MIAMI, April 2 /PRNewswire/ -- Reversal of Lou Gehrig's disease may now be possible using thrombopoietin and thyroid hormone to cause regeneration of endogenous stem cells of the central nervous system. Evidence suggests spinal cord injury can be reversed through regeneration by stem cells. This research will be presented by George R. Schwartz, M.D., a senior researcher at Neuroregeneron Co., this week at the annual meeting of the American Spinal Injury Association held in Miami, Florida.
The Food and Drug Administration (FDA) biologics division approved a unique clinical trial in May 2002, authorizing the use of thrombopoietin for Lou Gehrig's disease (amyotrophic lateral sclerosis) in a 40-year-old mother of three small children whose clinical condition was deteriorating rapidly. She was more than 90% paralyzed, with minimal speech capability, tremendous difficulty swallowing, and rapidly failing respiratory function.
The new treatment was approved for a clinical trial after it was demonstrated that platelet growth factors could be tremendously increased through use of thrombopoietin. Platelet growth factors act as stimulants for the growth and development of glial cells which act as repair cells for dying nerve cells. In addition, platelet growth factors can stimulate immature cells to differentiate into cells which act as neurons. Thyroid hormone was added to the trial after experimental evidence demonstrated that thyroid hormone acted as a signaling substance helpful for repair cells to function.
Platelets were raised in cycles to more than 10 times the normal level resulting is blood serum rich in platelet growth factors.
At day 42 of this clinical trial, this patient showed remarkably improved head and neck control and strength. At day 45, she exhibited improvement in tongue strength and motion with improved swallowing functions. As a result, a feeding tube was not necessary.
At day 60, increased leg muscle strength was clearly evident. Along with this motion, the patient was able to turn her arms and hands which had been paralyzed for more than a year. At day 110, she began to move her hands. Muscle strength throughout her body increased and her pelvic muscles could support more weight.
The patient showed clear reversal of a previously deteriorating condition, and return of functions. Her downhill course stopped.
The nerve cell regeneration and reversal of paralysis in this patient with Lou Gehrig's disease suggests that spinal cord injury and paralysis can also be treated with re-growth of the nerve cells of the spinal cord.
Further trials are urgently needed since the average length of life in ALS patients is 3-5 years after diagnosis. There is also some indication that regeneration in cases of spinal cord injury would be more effective soon after the injury.
The FDA has been extremely supportive of this clinical trial and has urged that other trials be conducted as soon as possible. "If this proves out, it is a very exciting result indicating a new treatment and approach to Lou Gehrig's disease and spinal cord injury," said a senior neurologist with the FDA in Rockville, Maryland.
However, despite the encouraging results and excitement generated by this clinical trial, the Genentech company has decided not to release the drug thrombopoietin for any further trials in neurologic disease or injury.
"We will not proceed with any further trials at this time," said Mary Stutts, director of corporate relations at Genentech. The medication was manufactured in substantial quantity in the late 1990s and the current stock of clinical grade thrombopoietin will expire in the year 2003. "Remanufacture is not planned at this time," confirmed Heather Mccauley, spokeswoman at Genentech. She offered no other explanation for the decision not to conduct any further clinical testing.
A director of the Lou Gehrig's clinic at the Massachusetts General Hospital and a professor at the Harvard Medical School, has prepared a trial for ten people with this disease. "I am totally puzzled," commented the doctor, who was rebuffed when he approached Genentech with his proposal. "This defies all common sense and scientific responsibility. We have no other treatments for these conditions," he explained.
Dr. Schwartz, who has been following his patient closely with the FDA approved trial, is also puzzled. "Are they blind to the implications of this drug for use in neurologic diseases or injury?" he remarked.
Monica Collier, one of the researchers who has been following this patient's ground-breaking clinical course, expressed amazement at the lack of compassion shown by the spokespeople at Genentech. "I cannot understand their approach," she said. "It would seem to be in their interest to try to develop this medication, and the patients just cannot wait."
Dr. Schwartz added, "I know the people at Genentech would be happier if there was a large amount of animal experimentation before the clinical trial. However, the reality is that animal models are not suitable to test for regeneration of nervous system cells at this time. We have a treatment which is ground-breaking and which is working in our patient. Let us go forward with further testing. Re-manufacture will take years. Meanwhile all the medication for clinical testing is literally going to waste. Patients are suffering and family and spouses are watching tragic deterioration in their loved ones."