BDNF Trial

Over the last year, we have participated in a treatment trial of ALS with brain derived neurotrophic factor (BDNF). Patients were treated in twenty-one (21) centers with either placebo or one of five dosage regimens. Amgen has indicated to the investigators that they wish to enroll additional patients at the 25 and 100 mcg/kg/day dose. This follow on study seeks to demonstrate the efficacy of BDNF as a treatment for ALS.

There are a number of reasons to be excited about the use of BDNF in the treatment of ALS. BDNF prevents degeneration of motor neurons after axotomy and in recent studies it has slowed the progression of paralysis in the wobbler mouse which is an animal model that shares some characteristics with human ALS.

Treatment (or placebo) will be given for nine months after which all patients will receive BDNF. The diagnosis of amyotrophic lateral sclerosis must fulfill criteria recently established by the World Federation of Neurology.

At the outset, patients will be seen by a study physician to confirm the diagnosis of ALS. Thereafter they will be seen at four week intervals during which time the course of ALS will be carefully quantitated using methods that have been demonstrated by us and others to be a useful paradigm for following patients in clinical trials. The drug will be given daily by subcutaneous injection. No significant side effects are expected.

Patients who have been treated with other growth factors such as ciliary neurotrophic factor (CNTF) or myotrophin (IGF-1) will not be eligible for this study. Also, patients must be under the age of 70 years and in early to moderate stages of the disease.

Patients will need to make visits to our center for this drug trial. The approximate number of visits will be 12 in 10 months. We know this maybe an inconvenience for most patients out of state, but these drugs cannot be shipped through the mail.

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