Advocacy > Recipe for Progress

Last update: 02/15/2005

Treatments | Improving Drug Development and Assessment | The Essential Role of the NCI

Survival statistics tell us that the treatments we have are not good enough.  We continue to lose those we love.  We continue to suffer through painful and toxic therapies. And our society suffers as patients with lymphoma become less productive and less able to earn a living and make contributions to their communities. 

Concerning current treatments for lymphoma, we believe that the gap between what is and what's possible has never been wider. So we dedicate this page to listing the abundant opportunities for success, and to remind all of the urgent need to seize them.

 

Treatments:

There is an urgent need to develop and clinically test new treatments that:

• have greater specificity, more potency, and less toxicity;

• can overcome bulky disease;

• can selectively kill indolent lymphoma cells, that are not in cell cycle;

• can induce and enhance active immunity - the way we naturally fight disease;

• combine targeted therapies that may complement each other in order to minimize toxicity and prevent tumor escape; 

• are rationally selected to match the differences in the disease by using genetic and metabolic profiling; 
  (please review information about the National Biospecimen Network an innovative blueprint for progress)

• are safer, thus allowing patients with indolent lymphomas to safely receive treatment early and more regularly;

• that can be safely combined with existing treatments in order to cure indolent lymphomas, and more patients with aggressive disease.

• that address the role of the microenvironment: (1) Altering the host environment in ways that silence or inhibit pro-survival signals concurrent with standard therapies. (2) Focusing, and expanding immune surveillance against tumors. (3) Turning off signals and inhibiting cells that contribute to drug resistance, depressed immunity and escape from immunity. (4) Targeting tumors in ways that minimize damage to immunity, or enhance it.  (5) Sequencing therapies in ways that restore positive elements of the microenvironment in order to consolidate the response to treatments. 
    
    

Improving Drug Development and Assessment:

• Ensure that there are sufficient incentives for pharmaceutical companies to take the financial risks to 
  develop and test therapies for cancer.  
   
  • Consider reducing incentives to develop "me-too" drugs for conditions that 
    are not life-threatening.
     
• Ensure that the FDA considers factors such as lower toxicity and improvements in the quality of life benefits in the 
  assessment of new therapies.
   
  • Encourage drug sponsors to properly account for and measure toxicities and quality of life in 
    clinical trials.
     
• Increase patient participation in clinical trials - an essential ingredient:
   
  • Encourage drug sponsor to consult with informed patients and treating physicians 
    to help design clinical studies that are in harmony with patients survival goals.
     
  • Educate the communities' doctors and patients about what studies are available, and which show promise for specific treatment settings.
     
  • Encourage treating physicians to discuss all appropriate therapies, including investigational therapies, 
    during treatment consults.
     
  • Ensure that tests associated with clinical trials are covered by government and / or insurance.
      
  • Provide, when possible, predictive tests to determine who is more likely to benefit from an 
    investigational therapy and/or a standard therapy used as a pretreatment in studies.
      
    • For example, include tests for polymorphisms in studies that utilize Rituxan.
      
• Encourage drug sponsors to test investigational bio- and immune-based therapies earlier in the course 
  of the disease, when they are more likely to provide benefit - especially for indolent lymphomas.
   
  • Ensure that watch & wait should not be routine.  Periods of stable disease provide an important 
    opportunity to test immune-based therapies and other more targeted therapies with safer toxicity profiles.
    We must encourage drug sponsors, patients, and treating physicians to use this opportunity:
     
    • Better immune competence
    • Less tumor burden
    • Less exposure to toxic treatments
    • Potential to improve quality of life
    • Potential to learn without precluding the use of standard treatments

The Essential Role of the National Cancer Institute (NCI):

• Ensure that funding of cancer research becomes a national priority.
   
  • Encourage all cancer groups to work cooperatively in this effort.
     
• Encourage the NCI to carry out its promise to conduct translational research in order to validate 
  and clinically test promising new therapeutics. 
   
• Ensure that gene-profiling technologies are fully funded and applied as quickly as possible.  
   
  We support the following rationale:
   
  • Enables scientist to more rapidly identify the gene expressions that distinguish normal from 
    malignant cells and assist in the "development of molecularly targeted therapies that have specificity 
    and potency for defined cancer types."
       
  • Enables scientists to link clinical behavior (how aggressive or indolent the cancer is likely to be) 
    to gene expression, and thus better advise patients about treatment selection and timing. 
    (Currently, the variable clinical course of patients given the same diagnosis stems, in part, from 
    the underlying molecular diversity among their tumors.)
      
  • Enables identification of viral factors that may be present and may contribute to causing, 
    promoting, or maintaining malignant behavior of lymphoma cells.
      
  • Enables scientists to discover how genes function in immune cells and apply that knowledge to 
    the treatment of numerous diseases. 
     
  • Enables retrospective testing to genetic expression with individual responses to treatments. 
     
    The potential benefits will enable researchers and/or doctors to:
     
    • select treatments that are far more likely to benefit the patients
    • spare patients from the side effects of ineffective treatments
    • identify patients in need of experimental approaches to treatment
    • reduce health costs by enabling rational treatment selection, instead of by wasteful trial and error
    • identify genes in tumors that lead to treatment resistance
    • select patients most likely to benefit from investigational drugs in clinical trials

    “Presently we are overlooking effective treatments for some people, while administering ineffective 
    and toxic treatments to others based on studies conducted with ill-defined aggregates of patients.”  – DF.  

     

  • Enable testing of multiple samples from the same patient, in order to:
     
    • identify changes to gene expression and other tumor characteristics in response to 
      treatments and over time.
    • identify changes to gene expression and other tumor characteristics that correlate to refractory
      disease, or transformation to aggressive disease, or the lack thereof.
         
  • Enable identification of tumor-associated antigens that can form the basis for therapeutic 
    cancer vaccines.
       
  • Enable discovery of cells in the lymphoid microenvironment that may be promoting or inhibiting 
    lymphomas.
       
  • Enable production of patient-specific therapeutics, such as cancer vaccines or therapeutic antibodies.
     
• Consider providing financial incentives to managers that bring new therapeutics to cancer patients, 
  and to scientist who make key discoveries to make this possible.  
• Encourage clinical studies of the many promising therapeutics and adjuvants that are not of 
  commercial interest, such as:
   
  • Combinations and sequences of agents that may complement one another.  
      
  • Immune-based strategies that have low toxicity on patients with indolent lymphomas.
     
  • Natural compounds that have potential in combination with chemotherapies, such as fish oil and melatonin.
    
• Last, but NOT least: Design compassionate use protocols in advance - anticipating the needs of patients who fail standard therapies, and are ineligible for clinical trials - so we can give patients every reasonable chance to survive, and also test new concepts in a timely manner.