Cell-Based Immune Therapy Shows Promise in Leukemia Patients


Doctors have traditionally had limited treatment options to offer adults with B cell acute lymphoblastic leukemia (ALL), a rapidly progressing form of blood cancer. The disease often returns, or relapses, after initial treatment with chemotherapy. At that point, patients are often resistant to additional chemotherapy and poor candidates for stem cell transplantation, which is usually effective only if the disease is in complete remission.

Now Memorial Sloan Kettering investigators report that genetically modified immune cells have shown great promise in killing the cancer cells of patients with relapsed B cell ALL. In fact, all five of the patients who have received the new therapy – known as targeted immunotherapy – have gone into complete remission, with no detectable cancer cells. The results of this ongoing clinical trial are reported online on March 20 in the journal Science Translational Medicine.

“This is a very exciting finding for patients with B cell ALL and a major achievement in the field of targeted immunotherapy,” says Michel Sadelain, Director of Memorial Sloan Kettering’s Center for Cell Engineering, who led the study along with medical oncologist Renier J. Brentjens.

Engineering Precise Weapons

Targeted immunotherapy is aimed at instructing the immune system to recognize and attack tumor cells. Over the past decade, Drs. Sadelain and Brentjens, and other Memorial Sloan Kettering researchers – including Isabelle Rivière, Director of Memorial Sloan Kettering’s Cell Therapy and Cell Engineering Facility, and physician-scientist Marco L. Davila – have investigated an approach that involves removing white blood cells called T cells from patients and introducing a new gene into the cells using an engineered viral vector. Viral vectors are viruses that have been disabled so they cannot replicate and that efficiently shuttle their genetic cargo into a host cell.

After the gene is transferred and expressed, the T cells are infused back into the patient, where they multiply and cause a variety of different immune responses aimed at attacking the cancer cells. The gene used in the targeted immunotherapy for ALL codes for the creation of a receptor on T cells that enables them to recognize the CD19 protein, which is present in B cell ALL tumor cells.

Much of the early research into this approach was supported by Memorial Sloan Kettering’s Experimental Therapeutics Center and benefactors of the Center for Cell Engineering.

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A Bridge to Stem Cell Transplantation

“We have been a leading center in developing this technology in the laboratory, and we were the first center to bring this CD19-targeted approach using viral vectors to the clinic,” Dr. Brentjens explains.

In the phase I clinical trial, five patients with relapsed B cell ALL had cancer that was detectable at varying levels in the blood. After receiving the genetically modified T cells, all five patients achieved complete remission, and even highly sensitive molecular analyses found no cancer cells remaining.

“Patients with relapsed B cell ALL resistant to chemotherapy have a particularly poor prognosis,” says Dr. Brentjens. “The ability of our approach to achieve complete remissions in all of these very sick patients is what makes these findings so remarkable and this novel therapy so promising.”

Four of the five patients subsequently received additional therapy in the form of a bone marrow transplant, the standard of care for those patients who successfully achieve complete cancer remissions after treatment for relapsed disease. To date, three of the four patients have remained in remission for between five and 24 months. One patient died from complications unrelated to the cancer therapy while in remission.

“By serving as a bridge to a stem cell transplant, this therapy could potentially help cure adult patients with B cell ALL that has relapsed and who are chemotherapy resistant. Otherwise, these patients have a virtually incurable disease,” Dr. Brentjens says. “We need to examine the effectiveness of this targeted immunotherapy in additional patients before it could potentially become a standard treatment for patients with relapsed B cell ALL.”

Further clinical trials, including a phase II study, have already been planned to test whether B cell ALL patients would benefit from receiving this targeted immunotherapy along with chemotherapy earlier in the disease stage, either as part of the initial frontline treatment or after remission has been achieved to help prevent relapse.

Read a New York Times story about this new therapy and the experience of a Memorial Sloan Kettering patient.

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do you have a clinical trial for aml patients
if so is it open

Would this research benefit Merkel Cell patients? If not what is the latest treatment for Merkel?
Thank You

Are there any new treatments and/or procedures for neuroendocrine tumors that have metastasized to the liver?

Is there any research going on for T-Cell Therapy for CML patients?

Thank You!

Thanks for your comment. We spoke with Dr. Brentjens, who says that unfortunately this is not currently a therapy designed to treat CML.

I was asking about Diamond Blackfan SYndrome? The body does not make red blood cells. I have a grandson with this disorder. He has lived on prednisone for 27 years. Does your new discovery offer hope for this condition?

Thank you for your comment. Dr. Brentjens says that unfortunately, this therapy is not designed to treat Diamond Blackfan Syndrome. He does not foresee that this approach could provide any benefit in this disease.

I had a rare form of stage 4 leiomyosarcoma that had metastized from my uterus through the bloodstream into my small intestines then my lungs 22 tumors and then the cavity of my heart - I also had a fibroid the size of a 5 month fetus with its own blood supply - three months to live - no chemo for me after surgery-I asked my oncologist Dr. S Lichtman to please speak to his research team in Manhattan to whip something up for me I knew I was going to live - so he did and here I am today 5 years later a one in one million survivor - Sloan is the best - there is no place like it thank God I live on Long Island - Janet

I have early stage CLL and keep praying for a medical breakthrough. This has changed my life so much.

My daughter aged 8 has pre B All and is being treated in England. I am sure there are many parent in our position and we would dearly like to know when there are going to be trials for children? Please understand that you are giving hope to people around the world. Thank you so much.

We spoke with Dr. Brentjens, who says, “We’re currently obtaining approval to treat a subset of children with B cell ALL. We hope to open up this trial by June of this year.” Thanks for your comment.

Will this treatment work for my young friend who has krappe . His parents are so despondent. Please reply asap. Thank you.

Thank you for your comment. This treatment is not currently being used for Krabbe disease.

The great mission of!

All of the ALL patient is unfortunate, but because of the new treatment, I think we will become the lamb of god!

I came to China, undergoing chemotherapy. How does this therapy is aimed at ALL is the best effect?

I think you will spend a minute to reply to you, isn't it?

A dear elderly friend of ours was suffering from the B type cancer mention in the above articles. She was being treated at SIU hospital, she and her family were told she only had a few months to live. She started a daily regimen using a supplement called Medizym (systemic enzyme formula) and she has been in complete remission for several years now. Her results were noticed by her Doctors shortly after using the formula mentioned above. The staff of doctors at SIUH attributed her success to some other unknown factor. The only thing more I can add is that this case is well documented
Yours truly,


When T-cell treatment is tested on CLL patients?
My friend, who is 32, is in the last stages of CLL and has bone marrow failure. Now, He can only be cured by hematopoietic stem cell Transplantation. To transplant, he has to need 9.5 HLA matching. whether he is eligible to participate in this clinical trial?

looks like quite a real game changer. this should get the widest possible publicity. i am deeply worried about anti-science and its increasing political gain, cutting things like research on weather, research on guns, research on sexuality, research on cells and stem cells, because of abject predatory ignorance subsidized by amoral predatory corporations. off topic but yours is just the kind of biology based research we can lose to the austerity cabalists.

Does this treatment work Metastatic Chondromyxoid Sarcoma? Please let me know. Thanks for your research and progress.

Thank you for your comment. This type of work is currently being tested in patients with leukemia. If you’d like to make an appointment or learn more about clinical trials available at Memorial Sloan-Kettering, please call 800-525-2225.

My 7 year old daughter has relapsed for the second time. She was diagnosed with ALL in Dec 2009. We are familiar with Emma Whitehead and the research with altered T-cells. It sounds to me that you are still in Phase 1 with adults. Is this correct? We would love to discuss this more if it could be an option for Amelia.

Yes, this is correct. Our experts are currently obtaining approval to treat a subset of children with B cell ALL as well. They hope to open up this trial by June of this year. Thank you for your comment.

Are you doing anything or will this work for patients with MDS and tranfusion dependent patients with myelodysplastic syndrome?

I presently am on procrit injections which I receive weekly. My RBC fluctuates between 8.8 and 9.6 and thus far, my RBC does not seem to climb to 10 or over. My doctors tell me that I do not have have lukemia.

I am a ALL patient, my friend is AML patients. Want to know what time will be a large phase 2 or will be applied to clinical. As you know, is bearing the brunt of leukemia and chemotherapy caused by torture us at this moment, sincerely look forward to your reply, thank you very much!

Thanks for your comment. Further clinical trials, including a phase II study, have already been planned to test whether B cell ALL patients would benefit from receiving this targeted immunotherapy along with chemotherapy earlier in the disease stage, either as part of the initial frontline treatment or after remission has been achieved to help prevent relapse.

Sorry, there is a problem. I know more in-depth research for you are collecting money, do not know whether you have the minimum donation amount.

Thank you to the team, to bring the gospel to the world of leukemia patients, God bless us!

Are there any clinical trials for lung cancer performed at this time,and are they in the line of Cell Based Immune Therapy?Thank you for your reply.
Catherine Hepprich

My sister has metatastic colon cancer staged at 1V, is this therapy being used for this type of cancer also, which is what I heard?

I had a very small fallopian tube cancer, cm and half, but grade 3, I had 3 rounds of chemo and 18 months later, it has returned in my liver (9 small tumors) and one pushing on my stomach. I have been on chemo since nov. - the stomach tumor has gone and the others very reduced. Would this quality me to be in your clinical trail. thanks peggy

Any new news on the subject of Lymphoblastic Lymphoma in patients who do not have HIV-Aids?

Thank you for your comment. We spoke with Dr. Brentjens, who said that some patients with lymphoblastic lymphoma may be eligible for treatment in this clinical trial.

(1) what resources (financial/medical) would you need to develop a cell- based immunotherapy like this focused on sclc? (2)how long would it take to create a sclc cell-based immunotherapy? (3)who do we contact if we can get you the resources you need to do this for sclc?

We spoke with Dr. Sadelain, who says, “We are developing a project for NSCLC, but we are not funded to do so in SCLC. Assuming targets could be readily identified and that the preclinical development were amply supported financially, it would take at least a year to establish proof-of-concept, manufacture reagents, and obtain regulatory approvals.” If you are interested in contributing resources for this research, please contact Audrey McInerney at [email protected].

Will that work with the MDL lukemia

This trial evaluated the treatment in acute lymphoblastic leukemia (ALL). If you’d like to make an appointment with one of our physicians, please call 800-525-2225. Thank you for your comment.

Does this have any connection to my LPL, • Waldenström macroglobulinemia (lymphoplasmacytic lymphoma)? It is in my bone marrow not my lymph nodes. I have gone through chemo treatments in 2012 with progress and will have a 3 month blood check April 30th to see how it's going.

We spoke with Dr. Sadelain, and he says that this approach is applicable in principle to WM. However, our experts do not currently have funding to undertake a study of this approach in WM. Dr. Malcolm Brenner at Baylor College of Medicine in Houston may be opening a trial open to patients with WM. Thanks for your comment!

My husband Carl has myelofibrosis. Are there any immunotherapy studies for this condition, since it is also involving bone marrow?

Lorna, this immune therapy is not currently being evaluated for myelofibrosis. If you’d like to make an appointment with a Memorial Sloan-Kettering physician to discuss other treatment options, please call 800-525-2225. Thank you for your comment.

I saw on the comments that clinical trials are planned for metastatic breast cancer. Which types of breast cancer are concerned ?