Understanding and Filtering Information

Understanding and Filtering the Information

Thomas A. Rando, MD, PhD Department of Neurology and Neurological Sciences Stanford University School of Medicine VA Palo Alto Health Care System

CONNECT Conference Parent Project Muscular Dystrophy Denver, CO June 24, 2010

The Information Revolution in DMD 1980 Minimal new information, Limited access

INFORMATION:

1990

2000

Much new information, Limited access

Molecular diagnostics

2010

Much more information, Unlimited access

Experimental Therapeutics

Clinical Trials

Quantity

Email, Listservs Access

Internet Websites

Social Networking

Doctors

Agencies

You Scientists

Friends, Relatives Pharmaceutical Companies

Understanding the Diagnosis: One Step at a Time - Clinical status - Short term: signs and symptoms - Long terms: prognosis

- Laboratory Tests - Short term: supporting the diagnosis, other testing?

- Long term: genetics, carrier status

- Therapeutics - Short term: physical therapy, steroids?

- Long term: other medical/surgical, clinical trials

What You Should Expect from Your Doctor

-  A clear explanation of the clinical diagnosis and prognosis;

What You Should Expect from Your Doctor

- A clear explanation of the clinical diagnosis and prognosis; - A general explanation of what the tests showed;

What You Should Expect from Your Doctor

- A clear explanation of the clinical diagnosis and prognosis; - A general explanation of what the tests showed; - Time to answer your questions of each of those;

What You Should Expect from Your Doctor

- A clear explanation of the clinical diagnosis and prognosis; - A general explanation of what the tests showed; - Time to answer your questions of each of those; - A near-term plan for further tests and any therapy;

What You Should Expect from Your Doctor

- A clear explanation of the clinical diagnosis and prognosis; - A general explanation of what the tests showed; - Time to answer your questions of each of those; - A near-term plan for further tests and any therapy; - Suggestions of where to go for further information;

What You Should NOT Expect from Your Doctor (especially if you are not being seen in an MDA Clinic)

-  A detailed understanding of the genetics

What You Should NOT Expect from Your Doctor (especially if you are not being seen in an MDA Clinic)

- A detailed understanding of the genetics - A specialist’s understanding of the test results

What You Should NOT Expect from Your Doctor (especially if you are not being seen in an MDA Clinic)

- A detailed understanding of the genetics - A specialist’s understanding of the test results - A familiarity with the clinical trials that are underway or planned

Where to Obtain the Best Information Parent Project Muscular Dystrophy: http://www.parentprojectmd.org

Muscular Dystrophy Association: http://www.mdausa.org

Muscular Dystrophy Campaign: http://www.muscular-dystrophy.org

Filtering the “Information�

Most accessible, Fewest details

Press Releases

Research Announcements

Journal Articles

Least accessible, Most details

Interpreting Press Releases Stem cell hope for Duchenne muscular dystrophy Posted on July 17, 2008 by speakingofresearch

Duchenne muscular dystrophy (DMD) is an inherited disease that affects about one in every 4,000 males born in the USA. It is caused by mutations in the DMD gene that lead to the protein dystrophin being either absent or faulty, which leads to muscle cell death, progressive muscle wasting and early death, with few patients surviving beyond their 40th birthday.

In recent years scientists have been investigating the possibility of transplanting healthy stem cells into the muscle of patients in order to replace the lost muscle cells and halt the progression of the disease, but it has proved difficult to identify muscle precursor cells that can both make new muscle cells and persist as a pool of precursor cells in the patient, the latter is an important consideration if repeated transplants are to be avoided since muscle cells wear out and need to be replaced. A paper published in this weeks issue of the scientific journal Cell by scientists at Harvard University is an important step towards developing a means to screen for the right cells and use them to treat DMD

Understanding Research Announcements

Assessing Journal Articles

Understanding the “Information�

Most accessible, Fewest details

Press Releases

Research Announcements

Journal Articles

Least accessible, Most details

Information Overload

-  Don’t try to become a neurologist/geneticist/cell biologist

Information Overload

- Don’t try to become a neurologist/geneticist/cell biologist - Understand the process from the laboratory to the clinic

Information Overload

- Don’t try to become a neurologist/geneticist/cell biologist - Understand the process from the laboratory to the clinic - Focus on issues related to current treatments (e.g. steroids)

Information Overload

- Don’t try to become a neurologist/geneticist/cell biologist - Understand the process from the laboratory to the clinic - Focus on issues related to current treatments (e.g. steroids) - Find the sources you trust for information on long term issues

Information Overload

Information is valuable.

Time is priceless.