DRUGS FOR ALS (Being Tested and on the Market) |
| DRUG |
MAKER(S) |
TYPE OF DRUG |
HOW IT WORKS |
STAGE OF DEVELOPMENT |
COMMENTS |
| BDNF (Brain-Derived Neurotrophic Factor) |
Amgen, Regeneron, Medtronic |
Neurotrophic Factor |
Shown to preserve motor neurons in laboratory situations where they would otherwise die. |
Being tested in ALS patients in multicenter clinical trial with delivery into cerebrospinal fluid (intrathecal delivery); small trial of subcutaneous delivery in planning stage. Intrathecal trial is open for enrollment. See [chaperones] |
An earlier, large trial of BDNF delivered subcutaneously wasn’t effective. Amgen and Regeneron are now testing the drug by putting it directly into the cerebrospinal fluid. The intrathecal pump is made by Medtronic. Amgen and Regeneron also plan to test subcutaneous delivery in an undefined subgroup of ALS patients whom they believe may benefit from this delivery method. |
| GDNF (Glial-Derived Neurotrophic Factor) |
Amgen |
Neurotrophic Factor |
Shown to preserve motor neurons in laboratory situations where they would otherwise die. |
Being tested in ALS patients. Delivery is directly into brain. Results expected by end of this year. Trial enrollment now closed. |
Amgen is also testing GDNF in Parkinson’s disease. |
| Myotrophin |
Cephalon, Chiron |
Neurotrophic Factor |
Derived from the natural neurotrophic factor IGF-1 (insulin-like growth factor 1). This factor shown to preserve motor neurons in laboratory situations where they would otherwise die. Administered by subcutaneous injection. |
Brought before an FDA advisory panel in 1996 and again in 1997. Panel recommended denial of permission to market for ALS both times, but FDA allowed Cephalon to distribute free to ALS patients by random selection in an "expanded access program." Still available through Cephalon to patients who are already in program but not to new patients. |
Main concern of FDA panels were data
from two large trials in ALS patients, one in North America and
one in Europe. North American trial showed some slowing of functional
deterioration in ALS but European trial showed no benefit and raised
questions about safety. A company spokesman says situation is uncertain
and that "ball is in the FDA’s court." Call
Cephalon at (800) 896-5855 about the expanded access program. |
| SR57746A |
Sanofi |
Neurotrophic Factor ‘Booster’ |
Theoretically, boosts body’s production of neurotrophic factors. Administered orally. |
Being tested in ALS patients in large, multicenter trial. Enrollment closed since mid-1998. Some patients are now taking drug on open-label basis (they know they’re getting drug and not placebo). Some results expected by end 2000. |
Increasing body’s own production of neurotrophic factors, if possible, would be an advantage over delivering these factors as drugs. A major problem with giving these large molecules is that they don’t readily enter the central nervous system. |
| Riluzole (brand name Rilutek) |
Rhone-Poulenc Rorer (RPR) |
Glutamate Inhibitor (Partial Blocker) |
Inhibits actions of glutamate by interfering with release from cells where it’s made and docking on target cells (at cellular receptors). Administered orally. |
On market since 1996. Rilutek is only FDA-approved drug for treatment of ALS. |
Rilutek showed modest prolongation of life in ALS patients in very large U.S. and European trials. Liver toxicity is a frequent side effect. Regular blood tests to check for this are needed. Cost is about $700 a month. Usually covered by insurance, since approved for ALS treatment. RPR runs patient assistance program through Athena Home Pharmacy to help find financial aid. Call (800) 528-4362. |
| Gabapentin (brand name Neurontin) |
Parke-Davis |
Glutamate Inhibitor |
Probably partially blocks glutamate synthesis. |
On market for epilepsy. Available to ALS patients as an "off-label" prescription. Being tested in large, multicenter trial in ALS patients after pilot trial showed promise. Results are expected late this year. |
Neurontin isn’t FDA-approved for ALS, but doctors can prescribe it, since it’s on the market. This is called an "off-label" prescription, and it’s legal, but slightly risky, for the doctor and patient if something should go wrong. Neurontin is also expensive -- estimates are about $260 a month, depending on the dosage. Parke-Davis has a patient assistance program to help find financial assistance for Neurontin. Call (908) 725-1247. |
| LY300164 |
Eli Lilly |
Glutamate Inhibitor |
Blocks glutamate docking sites (receptors) on target cells. |
Being tested in small clinical trial. Enrollment is complete. Some results expected in July. |
This is another strategy to block excess glutamate. Little data available at present. |
