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Doctors Cure Leukemia with HIV?

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sick child

Curing leukemia with HIV may sound like science fiction, but in the modern world of medicine, it’s another hopeful story of how gene therapy is changing the way doctors fight disease.

In December 2012, researchers at the University of Pennsylvania’s Perelman School of Medicine presented the first results of their cell therapy (or immunotherapy) to treat three types of blood cancers to the American Society of Hematology (ASH) Annual Meeting and Exposition. (1)

Dr. Stephan Grupp first presented some of the early results of this cell therapy (CLT019). CLT019 is the official name for the clinical trial for patients with:

–> Acute lymphoblastic leukemia (ALL)
–> B cell non-Hodgkin lymphoma (NHL)
–> Adult disease chronic lymphocytic leukemia (CLL)

The therapy consists of using the patient’s own immune cells that are harvested from her or his blood. These cells are known as T cells. The doctors then alter these cells genetically “to express a protein which will recognize and bind to a target called CD19, which is found on cancerous B cells.” When a patient has certain types of leukemia or lymphomas as in ALL, NHL and CLL, the B cells mutate into cancerous ones.

T cells in the human body serve as soldiers of the immune system. These cells are naturally coded within a healthy body to recognize diseased cells and are programmed to attack them. This is how the human immune system works. Cancerous B cells are able to fool T cells and are cloaked from T cell recognition.

How It Works

The doctors discovered if they harvested the patients T cells and re-engineered them “using a HIV-derived lentivirus vector” (2) so that they were reprogrammed to recognize the B cell protein, the T cells would then attach to the diseased cell protein. These re-engineered T cells are known as “chimeric antigen receptor (CAR) T cells”.

Dr. Carl June, the trial’s leader, stated that he believed it would be possible to use this therapy to “reduce or replace the need for bone marrow transplantation”. (2)

Once reprogrammed, the T cells are transfused back into the patient’s body and these newly coded soldiers begin attacking the invading cancer cells. In addition, the T cells divide and multiply and these increased numbers of cells can then fight the cancer cells more effectively. Even if the T cells wipe out of all of the B cells, they will then live in the body for several months and can rally again should the invading B cells return.


Why Use HIV Virus?

The reason doctors chose the HIV retrovirus is because the lentiviral vectors can infect not only dividing, but also non-dividing cells. This is because the virus shell known as a preintegration complex is able to penetrate the nucleus membrane of the B cells.

The lentivirus is capable of changing the expression of the target cell gene and will stay active up to six months. HIV has a perfected method of penetrating cells in order to express its genes into the helper T cells. Only one cell is impervious to HIV and that’s the quiescent cells in G0 phase (resting phase). The quiescent cells are cells that aren’t dividing. (3)

CLT019 Trial Results

In the CLT019 trail, a total of 12 patients participated. These patients had exhausted all traditional means of therapy and the CLT019 trial was their last hope of finding a cure or at the least a way to slow the cancer growth.

There were 10 adults with CLL and 2 children with ALL in the CLT109 trial conducted by The Children’s Hospital of Philadelphia and the University of Pennsylvania.
Of the 12 patients treated with this cell therapy, the doctors found that nine of the patients had T cells that killed “large quantities of ALL or CLL cells”. (1) Two of the patients have been in remission for over two years.

Side Effects

According to the CLT019 trial results, the T cells that destroyed the leukemia cells also generated a higher than normal immune response in one of the children, Emma Whitehead. This stimulation caused Emma to develop cytokine release syndrome and the sick child was admitted into ICU (Intensive Care Unit).

The resolution was the use of two immunomodulating drugs to reduce the toxic side effect of the ramped up T cells. The child responded very quickly to this counter measure. The protocol was then incorporated into all of the CTL019 treatments. (2)

emily whitehead

Success Story of Emily “Emma” Whitehead

In December 2012, ABC reported about Emma Whitehead, a now 7-year-old girl. She suffered from what had been deemed as “unbeatable” leukemia (ALL). With no more treatments left to try, in April 2012, her parents gave permission for Emma to become one of the participants in the CLT019 trial.

Dr. Grupp reported that bone marrow tests taken three weeks (May 2012) after Emma’s cell therapy revealed that she had gone into remission. It will require two years in remission for Emma’s prognosis to be deemed a successful treatment therapy.

It is too early to definitively state that CLT109 is a new and effective form of therapy for B cell cancer patients since there have only be a handful of patients treated so far. In fact, the trial program is seeking applicants for consideration of participation. Those interested in possible participation in the program can visit Clinical Trials (6) or visit the website for CHOP Children’s Hospital of Philadelphia. (7) If you wish to keep up with Emma’s story, she can be found on facebook. (8)

References & Image Credits:
(1) University of Pennsylvania Health System
(2) Genetic Engineering & Biotechnology News
(3) Kenyon College Biology Department
(4) Wikipedia
(5) ABC News
(6) Clinical Trials
(7) Children’s Hospital of Philadelphia
(8) Emily (Emma) Brooke Whitehead Facebook
(9) searching4jphotography via photopin cc
(10) lu_lu via photopin cc
(11) All Voices

Originally published on

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