A new hope for Lou Gehrig’s disease patient
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Laura Holguin knew long before her diagnosis in May that she was suffering from the early stages of amyotrophic lateral sclerosis (ALS), an incurable neuromuscular disorder named for baseball legend Lou Gehrig. She had seen the symptoms before in her father, who began exhibiting signs in his early 70s but was never officially diagnosed. The “creeping paralysis” of ALS has taken a toll on Holguin, who has slurred speech and problems walking. She uses a wheelchair because of a year-old broken ankle that has not been able to heal properly because of the muscle degeneration she is experiencing. But even in the midst of the changes taking place in her body, the 64-year-old former nurse has not lost her smile or her spirit. “I feel alright. It is not a painful disease,” she said. “But it is no fun to be unable to do activities and things you were able to do quite well before.” Faced with an uncertain future and little in the way of treatment for the disease, Holguin opted to join 20 ALS patients at the Health Science Center in an international study of an experimental drug with the potential to slow down or stop muscular degeneration, and possibly even promote the growth of motor neurons damaged by the disorder. Principal investigator Carlayne Jackson, M.D., a neurologist and associate professor in the Department of Medicine, began working with ALS patients in November on the clinical trial, which will last 18 months and partners the Health Science Center and the South Texas Veterans Health Care System. The drug, a genetically engineered growth factor known as brain-derived neurotrophic factor (BDNF), has shown encouraging results in cell cultures and animal models. Earlier human trials did not fare as well because investigators believe the drug, which was initially injected subcutaneously, was unable to penetrate into the nervous system because of the blood-brain barrier that protects the brain and spinal cord from toxins. In an effort to remedy past problems with drug delivery, the current trial has coupled BDNF with a Medtronic pump system the size of a hockey puck. The device is surgically implanted in the abdomen and filled with medication, which is delivered through a catheter directly into the cerebrospinal fluid. The pump is drained and refilled with doses of BDNF every month. The clinical trial is placebo-controlled, with two-thirds of the patients receiving the drug and the other participants receiving a buffered saline solution. Patient response will be tracked during an 18-month period period and investigators will then determine if the drug and pump systems are safe and effective treatments. Laura Holguin is betting on it. She has a vested interest in the study, not just for herself, but for her six children, who all have a 50 percent chance of being diagnosed with familial ALS later in life. Five percent to 10 percent of all ALS patients suffer from the familial form of the disease, which is passed down from parents to children. “Enrollment in any type of clinical trial provides people with some sense of hope that something is being done and that even if this isn’t the drug that cures this disease, the information we receive will continue to lead us in the direction of finding a treatment,” Dr. Jackson said. “If this drug can help her immediately then that is the real push, but if it can help others in the future—that is ideal,” said Laura’s daughter, Mary Holguin. If the clinical trials prove successful, the drug and pump system may be beneficial in treating other neurological disorders. Dr. Jackson said she believes researchers are closer than ever to a viable treatment for ALS patients, and clinical trials such as this one, are pointing the way ahead. |
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