New Drugs and Therapies at the Forefront of treatment for Acute Lymphoblastic Leukemia

New Drugs and Therapies at the Forefront of treatment for Acute Lymphoblastic Leukemia

J.R.Stokes Illustrations: Natural Killer: T-cell vs. Leukemia
J.R.Stokes Illustrations: Natural Killer: T-cell vs. Leukemia

Researchers received encouraging news on March 22, 2012, with the information that the novel drug to treat acute lymphoblastic leukemia, known as Marqibo won an FDA panel vote. The group of oncology advisers voted 7-4 endorsing the Vincristine Sulfate CAS# 2068-78-2 liposomal injection, also known as the brand Marqibo®, for treatment of ALL. The patients participating in this particular study had a rare form of ALL, and multiple therapeutic attempts had been deemed unsuccessful for them. Of the 65 patients in the single-arm study, 20% experienced a complete or nearly complete response, with a median duration of 5.4 months, and an overall 7.7 month survival rate. This news is the latest in several galvanizing reports released regarding new and innovative treatments for acute lymphoblastic leukemia. In addition to evidence that significant gains have been made in ALL survival rates in children, reports of unique approaches to care using Theranostics have surfaced as well.

Promising news for families with children suffering from acute lymphoblastic leukemia or ALL, was published in Reuters Health on March 16, 2012. Results were released from a study conducted at the University of Colorado School of Medicine and Children’s Hospital, which revealed significant gains in the fight against childhood leukemia have been made. Data gleaned from this study showed that out 20,000 babies, children and adolescents who had ALL, the survival rate of at least five years after the initial diagnosis increased, from 84% in the early 1990’s to 90% a decade later (Figure 1).

Figure 1. Survival rate of 20,000 patients with ALL 5 years after initial diagnosis



This research is hopeful, especially considering the fact that 98% of childhood leukemias are classified as acute lymphocytic leukemia. In addition, 25% of all childhood cancers are leukemia, which amount to roughly 2,200 American children each year. As a cancer of the bone marrow and blood, the child with leukemia has bone marrow which begins to make blood cells that don’t mature properly. These immature cells continue to reproduce, thus crowding out the healthy cells. Effective treatment is not only crucial, it is also completely within reach for scientists today. Only fifty years ago childhood leukemia was considered an incurable disease, and literally all children affected by it did not survive. Drug treatments for ALL are varied, and include cyclophosphamide, which is an alkylating agent;  methotrexate, which is an antimetabolite, and  daunorubicin, classified as both an antibiotic and antineoplastic.

Dr. Stephen Hunger and colleagues, from the University of Colorado School of Medicine and Children’s Hospital in Colorado, reported encouraging results from the Children’s Oncology Group studies between 1990 and 2005. This data included about half of the children in the U.S. diagnosed with leukemia in this time frame. Out of 21,600 kids and adolescents up to age 22, researchers found that 83.7% of the childhood patients diagnosed with leukemia between 1990 and 1994 were alive five years later, and 80.1% were alive ten years later. Reports of improved survival rates for children from the UK and the Netherlands show similar gains in the fight against ALL, but we are still in need of a cure. The sad fact remains that 10% to 15% of children diagnosed each year with ALL will not survive, and more research needs to be completed to make this percentage nonexistent (Figure 2).

Figure 2. 21,600 patients diagnosed with ALL from 1990-1994

5 years after diagnosis10 years after diagnosis


Further gains in research for new and innovative treatments for acute lymphoblastic leukemia have been exhibited as well. A recent report on Daily Rx, dated March 12, 2012, highlighted the new therapies under development at Case Western Reserve University School of Medicine, as published online, March 6, 2012 in ACS Chemical Biology . These therapies involve using personalized treatments and “Theranostic” agents when treating children with ALL. Researchers have discovered a link in 90% of children with ALL-unnaturally high levels of terminal deoxynucleotidyl transferase, also known as TdT. Working on an agent to target this enzyme is at the core of this research, along with continued evaluation of the activities of anti-leukemia molecules in patients. Information gleaned from research shows certain leukemia cells express molecules called CD19 on their surface. This knowledge has led scientists to explore collecting T-cells from the blood of patients, and engineering these cells to express a molecule called CAR, or chimeric antigen receptor. As the CAR molecule easily binds to the CD19 molecule, the CAR T cells can then be infused back into patients. The supposition is that this infusion, which targets the CD19 cancer cells, will create a formidable attack on these cells, thus causing their destruction. These novel and inspiring therapies are being pursued by researchers, who hope to acquire funding for additional research and future clinical trials.

Theranostics can be defined as diagnostics which are closely tied to a specific drug treatment. The FDA will require their use if a specific drug is to be prescribed. An example of this drug diagnostic junction, or theranostic, is the Her-2 neu testing followed with treatment, if appropriate, of Herceptin, or trastuzumab. Another example of a theranostic approach is a C-kit protein test for patients, with a follow up course of treatment with Gleevac, or imatinib, for treating chronic mylogenous leukemia or gastrointestinal stromal tumors.  LGM Pharma supplies APIs to University and research companies who develop new therapies and drugs to help treat these children.  The exploration of personalized medical care through the use of Theranostics is poised to become the next wave of pharmaceutical revelations and customized care.

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