other ASPS patients who we personally knew who participated in the Trial at the same time as Brittany had disease progression and have tragically now lost their ASPS battles. One part of the GVAX Vaccine trial was a voluntary biopsy mid-way through the trial to try to determine if the vaccine was having a positive effect, but we declined the biopsy fearing the spread of the cancer cells. I don't know if the other four patients agreed to and received the biopsies, and if they did if that may have contributed to their aggressive post trial disease progression, but it is certainly something to be aware of and to consider if Tom decides to participate in the MD Anderson immunotherapy trial and a biopsy is requested.
I Hope that Tom and you find Dr. Tawbi to be knowledgeable, responsive, caring, and helpful in his patient care of Tom and providing guidance for his treatment. I will be there with you in heart and thought for your appointment with Dr. Tawbi, and want you to know that you are never alone in this difficult battle dear Kathy because Tom and you are a very special part of our ASPS Family and we all share this journey together and are always here for each other to help in any way that we can with shared information and strengthening support and encouragement.
Reaching out across the miles to embrace Tom and you with special hugs, deepest caring, healing wishes, warm friendship, love, and continued Hope,
Bonni

Kathy, they say that this trial is for the GD2-positive Sarcoma but I do not see anywhere in the text that the patient's tumor is tested for it pre-trial? Do you want to contact the trial investigator to find out how it works? I had Ivan's tumor tested for other markers for some immonotherapy trial awhile ago that had some responders from the people I know trough the general sarcoma mailing list, but his was negative so he did not qualify.

Bonnie and Olga,
Thank you both for your speedy responses. I have already emailed and will let you know what response I get. My next question and it may sound silly. Tom and had on and off jaw pain for almost a year. Would the brain mri show the jaw? Would he need a different test?

I do not know how wide is the MRI field they take, ask the doctors there and do not be shy about it, jaw pain is not something we should overlook based on our community experience. I advocate everyone to keep their teeth well so when there is a pain it won't be masked by some dental problems.

I just called his current oncologist. A new mri is being ordered. I feel like such a fool. How could I have let this slip. He complained on and off for a while. I am just praying so hard that this is not related to his cancer. I cannot believe I did not act on this earlier. Thank you Olga - once again.

Hello again dear Kathy,
I am so grateful that the oncologist was so responsive in ordering an MRI to evaluate Tom's jaw pain. I am holding very tight to Hope that it is not ASPS related, but agree with Olga that jaw pain (or any pain for that matter) should not be overlooked or disregarded given the nature of this disease. You should not be critical of yourself dear Kathy for "letting this slip" as it is so hard to determine what is just normal body aches and pains, and what may be ASPS caused. Brittany gets reluctant to tell me about any new pain she may have because she thinks that I worry too much and always over react, but I just think it is better to be safe than sorry and have everything checked out. It seems that the oncologist should have been aware of Tom's jaw pain if he was doing a thorough job at Tom's appointments in asking him if he had any new pain or symptoms, but maybe Tom wouldn't have mentioned it anyway, and Hopefully his jaw pain was just the result of something benign such as TMJ or stress caused, especially since it has not seemed to progress and increase. Take care dear Kathy, keep the Board updated on the results of the MRI, and know that most positive thoughts and very best wishes are with Tom and you.
With more hugs, deepest caring, healing wishes, love, and continued Hope,
Bonni