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    Norris Center Clinical Trials

    We are dedicated to finding the cause and a cure for ALS.

    A Cautionary Note
    Some ALS drug studies are using medications that are already on the market for another purpose. In two previous such studies, the medication actually caused further harm. Most recently, minocycline caused patients to worsen 25% faster than the group that took placebo. Until a study is complete and the results are known to be positive, it is NOT a good idea to ask your doctor for a prescription for a drug in study.

    Qualifications for Participation
    There are criteria that must be met for each different study. Generally, you cannot have had ALS symptoms for more than 3 years and your breathing must be quite good, usually above 75%. The criteria are specific for each study, so we welcome your phone call to learn more.

    See all of our current clinical trials.

    Lithium  |  KNS-760704  |  Talampanel  |  Ceftriaxone  |  SB509 or VEGF


    A small pilot study of lithium in Italy last winter got a lot of exciting press. However, some of that press was misleading. A careful reading of the Italian article shows that lithium does not stop ALS, but the 16 patients who took lithium did not progress as quickly as the 28 patients who took placebo. The study had too few patients in it to be statistically meaningful. The current study is trying to see if lithium really does show promise in ALS.

    Lithium is FDA approved for use in bipolar disorder, depression and manic episodes. It has shown effectiveness in protecting nerves in epilepsy and other disorders. The hope is that lithium will protect motor nerves from ALS.

    The Forbes Norris ALS Center, with funding from the Muscular Dystrophy Association, has taken the lead in organizing this study for collaboration with 10 ALS centers nationwide. The study will last one year and is already filled to capacity.

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    Knopp Pharmaceuticals is funding a national study of KNS-760704. The drug is very similar to Mirapex (pramipexole), a drug that works well for Parkinson’s. Parkinson’s and ALS are both diseases in which specific nerves are damaged, degenerate and die. Knopp has modified Mirapex so that it is stronger and has fewer side effects.

    This study lasts for 10 months. In the first part (3 months), only one group out of the four study groups takes placebo. In the second part (7 months), everyone gets the real drug. This study is currently enrolling patients. For information, please contact Marguerite Engel at 415-600-3758 or

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    TEVA Pharmaceuticals is funding a national study of Talampanel. This drug belongs to the family of drugs called “AMPA receptor antagonists” which has shown great promise in the laboratory. It is also being studied in the treatment of brain tumors. A receptor receives and responds to messenger molecules. Some messenger molecules are thought to be toxic in ALS. Thus, a receptor “antagonist” like talampanel may block these toxins from binding to nerve cells.

    TEVA Pharm tested talampanel in 59 ALS patients and noted a trend towards a lower rate of deterioration in ALS symptoms. They now want to test this drug in a larger group of people with ALS to see if it is safe and if it does slow deterioration.

    The study lasts for one year. 60% of patients will get the real drug and 40% will get placebo. After the initial start-up period, visits are about every three months. This study is currently enrolling patients. For information, please contact Marguerite Engel at 415-600-3758 or

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    Ceftriaxone is a powerful antibiotic that is FDA approved for infection. In the study for ALS, ceftriaxone is being used at a higher dose than for infection and for a longer period of time. It is thought that this might have an effect on the inflammatory process that occurs in ALS nerve cells. The first phase of the study showed that it is safe in people with ALS. The second phase of the study will start in December or January and is aimed at seeing if it slows progression in ALS. We will begin to enroll patients in January for the second phase

    This trial, funded by the National Institutes of Health (NIH), is a difficult, complex study that is for only patients who want to be very aggressive in pursuit of treatment. The medication is given through a special catheter in the chest that is threaded into the jugular (neck) vein. The patient’s caregiver (usually the spouse) gives the medicine twice a day. The regular research visits are monthly and the study lasts for a year. For information, please contact Marguerite Engel at 415-600-3758 or
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    SB509 or VEGF

    Sangamo Pharmaceuticals is funding a study of SB509 or VEGF, a body protein that is important for cell growth and maintenance. VEGF has been found to be low in people with ALS. SB509 is aimed at increasing VEGF proteins in ALS patients’ so that protection and repair of damaged motor nerves could occur.

    This, too, is a very difficult and complex study and is only for people who want to be very aggressive pursuing treatment possibilities. Using a very tiny needle, the drug is injected into many muscles fibers of the neck, arms and legs. There will be two sets of injections given three months apart.

    The study lasts for 11 months with visits about every month. Everyone in the study will receive real drug. There is no placebo. We anticipate enrolling patients in December or January. For more information, contact Giovanna Kushner at 415-600-3983 or
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