Leukemia Treatment Approaches
Patients with acute leukemia usually require induction treatment to reduce symptoms and return blood counts and bone marrow to normal. Once remission is achieved, additional treatment is given to help prevent recurrence of the disease.

Patients with chronic types of leukemia may receive treatment, or they and their City of Hope leukemia team may decide to wait until symptoms appear before taking action.


Chemotherapy is the main treatment for all forms of leukemia. Because leukemia is a cancer of the blood, surgery is not an option as it is with solid tumors. As a result, chemotherapy uses powerful drugs to kill leukemic cells. The drugs target fast-dividing cells by disrupting critical parts of the cell cycle. Because cancer cells divide faster than normal cells, more cancer cells than normal cells are killed; however, a significant number of normal cells are damaged, which causes the many familiar side effects of chemotherapy. Chemotherapy may be given orally, intravenously through an IV or catheter, or into the cerebrospinal fluid. Most often, combinations of chemotherapy drugs are used to achieve the optimal therapeutic outcome.
City of Hope’s New Drug Development Program is actively developing and testing novel drugs and drug combinations designed to improve outcomes for all types of leukemia. Chemotherapy is usually given in cycles, sometimes starting with intensive induction treatment, which takes several days. This is followed by a few weeks without treatment, allowing the patient to recover from side effects, mostly related to lower blood counts. The sequence is then repeated.  Patients who achieve initial remission require additional treatment, usually given over a period of months (in chronic lymphocytic leukemia and acute myeloid leukemia) or years (in acute lymphoblastic leukemia and chronic myelogenous leukemia) in order to prevent recurrence.

Chemotherapy for Acute Leukemia

Treatment for acute leukemia is intensive and usually requires hospitalization.

Chemotherapy for Acute Lymphocytic Leukemia (ALL)

Induction chemotherapy for ALL usually involves a number of agents. The following is a common combination protocol:
  • L-asparaginase (Elspar) or PEG-L-asparaginase (pegaspargase, Oncaspar)
  • Daunorubicin, also known as daunomycin (Cerubidine)
  • Vincristine (Oncovin)
  • Prednisone (numerous brand names)
Post-remission therapy is determined by the patient’s adverse risk factors and will be determined via cytogenic and other genetic markers. If the patient’s leukemia falls in a low-risk group, Cytarabine consolidation therapy may be used. If the patient falls into a high-risk group, we would then recommend a stem cell transplant.

Chemotherapy for Acute Myelogenous Leukemia (AML)

Induction chemotherapy for AML usually involves a combination of two or three different anticancer drugs. One often-used combination is the following:
  • Daunorubicin, also known as daunomycin (Cerubidine) or idarubicin (Idamycin)
  • Cytarabine, also known as cytosine arabinoside or ara-C (Cytosar)
Post-remission therapy is determined by the patient’s adverse risk factors and will be determined via cytogenic and other genetic markers. If the patient’s leukemia falls in a low-risk group, Cytarabine consolidation therapy may be used. If the patient falls into a high-risk group, we would then recommend a stem cell transplant.

Chemotherapy for Chronic Leukemia

For patients with chronic leukemia, such as chronic myeloid leukemia (CML) or chronic lymphocytic leukemia (CLL), oral or intravenous drugs may often be given in an outpatient setting.

Chemotherapy for CML

First-line treatment for CML is either Nilotinib or Dasatinib.  Gleevac was the first recommended treatment for CML and works by targeting tyrosine kinase, the protein produced by a gene on the abnormal Philadelphia chromosome, which is a hallmark of CML. Recent studies have shown that Dasatinib and Nilotinib have improved the response rate and are now recommended as upfront therapy.
Follow-up care requires close monitoring of the VCR-ABLE response to treatment and making sure that certain landmarks are achieved. Patients are monitored to ensure they are not developing any mutations that might require a different therapy. If these treatments fail to work for a patient, he or she would be a candidate for a stem cell transplant.

Chemotherapy for CLL

Chemotherapy for chronic lymphocytic leukemia (CLL) is used when the course of the disease appears aggressive rather than indolent, based on factors such as blood counts, cytogenetics / fluorescence in situ hybridization (FISH) testing and symptoms.

New Approaches to CLL

Current upfront therapies and new approaches to CLL include:
  • Monoclonal antibody-targeted therapy monoclonal antibodies are increasingly being used in the treatment of CLL, including Rituximab(Rituxan), and a combination of fludarabine, cyclophosphamide and rituximab (FCR), or ritoxin and dendeamustine.

For recurrent diseases, we have numerous  clinical trials, including the use of Bruton’s tyrosine kinase inhibitors.

Beyond Chemotherapy: Advances in Treating Leukemia

City of Hope utilizes  stem cell transplantation and pioneering approaches such as radioimmunotherapy and adoptive T cell therapy in treating leukemia patients. These methods augment the effectiveness of chemotherapy.  In fact, for many patients, a stem cell transplant offers a real chance at curing leukemia rather than just achieving remission.
City of Hope’s acclaimed leadership in stem cell transplantation ensures that patients receive the latest treatment protocols designed to maximize successful transplant engraftment, while minimizing rejection and debilitating side effects.

Stem Cell Transplantation

Hematopoietic cell transplantation (HCT) and peripheral blood stem cell transplantation are therapeutic treatments that use stem cells (immature blood cells) to treat a patient's malignancy or repair diseased or defective bone marrow. Transplants are sometimes performed early in the course of treatment to improve outcomes. In some patients, they are utilized when other treatments are not working.
These transplant procedures include intensive chemotherapy, with or without radiation therapy, to destroy the cancerous cells. This is followed by an infusion of healthy new stem cells, which have the ability to grow back into the bone marrow and begin making normal blood cells again.

If a patient receives stem cells from a matched donor (using related, unrelated or cord blood), the transplant is called allogeneic. Like other tissue transplants, allogeneic stem cell transplants require a genetic match between the donor and recipient.

In allogeneic transplants, the donor is preferably a sibling. Alternatively, a matched unrelated donor who has a similar genetic type may be used. In fact, 45 percent of allogeneic transplants at City of Hope come from volunteer donors unrelated to the patient.
New alternative donor sources include cord stem cells obtained from a cord stem cell bank. These are cells that are cryopreserved and obtained from the umbilical cords of newborn babies.  Another option is a 50 percent match from the patient’s mother or father.

In some cases, a patient’s own stem cells may be used. This is called an autologous (self) transplant. First, stem cells are removed from the blood or bone marrow of the patient and stored. When the intensive chemotherapy and/or radiation are completed, the stem cells are then infused back into the patient. As these new stem cells grow, they restore the body's own blood cells.

In the transplant process, intensive chemotherapy also causes weakening of the immune system (immunosuppression). This helps to prevent the body’s rejection of the newly transplanted stem cells from a related or unrelated donor. In contrast, with autologous transplants, there is little risk of rejection of a patient's own stem cells.

Non-Myeloablative HCT

A new transplant procedure has been developed to treat patients with leukemia and myelodysplasia who are older or have underlying medical problems. Non-myeloablative HCT, also called “mini-HCT or “mini transplant,” involves less intensive chemotherapy and radiation treatments.

Researchers now understand that the immune cells created by the transplanted donor stem cells may recognize any remaining cancer cells in the patient as “foreign” and kill them, helping to fight the cancer. This mini-HCT strategy is showing great promise for leukemia and many other cancers and is being used to treat patients into their 80s. Chronic lymphocytic leukemia (CLL), as an example, cannot be cured without a successful stem cell transplant. Because CLL patients are often older than 70, mini-HCT has provided a viable and powerful treatment option in patients with aggressive or late-stage disease.


City of Hope has developed novel ways of delivering radiation utilizing monoclonal antibodies. These antibodies (part of the immune system) can be combined with small amounts of radioactive substances. When administered to the patient, they seek out cancer sites and deliver high-dose radiation directly, while minimizing damage to normal tissues and toxicity to the patient.

Adoptive T Cell Therapy

T-cells are a powerful part of the immune system. A new approach being studied at City of Hope involves redirecting T cells to recognize leukemia cells and destroy them. By targeting microscopic traces of disease, treatment with adoptive T cells may help prevent a relapse. Trials with T cells to treat acute lymphoblastic leukemia are ongoing at City of Hope.

Radiation Therapy

Radiation therapy uses high-energy X-rays or other types of radiation to kill cancer cells. Our Department of Radiation Oncology was the first in the western United States to offer the Helical TomoTherapy Hi-Art System, one of the first radiation therapy systems of its kind to incorporate not only radiation therapy, but also tumor imaging capabilities comparable to a diagnostic computed tomography (CT) scan.
Two types of technology are integrated – spiral CT scanning and intensity modulated radiation therapy (IMRT) – producing hundreds of pencil beams of radiation (each varying in intensity) that rotate spirally around a tumor. The high-dose region of radiation can be shaped or sculpted to fit the exact shape of each patient’s tumor, resulting in more effective and potentially curative doses to the cancer. This also reduces damage to normal tissues and results in fewer complications.