New effective leukemnia treatment. Big pharma did not initially want to fund it…

A new leukemia treatment is wowing even the researchers behind its creation, providing results beyond their wildest expectations.

Within three weeks, the tumors had been blown away, in a way that was much more violent than we ever expected, senior author Carl June, MD told Penn Medicine.

The treatment uses the body’s own t-cells to fight the tumors. The t-cells are removed from the body and then reprogrammed to attack the specific type of cancer cells, and ONLY THEM, and then reinserted into the body.

Pharmaceutical companies did not want to fund this research, probably as it was not a half-bad drug that could be patented and sold, but a genetic therapy that was envisioned at a university.

Update: In August of 2012 (One year after this blog post was first written), Novartis and the University of Pennsylvania announced a global research and a licensing agreement that gives Novartis exclusive licensing rights to the technology worldwide. Novartis is also contributing $20 million to build a Center for Advanced Cellular Therapies on the University of Pennsylvania campus that aims to develop and manufacture adoptive T-cell immunotherapies such as the CAR technology. – Well well, whadda ya now. Novartis saw a business opportunity after all…

In any case, extremely good news and it should work for other forms of cancer as well, if i have my microbiology correct, corroborated by the following statement:

Additionally, the team has engineered a CAR vector that binds to mesothelin, a protein expressed on the surface of mesothelioma cancer cells, as well as on ovarian and pancreatic cancer cells.


New method uses body’s own t-cells to kill cancer cells, and only those cells.

Here is some news on this:

Success story here:

And here:


2 thoughts on “New effective leukemnia treatment. Big pharma did not initially want to fund it…

  1. The summary written is actually incorrect – this treatment DOES NOT ONLY kill cancer cells – it kills healthy/benign cells. The treatment is based on targeting cells that contain the protein CD19 which is often found in certain B-Cells malignancies like this type of Leukemia. While the treatment is designed to annihilate the cancer cells that contain CD19, our bodies contain benign (non cancerous) CD19 positive cells. This treatment kills off both. Because a person cannot survive without these normal healthy B-cells, the patients were and continue to take medications/treatments to replace them and according to the researchers this is expected to be required for life. As more of these types of targeted protein treatments develop, part of the success of them will depend not just on an ability to kill cancer cells (and deal with this type of cell death which has very high risks as well) but for science to have in place treatments to replace the healthy naturally occurring protein positive cells that will be destroyed in the process. Depending on the significance of the protein, absence of such medicinal “replacements” may cause serious harm or death.

  2. “Patients who responded to the treatment had various degrees of what is known as a cytokine release syndrome (CRS) characterized by fever, nausea, hypoxia, transient hypotension, and anorexia. The CRS correlated with spikes in the cytokine IL-6 level, and although steroid treatment was partially beneficial, the IL-6 receptor antagonist, tocilizumab was effective in resolving hypoxia, hypotension, and fever in these patients.
    “Though we have found our therapy is associated with significant toxicity, it is very different than bone marrow transplants,” said Porter. “For one, the side effects seem to transient. In bone marrow transplant patients, there remains a risk from long-term immune suppression and slow immune reconstitution, as well as long-term side effects, symptoms, and risks from graft-vs-host disease.” The side effects with CAR appear earlier and resolve without long-term risks, according to Porter. Additionally, the chemotherapy used is more tolerable compared to that used with transplantation. “We have treated patients well beyond the upper age limit that is practical for bone marrow transplants,” said Porter.
    University of Pennsylvania researchers are about to open a larger trial for patients that will determine an optimal treatment dose.”

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