Living in a time of massive technological advancements has allowed us to witness amazing feats. We walk around with small, yet powerful computers in our pockets, we have put humans on the moon, and now we may have found a cure for leukemia.
Leukemia is a type of cancer where white blood cells, also called leukocytes, grow uncontrollably in bone marrow and crowd out marrow cells and normal blood cells.
Leukemia is often treated with chemotherapy, radiation therapy, medication, and/or bone marrow transplants.
With these treatments, some patients have been known to live fairly long lives with the median age of death being in the mid-70s. However, their lives are riddled with doctors’ visits and the terrible symptoms of the cancer.
Now with a possible cure on the horizon patients and their families can begin to hold hope that their struggles may soon be eased.
The new treatment focuses on administering genetically engineered immune cells into the patients’ body. The method is CAR (chimeric antigen receptor) T-cell therapy.
T-cells are a type of immune cell that fight diseases in the body. CAR-T cell therapy works by taking some of the patients T-cells and then splicing them with genetic material that will help them fight the cancer cells.
Clinical trials have seen much success with this method with many patients experiencing long-term remission.
In 2016, the University of Pennsylvania, one of the leading groups in this type of research, opened a $27 million center whose focus will be on developing treatments for cancer. They have collaborated with Swiss pharmaceutical company, Novartis. Novartis has invested $20 million in the center and is working on putting the treatment on the global market.
Penn Medicine posted a news release in 2014 stating, “A University of Pennsylvania-developed personalized immunotherapy has been awarded the U.S. Food and Drug Administration’s Breakthrough Therapy designation for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL). The investigational therapy… is the first personalized cellular therapy for the treatment of cancer to receive this important classification.”
Penn Medicine and Novartis are not the only companies racing to finish the development of the treatment though. Dozens of companies around the world are working hard to get their treatments done first and out on the market.
Doctors at the Great Ormond Street Hospital in London have announced that they are seeing positive results now too. These Doctors are using a different method than those at Penn Medicine and Novartis.
They are nicknaming their method the “off-the-shelf” approach. This approach is a bit controversial as only two infants have been cured and they were on a chemotherapy regimen before the experiment took place.
This has caused some scientists to question whether it was the chemo, the off-the-shelf approach, or a combination of the two that caused remission.
If the “off-the-shelf” approach is what ended up curing them it could be a much cheaper option for those seeking CAR-T cell therapy.
Penn Medicine and Novartis’s method is to take the patients’ own T-cells to genetically modify then put back in the body.
Great Ormond Street Hospital’s method is to collect donor blood, modify it, then freeze and store it to give to patients. It would cost about $4000 per dose. The more personal method of using the patient’s T-cells may cost $50,000 per dose.
While it is exciting to hope for a less expensive cure, it is still unknown whether or not this “off-the-shelf” method works as well as the one used by Penn Medicine and Novartis.
Neither method is available commercially yet though Penn Medicine expects to receive FDA approval sometime this year, 2017.
Penn Medicine has also been using CAR-T cell therapy to target brain tumors, formed from a brain cancer known as glioblastoma (GBM), and they have found success. With even solid tumors attacked by the genetically modified immune cells.
Genetically modified cells have great promise in curing diseases and within the year, we can expect to see the cure for Leukemia being available commercially. This fantastic research will save many lives.