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Acute Lymphocytic Leukemia - Unmet Needs in Adult and Pediatric Populations Drive Recent Market Developments


Description: The heterogeneity of acute lymphocytic leukemia (ALL) makes a universal treatment for the disease extremely difficult to discover and develop: because pediatric and adult ALL are biologically and clinically distinct, treatments that work for one group of patients will not necessarily work for others. For example, effective pediatric ALL treatments have not yielded the same results in adult ALL patients. This treatment gap is compounded by lack of understanding of the etiology of the disease.

Although recent drug approvals in ALL will help certain niche populations of patients, until the causes of the disease are better understood, treatments better tailored to adult ALL patients will be lacking.

Questions answered in this report:

- ALL is the most common pediatric cancer. How common is the disease in non-pediatric populations? What is the incidence of ALL in the overall population?

- Despite successes in treating pediatric ALL, effective adult ALL treatments are still lacking. What are some of the underlying causes of the diffi culty in treating adult ALL?

- ALL treatment is considered the most complicated of all hematological cancer treatments. What factors make ALL treatment so complex?

- The clinical ALL pipeline is less robust than pipelines for other acute leukemias, such as acute myelogenous leukemia (AML). What are the reasons for the low number of ALL pipeline agents? What are the leading agents in development?

- Despite the diffi culties in treating ALL, four therapies have recently received marketing approval in this indication. What are these four therapies? For which ALL populations did they receive marketing approval?

Scope of the report:

- Markets covered: United States, France, Germany, Italy, Spain, United Kingdom, and Japan.

- Primary research: Survey of 50 U.S. hematological oncologists, conducted in January 2010.

- Epidemiology: Diagnosed incident ALL cases; treatable pool of ALL cases; forecast of cases from 2008-2018.

- Prognostic indicators: Age at diagnosis; white blood cell count at presentation; immunophenotype; cytogenetic abnormalities; early response to therapy and minimal residual disease (MRD); in vitro response to chemotherapy.

- Emerging therapies: Phase II: 5 drugs; Phase III: 3 drugs. Identifi cation of 2 select Phase I and Phase I/II drugs.

Key terms for this report:

Acute lymphocytic leukemia (AML), Acute lymphoblastic leukemia (ALL), Adult leukemia, Arranon, Biomarkers, Chronic therapy, Clolar, Gleevec, Hematological oncology, Pediatric leukemia, Targeted therapies


Contents: Executive Summary
- Strategic Considerations
- Stakeholder Implications

Introduction

Pathophysiology, Diagnosis, and Etiology
- Pathophysiology and Symptoms
- Diagnosis and Classification
-- Cell Morphology
-- Immunophenotype
-- Cytogenetics
- Biomarkers
- Prognostic Factors
-- Age at Diagnosis
-- White Blood Cell Count at Presentation
-- Immunophenotype
-- Cytogenetic Abnormalities
-- Early Response to Therapy and Minimal Residual Disease
-- In Vitro Response to Chemotherapy
-- Central Nervous System Status at Diagnosis
- Risk Stratification
- Etiology
-- Genetic Abnormalities
-- Environmental Exposures
-- Other Factors

Epidemiology
- Overview
- Methods
- Subpopulations
- Diagnosed and Drug-Treated Populations
-- Percentage Diagnosed
-- Percentage Drug-Treated

Current Therapies
- Overview
- Comparison of Key Current Therapies
-- Induction Therapy
-- Consolidation Therapy
-- Maintenance Therapy
-- Relapsed or Refractory Acute Lymphocytic Leukemia
-- Genzyme’s Clolar/Evoltra (Clofarabine)
-- GlaxoSmithKline’s Arranon (Nelarabine)
-- Tyrosine Kinase Inhibitors Gleevec (Imatinib) and Sprycel (Dasatinib)
-- Central Nervous System Prophylaxis
- Drug Resistance
- Stem Cell Transplantation
-- Myeloablative Regimens
-- Allogeneic Stem Cell Transplantation
-- Autologous Stem Cell Transplantation

Emerging Therapies

Challenges and Opportunities for the ALL Market
- Agents to Prevent and Treat Relapse
- Targeted Therapies for ALL Subpopulations
- More Therapeutic Options for Elderly Patients

Bibliography

Tables:
1. Immunophenotype of Acute Lymphoblastic Leukemia
2. Risk Stratifi cation for Adult Acute Lymphoblastic Leukemia
3. Children’s Oncology Group Risk Stratifi cation for Pediatric Acute Lymphoblastic Leukemia
4. Genetic Abnormalities Associated with Acute Lymphoblastic Leukemia
5. Leading Drugs Used for Acute Lymphoblastic Leukemia, 2010
6. Leading Drugs Used for Acute Lymphoblastic Leukemia—Mechanism of Action
7. Survey Question: Which of the Following Attributes Are Most Important to Your Prescribing Decision?
8. Emerging Therapies for the Treatment of Acute Lymphoblastic Leukemia, 2010

Figures:
1. The Hematopoietic Cascade: Development of Mature Blood Cells from Pluripotent Hematopoietic Stem Cells
2. Survey Question: What Percentage of Your ALL Patients Experience the Following Symptoms?
3. Number of ALL Incident Cases in the United States, Europe, and Japan, 2008-2018
4. Total Treatable Pool of ALL Cases in the United States, Europe, and Japan, 2008-2018
5. Survey Question: How Many of Your ALL Patients Move to/Add on a Second-Line Therapy Within These Time Frames Because of Relapsed or Refractory ALL?
6. Survey Question: Among Your ALL Patients Who Proceed to Second-Line Therapy (Whether Switching or in Addition to First-Line Therapy), in What Percentage of Patients Do the Following Factors Trigger the Move to Second Line?
7. Survey Question: What Percentage of Your ALL Patients Experience These Side Effects as a Direct Result of Treatment?


Companies Mentioned - Baxter Laboratories - Bedford Labs - Bristol-Myers Squibb - Cephalon - Gate Pharmaceuticals - Genzyme - GlaxoSmithKline - Meiji Seika - Merck & Co. - Merck Serono - Novartis - Pfizer - Sanofi-Aventis - Shionogi


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