ASH 2013: Can New Gene Therapy Help Without Causing Leukemia?

A presentation at the American Society of Hematology meeting demonstrated a very small study indicating that kids with so-called "bubble-boy disease" may benefit from gene therapy without the common side effect of leukemia.

The study was conducted by Sun-Yun Pai, M.D., of the Dana Farber Cancer Institute and Boston's Children's Hospital, and colleagues. Out of nine children under one year old and diagnosed with X-linked severe combined immunodeficiency (SCID) who were given a self-inactivating (SIN) gammaretroviral vector, seven were doing well after three years.

Typically the treatment for SCID involves a stem cell transplant, but finding donors takes a very long time in most cases.

Gene therapy an 'attractive option'

Pai was quoted by MedPage Today as saying:

Without curative therapy such as bone marrow transplantation, these boys die of opportunistic or community-acquired viral infection. Outcomes for boys who do not have well-matched donors are suboptimal. For these patients, gene therapy is an attractive option.

SCID is an otherwise fatal disease and genetic therapy has been attempted in these kids before, but several of them from a French study who were given a different gammaretroviral vector developed T-cell acute lymphoblastic leukemia. One of the patients died while the others recovered. Since the other kids recovered and are said to be doing well, this justified a similar gene therapy approach, but one that tried to eliminate the leukemia risk.

While it appears that Pai's group has done that, it should be added that in the other trial, the kids who developed leukemia developed the disease at the three- to five-year mark, so there's no assurance that kids from this group will not develop the blood cancer too.

Source: MedPage Today

More Articles

More Articles

This article looks at the Non-Hodgkin's Lymphoma survival rate as well as the Non-Hodgkin's Lymphoma mortality rate ....

The term NK T cell lymphoma refers to one of two subtypes of lymphoma that affect the NK (Natural Killer)...

Canine lymphoma is a fairly common cancer in American dogs. The most commonly seen lymphoma in America is a type called Lymphosarcoma, although...

Prednisone is a glucocorticosteroid (a steroid) used in the treatment of many types of cancers. It functions as an anti-inflammatory medicine that...

Marginal zone lymphomas (MZL) are indolent lymphomas that affect the B-cells. The...

Large Cell Lymphoma (LCL) is typically an aggressive (fast growing) cancer of either the B cell or T cell type. They are one of the most common...

A B cell is a type of lymphocyte that produces antibodies to fight infections. These are the most prevalent lymphocytes in the bloodstream and are...

Aggressive Non-Hodgkin’s Lymphomas (NHLs) are fast growing cancers (as opposed to indolent cancers). They involve...

A lymphoma prognosis varies greatly depending on the type of lymphoma. There are more than 35 types of lymphoma, including 5 types of...

What causes lymphoma is not well known. DNA mutations may be what causes lymphoma to develop but what triggers these mutations is...

Often the one who makes the first diagnosis of Hodgkin's Lymphoma / Disease is the person affected. There are some...

Lymphedema is abnormal swelling due to the presence of excess lymphatic fluid within the tissues. This swelling occurs when the...

RICE is an acronym for an anti-cancer treatment that expresses a combination chemotherapeutic regimen. This regimen is written variously as "R+ICE...

Hodgkin's Disease—also referred to as Hodgkin's Lymphoma, these are the exact same diseases, just...

Lymphoma is a cancer affecting the white blood cells (lymphocytes) of the body's immune system. The cells begin to grow abnormally and much faster...