A retired Texan had endured 12 years of chemotherapy for blood cancer only to see the disease come back stronger and meaner each time.
It was long, hard and terrible, J.C. Cox, 66, said.
So when he was told that he could take part in a clinical trial of a newly modified form of immune therapy, he signed on.
In that small preliminary trial, the results of which were published Wednesday in the New England Journal of Medicine, nearly two-thirds of the patients, all of whom had cancer so advanced that just a decade ago there would have been no hope for them, went into complete remission. Cox was among that two-thirds.
The new treatment involves tweaking a type of therapy called CAR-T that helps the immune system home in on cancer cells. Those tweaks appear to have made it more effective than its predecessor while also leading to fewer side effects, the study found.
In CAR-T therapy, doctors equip a patients own T-cells with a sensor that essentially sniffs out a protein on cancer cells, allowing them to glom onto the protein and then destroy the diseased cells. CAR-T therapy has been approved by the U.S. Food and Drug Administration to treat several types of blood cancer.
The altered T cells end up working like a heat-seeking missile, said study co-author Dr. Katayoun Rezvani, a professor of stem cell transplantation and cellular therapy at The University of Texas MD Anderson Cancer Center.
There were several drawbacks to CAR-T, including the time it took to make the revved up T cells which needed to be harvested from patients and then sent to a lab, their cost, and most important, the possibility of life-threatening side effects.
To try to make a cheaper, safer therapy that would potentially work for all patients, Rezvani and her colleagues switched from T-cells to a different type of immune cell, called natural killer cells.
Let our news meet your inbox. The news and stories that matters, delivered weekday mornings.
Natural killer cells are the best killers of virally infected and abnormal cells, she said. They can continue to patrol and recognize abnormal cells.
There were several advantages to the natural killer cells, not the least of which was that, unlike T cells, they wouldnt make the patients sick by spewing out a flood of inflammatory proteins, leading to a severe condition called a cytokine storm. Another big advantage was that the natural killer cells from one patient could be given to another without any tissue matching. That meant that such cells from healthy donors or from donated umbilical cord blood could be banked and ready to use at any time.
Presumably, this would cut down on the wait time and the costs of the therapy, because the treatments werent being tailor-made for each individual patient.
Rezvani and her colleagues genetically modified the natural killer cells to have a receptor (the R in CAR) for a protein that is on the surface of the cancer cells they were targeting. The receptor would lock onto the protein and then the cell would do its work.
The researchers also tweaked the natural killer cells in two other ways. Unlike T cells which live for a long time, natural killer cells normally have a lifespan of just a couple of weeks, so the researchers added a growth factor that would keep them around for a lot longer. And as a precaution, they also inserted a switch that would allow the researchers to kill off the altered cells if they became too abundant.
Rezvani and her colleagues tested the new treatment in 11 blood cancer patients. When the patients were checked two months after treatment, seven had no signs of cancer while one other showed improvement but not complete remission. The other three had no response to the treatment.
Cox was the eighth patient to receive the new treatment, and initially had misgivings.
I didnt have any other options, Cox, who received the treatment for non-Hodgkin lymphoma, said. But it was scary knowing I would be No. 8 and would be getting the biggest dose.
The trial had been set up to start with a low dose, and then wait to see if there were any serious side effects. If not, the plan was to increase the dose in later patients.
Coxs years of chemotherapy made him worry about possible side effects. But it was probably the easiest thing Ive ever done, he said.
The researchers themselves werent sure what to expect. We were amazed at the safety, Rezvani said. And it didnt seem to matter what dose we gave. This truly is a living drug. It gets inside of the patients body and starts growing and attacking the cancer cells.
Larger studies are needed, but if the treatment which has been licensed to Takeda Pharmaceutical Co. lives up to its early promise, Rezvani hopes to try it on other cancers, such as ones that affect the brain and the breasts.
The response of the patients in the new study is impressive, said Dr. David Porter, the director of cell therapy and transplantation at the University of Pennsylvania Health System. I think this is a major advance in the field of targeted cellular therapy.
Moreover, the natural killer treatments dont seem to have the same life-threatening complications as the original CAR-T therapy, Porter said in an email. Porter was involved with previous CAR-T research, but was not involved with this trial.
But, Porter cautioned, the study included a very small number of patients.
Cox wasnt sure what to expect when he went to be checked two months after receiving his treatment. The news was better than he could have imagined: there was no sign of his cancer.
I did a lot of crying, but they were happy tears, he said. I still get emotional when I talk about it.
Follow NBC HEALTH on Twitter & Facebook.
The rest is here:
Immune therapy tweak offers new hope to blood cancer patients - NBC News
- Doctors To Use 'Trained' Stem Cells To Heal Heart [Last Updated On: September 5th, 2011] [Originally Added On: September 5th, 2011]
- Doctor Shocks Oprah with News of Ethical Stem-Cell Successes [Last Updated On: September 5th, 2011] [Originally Added On: September 5th, 2011]
- 8-18-11 wound care doctor... A day in life stem cell transp [Last Updated On: September 5th, 2011] [Originally Added On: September 5th, 2011]
- Doctors Use Stem Cells for First Trachea Transplant [Last Updated On: September 6th, 2011] [Originally Added On: September 6th, 2011]
- Doctor discussing fat stem cell therapy [Last Updated On: September 7th, 2011] [Originally Added On: September 7th, 2011]
- First American Doctor to talk about results of Stem Cells to treat Autism! [Last Updated On: September 8th, 2011] [Originally Added On: September 8th, 2011]
- Stem Cell Doctor Robert Trossel [Last Updated On: September 8th, 2011] [Originally Added On: September 8th, 2011]
- Doctors recommend StemSave for banking stem cells from teeth for those parents who missed the opportunity to save their childrens cord blood [Last Updated On: September 9th, 2011] [Originally Added On: September 9th, 2011]
- American certified doctor discussing stem cell therapy [Last Updated On: September 9th, 2011] [Originally Added On: September 9th, 2011]
- Doctors defend stem cell research [Last Updated On: September 11th, 2011] [Originally Added On: September 11th, 2011]
- New Stem Cell Breakthrough [Last Updated On: September 11th, 2011] [Originally Added On: September 11th, 2011]
- Stem cells used in organ surgery - 20 November 2008 [Last Updated On: September 14th, 2011] [Originally Added On: September 14th, 2011]
- Brilliant Minds - Stem Cell Research | Tomorrow Today [Last Updated On: September 14th, 2011] [Originally Added On: September 14th, 2011]
- Houston doctors use stem cells in new way.flv [Last Updated On: September 14th, 2011] [Originally Added On: September 14th, 2011]
- Discovering Religion: Ep 16 - Embryonic Stem Cells [Last Updated On: September 16th, 2011] [Originally Added On: September 16th, 2011]
- Stem Cell Event [Last Updated On: September 17th, 2011] [Originally Added On: September 17th, 2011]
- Bone Marrow Transplant and Stem Cell Transplant Treatment in India for International patients [Last Updated On: September 18th, 2011] [Originally Added On: September 18th, 2011]
- Dr. Thomas Barnes' Stem Cell FaceLifts and cell therapies with the CHA Station [Last Updated On: September 20th, 2011] [Originally Added On: September 20th, 2011]
- Worlds First Synthetic Organ Transplant: Swedish doctors sets world record [Last Updated On: September 24th, 2011] [Originally Added On: September 24th, 2011]
- Breakthrough Windpipe Transplant Uses Stem Cells [Last Updated On: September 25th, 2011] [Originally Added On: September 25th, 2011]
- Stem Cell Therapy- Interview with Indian Surgeon [Last Updated On: September 27th, 2011] [Originally Added On: September 27th, 2011]
- Stem Cells, Sight For The Blind [Last Updated On: September 27th, 2011] [Originally Added On: September 27th, 2011]
- "5000 More" Music Video [Last Updated On: September 29th, 2011] [Originally Added On: September 29th, 2011]
- French Doctors Using Red Blood Cells Created From Stem Cells [Last Updated On: September 29th, 2011] [Originally Added On: September 29th, 2011]
- Stem Cell Transplant Program [Last Updated On: September 30th, 2011] [Originally Added On: September 30th, 2011]
- Prof Ian MacKenzie on cancer stem cells part 3 of 3.avi [Last Updated On: October 2nd, 2011] [Originally Added On: October 2nd, 2011]
- Stem Cell Treatment in New Jersey- Colon's story (Dr Damon Noto) [Last Updated On: October 3rd, 2011] [Originally Added On: October 3rd, 2011]
- Prof Ian MacKenzie on cancer stem cells part 1 of 3.avi [Last Updated On: October 3rd, 2011] [Originally Added On: October 3rd, 2011]
- Stem cell treatments and rejection reactions [Last Updated On: October 4th, 2011] [Originally Added On: October 4th, 2011]
- Stem cell therapy helps dog walk [Last Updated On: October 4th, 2011] [Originally Added On: October 4th, 2011]
- SCA stem cell treatment evaluation in China for Jeff Crowe [Last Updated On: October 4th, 2011] [Originally Added On: October 4th, 2011]
- Stem Cells: Seeds of Hope? [Last Updated On: October 4th, 2011] [Originally Added On: October 4th, 2011]
- American Stem Cell And Anti Aging Center [Last Updated On: October 4th, 2011] [Originally Added On: October 4th, 2011]
- Stem Cell Implications for ALS (Amyotrophic Lateral Sclerosis) [Last Updated On: October 4th, 2011] [Originally Added On: October 4th, 2011]
- Preview: Stem Cells, New Treatments [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- Life-saving research in to stem cells - Horizon - BBC [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- Explore Health Don Margolis Stem Cells 101 #9 [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- Dr. Steenblock and Stem Cell Therapies - Testimonial from Dr. Meyers [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- Are doctors receptive to stem cell therapy [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- Stem Enhance - Support The Natural Release Of Adult Stem Cells (part 4).flv [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- Welcome to StemCellTV [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- Doctors and Nurses speak out about Zoe's Bone Marrow Buddies [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- Diabetes Type 1 Cure [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- Manning, Owens Try Stem Cell Therapy [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- Sickle Cell Transplant Program [Last Updated On: October 7th, 2011] [Originally Added On: October 7th, 2011]
- Prof Ian MacKenzie on cancer stem cells part 2 of 3 [Last Updated On: October 7th, 2011] [Originally Added On: October 7th, 2011]
- Woman gets a new body part made from her own stem cells [Last Updated On: October 8th, 2011] [Originally Added On: October 8th, 2011]
- Sabrina Cohen "CELLebrity Doctors Calendar Kickoff.mov [Last Updated On: October 8th, 2011] [Originally Added On: October 8th, 2011]
- Eyes repaired with Stem Cells [Last Updated On: October 8th, 2011] [Originally Added On: October 8th, 2011]
- Interview w/ Dr. Lawrence Goldstein, stem cell research expert and advocate [Last Updated On: October 8th, 2011] [Originally Added On: October 8th, 2011]
- British boy receives pioneering stem cell surgery WOW - Video [Last Updated On: October 15th, 2011] [Originally Added On: October 15th, 2011]
- Complications of Diabetes Mellitus Cured by Stem Cell Treatments - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Science Daily: New Discovery May Eliminate Potentially Lethal Side Effect of Stem Cell Therapy - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- Till and McCulloch, fathers of the field of stem cell science : Legacy - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- PRP on the Doctors show - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- UF scientists devise way to sort brain cells for potential transplants - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- Adivive (Lipokit) Autologous Fat Transfer with Adult Stem Cells - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- Injecting Patients With Own Stem Cells to Save Legs - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- Stem cell treatment - the worst case scenario - Video [Last Updated On: October 24th, 2011] [Originally Added On: October 24th, 2011]
- Stem Cells/Burn Recovery - Video [Last Updated On: October 26th, 2011] [Originally Added On: October 26th, 2011]
- Kevin's 2 Heart Transplants and Stem Cell Transplant - Video [Last Updated On: October 26th, 2011] [Originally Added On: October 26th, 2011]
- Stem Cell Breast Augmentation | Laser Liposuction - Video [Last Updated On: October 26th, 2011] [Originally Added On: October 26th, 2011]
- Ciska - Glaucoma Stem Cell Patient - Video [Last Updated On: October 31st, 2011] [Originally Added On: October 31st, 2011]
- Adult Stem Cell treatments center - American Stem Cell [Last Updated On: November 10th, 2011] [Originally Added On: November 10th, 2011]
- Toonari Post Health News: Europe's first clinical trial using human embryonic stem cells - Video [Last Updated On: November 12th, 2011] [Originally Added On: November 12th, 2011]
- Till and McCulloch, fathers of stem cell science : Mentors - Video [Last Updated On: November 12th, 2011] [Originally Added On: November 12th, 2011]
- Stem Cell Transplant Situation - Video [Last Updated On: November 12th, 2011] [Originally Added On: November 12th, 2011]
- Stem Cell/Natural Cosmetic - San Diego KUSI-TV Dr. Maurice Sherman - Video [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- Vampire FaceLift Procedure Discussed on The Doctors.mp4 - Video [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- Tandem stem-cell transplants for myeloma improve survival - Video [Last Updated On: November 24th, 2011] [Originally Added On: November 24th, 2011]
- Doctor's speaks on Cryobanks India - 1 - Video [Last Updated On: December 8th, 2011] [Originally Added On: December 8th, 2011]
- Doctors recommend StemSave for banking stem cells from teeth for those parents who missed the opportunity to save their childrens cord blood - Video [Last Updated On: December 8th, 2011] [Originally Added On: December 8th, 2011]
- Science BREAKTHROUGH | Fountain of Youth | Forever Young Pill - Video [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]
- 11-09-04 GH - Sam McCall [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]
- Ask the Doctors - Dental Stem Cell Banking - Video [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]
- SECRETS TO PERFECT SKIN for Valentine's Day I Naturesknockout - Video [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]
- BioLife Cell Bank - Overview of Provider/Doctor Application - Video [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]
- Stem Cell Implications for ALS - Video [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]
- Stem Cells May Repair Broken Hearts - Video [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]
- Adult Stem Cells Use Special Pathways To Repair Damaged Muscle - Video [Last Updated On: January 23rd, 2012] [Originally Added On: January 23rd, 2012]