Press Release

240+ New Medicines to Treat Leukemia & Lymphoma

PhRMA April 25, 2013

WASHINGTON, D.C. (April 25, 2013) – America’s biopharmaceutical research companies are developing 241 medicines for blood cancers – leukemia, lymphoma and multiple myeloma, according to a new report and overview released today by the Pharmaceutical Research and Manufacturers of America (PhRMA).

Hematologic (blood) cancers, which include leukemia and lymphoma, can affect everyone, including children. Every year, more than 100,000 cases of blood, bone marrow, and lymph node cancers are diagnosed in the United States, and more than 50,000 people die from these cancers. Among children and teens, leukemia is the most common cancer and the leading cause of cancer death.

Over the last few decades, significant progress in biopharmaceutical research and development has led to steady improvements in cancer survivorship rates in the U.S.

In 1960-1963, five-year survival for non-Hodgkin lymphoma was 40 percent, and for leukemia, just 14 percent. In 2001-2007, they had climbed to 86 percent and 57 percent respectively, according to the Leukemia and Lymphoma Society (LLS). Nearly all of these gains were due to improvements in treatment.

“Complex diseases like blood cancer provide a daunting health care challenge for patients, their doctors and the biopharmaceutical research ecosystem,” said PhRMA President and CEO John J. Castellani. “The more than 240 medicines in development for leukemia, lymphoma and other forms of blood cancer reflect biopharmaceutical research companies’ commitment to build on progress to date and help bring new treatment options to patients.”

The 241 medicines in the pipeline represent new approaches to treating blood cancers, with many using new mechanisms to attack the disease.  These medicines – all in clinical trials or under review by the Food and Drug Administration (FDA) – include: 

  • 98 for lymphoma – including Hodgkin and non-Hodgkin lymphoma – which impacts nearly 80,000 Americans each year.
  • 97 for leukemia, including the four major types, which affect nearly 50,000 people in the United States each year.
  • 52 for multiple myeloma, a cancer of the plasma cells, which affects more than 22,000 people each year in the United States.
  • 24 medicines targeting hematological malignancies, which affect bone marrow, blood and lymph nodes.

“We are advancing breakthrough therapies, particularly in areas of unmet need, by forging partnerships with academic researchers and biopharmaceutical companies, helping to bridge the gap from discovery to drug development,” Richard Winneker, PhD, LLS senior vice president of research. “We have seen many promising advances – LLS-funded therapies that are helping not just blood cancer patients, but patients with other types of cancers and other serious diseases. And we have seen survival rates for many blood cancer patients doubled, tripled, and even quadrupled since the 1960s. But half of blood cancer patients still do not survive, so more research is needed to discover better therapies and help save more lives.”

Hear more from Dr. Winneker on Monday, April 29th at 1:00 p.m. EDT during a panel discussion at PhRMA on the exciting research underway in leukemia and lymphoma, challenges to future progress, the importance of partnerships in medical innovation, and the difference this work is having in extending and improving patients’ lives. The conversation will be streamed live on our web site so interested stakeholders can tune in and engage.

Several key scientific and research trends are having a significant impact on cancer research.  With the understanding that cancer is actually more than 200 separate diseases, targeted therapies and combination treatments are growing in importance. Also, our evolving understanding of the genetic and molecular make-up of cells has helped researchers develop better treatments for blood cancers, including therapies that allow blood cancer patients to recover more quickly after treatment.

Among the potential medicines listed in the report are:

  • A medicine for leukemia that may block the activation of a cell receptor that is mutated in about one-third of all patients with acute myeloid leukemia (AML).
  • A medicine consisting of two components, a toxic portion and an attached trigger molecule, that prevents general toxicity in leukemia and myeloma.
  • A potential first-in class medicine designed to focus the body’s cell-destroying T-cells against cells expressing CD19, a protein found on the surface of B-cell-derived leukemia and lymphoma. 
  • An oral version of an approved injectable medicine that regulates the expression of certain genes such as tumor suppressor genes, to stop the uncontrolled proliferation of malignant cells.

“Research has brought new and improved treatment options to cancer patients, creating hope that didn’t exist before,” said Castellani. “Emerging scientific advances and knowledge are creating unprecedented opportunities for new therapeutic options.”

Demonstrating the clinical benefit of a treatment is an ongoing process in which researchers and clinicians evaluate all aspects of how the medicine is used and how it affects the disease to improve the outcomes of patients. The full impact of a therapy is often recognized only after years of additional post-approval research.  

The Medicines in Development for Leukemia, Lymphoma and Other Blood Cancers report, as well as an overview and other supplementary materials, are available on PhRMA’s website at

Additional information on blood cancer, including recent statistics on incidence, mortality and survival, as well as current information on symptoms, risk factors and treatment options, is available in LLS’ Facts Spring 2013 report.


The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research and biotechnology companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier, and more productive lives. Since 2000, PhRMA member companies have invested approximately $550 billion in the search for new treatments and cures, including an estimated $48.5 billion in 2012 alone.


Find PhRMA Online:

For information on how innovative medicines save lives, visit:
For information on the Partnership for Prescription Assistance, visit:
For information on ensuring the flow of medicines during public health emergencies, visit