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disease, but the patient who is just beginning to show the early symptoms. Can we arrest the development of the disease? Can we stop it before it becomes incapacitating? Again, I remind you, once a brain cell is dead, it is gone forever.
We have evaluated one drug which was released last year called deprenyl which seems to arrest the progress of Parkinson's disease. We are following patients on that drug because the issue is, can we delay the need for use of L Dopa. Patients with advanced Parkinson's disease get the drug L Dopa, but there are side effects to L Dopa. And the thought is can we delay the progression of the disease so that they do not need L Dopa for 2, 3, 4, or 5 years. And the answer is, yes. The drug is now available.
What we are trying to do now is improve on that drug. It does not work on Alzheimer's disease.
ALZHEIMER'S DISEASE COORDINATION Senator HARKIN. We have two back-to-back votes.
I had one other question, Dr. Goldstein, on Alzheimer's. Your Institute got a $12 million increase for Alzheimer's research in 1991, a total of $34.5 million.
Dr. GOLDSTEIN. Yes, sir.
Senator HARKIN. There were eight other institutes that spend funds on Alzheimer's research as well as the National Institute of Mental Health.
Dr. GOLDSTEIN. Yes, sir.
Senator HARKIN. It has been suggested to this committee that a new office to coordinate Alzheimer's research may be useful. What is your view? Do we need an office to coordinate Alzheimer's research?
Dr. GOLDSTEIN. Sir, in terms of the Federal establishment and particularly the NIH institutes and NIMH, we already have that coordination in operation. In the Office of the Assistant Secretary, a committee is functioning which does coordinate the national Alzheimer's disease effort. My colleague, Dr. Williams, is a key person in the running of that committee. I am not really sure what another layer of bureaucracy would accomplish which that com isn't already doing very effectively. I already see more of Dr. Williams about Alzheimer's disease than I want to, sir. (Laughter.]
Dr. WILLIAMS. It's a pleasure. (Laughter.)
Senator SPECTER. Mr. Chairman, we have a full Appropriations Committee markup at 2:30, had you heard, on the appropriations bill? Senator HARKIN. Well, I was told.
GAUCHER'S DISEASE Senator SPECTER. I wonder if I might ask Dr. Goldstein just one question. I had written you, Dr. Goldstein, back on February 11, concerning Gaucher's disease type III research.
Dr. GOLDSTEIN. Yes, sir.
Senator SPECTER. And I had requested a response prior to the hearing today. And we did receive a telephone call yesterday about it. But the question I have is was there insufficient time for you to respond in writing in the intervening month?
Dr. GOLDSTEIN. Yes, sir; the problem is that there is a research protocol that has to be developed specifically for type III. It is a somewhat different disease from type I, and we couldn't use the type I research protocol. We have been diligently trying to get the details of that protocol developed and introduce it into the scientific review process. I apologize that we were able to get our act together only yesterday. But it was too late to write you, and that is why we called.
Senator SPECTER. Well, if you have a problem on getting the information together in the interim, say from the February 11, 1991, letter to the March 14, 1991, hearing date, I would just appreciate it if you would drop us a note and tell us what the progress is.
Dr. GOLDSTEIN. You will absolutely have the information this coming week, sir.
Senator SPECTER. Well, my point is to let us know, if you could, in writing in advance of the hearing date
Dr. GOLDSTEIN. Yes, sir.
Senator HARKIN. I do have a problem. I was just informed not more than 30 minutes ago that we have a full committee markup on the supplemental at 2:30.
Senator SPECTER. Yes, that's what I commented. I regret it myself, but we are at the call of the Chair. And when the Chair calls the full committee, the full committee responds.
Senator HARKIN. I assume that we will get a quorum. I had to cancel next Tuesday, the incoming Secretary of Education, because of the delay and everything, and we are rescheduling him in April sometime. I do have some time on Tuesday morning, Dr. Raub, that we could use to finish.
We have to go vote. We have two back-to-back votes. I will have someone go with me. If I see my way clear to get back here in about 20 minutes, I may ask you to stay. And if it looks like that I have to be there for the opening of the full committee, I will send word back and we will just dismiss you and see if you could come back next Tuesday morning.
Dr. RAUB. Fine. We are here at your pleasure.
Senator HARKIN. So, if you can give us about 20 minutes, I will know in about 20 minutes whether
I can come back or not.
Senator HARKIN. The subcommittee will resume its sitting. I appreciate your staying here. We had two votes.
I thank you for the applause, but it really should be for Senator Byrd. I went to him and said we had all of you here and you came all the way down from Bethesda and Rockville and down here from NIH. I said we cannot have them turn around and come back, waste gas and time, all these important scientists. So, he said, OK, we'll bring you up first. So, we got up first and got out of there. So, that is why I am here. The rest of them are stuck over there till about 7 tonight I think. (Laughter.]
Actually, I want to thank you also. I didn't want to sit there till 7 either. (Laughter.)
Dr. GOLDSTEIN. I was about to say you owe us one, Senator.
Senator HARKIN. I owe you one. That's right. (Laughter.)
QUESTIONS SUBMITTED BY THE SUBCOMMITTEE I have some more questions, Dr. Goldstein, for you, but we will submit those for the record. OK?
[The following questions were not asked at the hearing, but were submitted to the Institute for response subsequent to the hearing:) QUESTIONS SUBMITTED BY THE SUBCOMMITTEE
NERVE CELL DEATH--TRAUMA VS. DISEASE
Question. Dr. Goldstein, I understand that nerve cells that have been damaged by trauma can repair themselves especially if the head and spinal cord injury patient is treated within the first 4 to 8 hours.
What kinds of treatments are most effective for head and spinal cord injury patients?
Answer. Findings from an NINDS-supported clinical trial demonstrate that high-dose methylprednisolone, given within 8 hours of an acute spinal cord injury, can reduce the extent of secondary damage to the nervous system. Methylprednisolone is the first treatment to improve the outcome of an acute spinal cord injury. How methylprednisolone is able to exert this effect is still not clear. NINDS is actively pursuing laboratory and clinical studies to develop further this treatment and identify similar strategies for head injury.
Question. What is it about early treatment that is so critical?
Answer. Within the first hours following severe head or spinal cord trauma, & cascade of biochemical events can occur to make the injury worse (secondary injury). As demonstrated by an NINDSgupported clinical trial, high-dose methylprednisolone given within 8 hours of an acute spinal cord injury can reduce the extent of this secondary damage to the nervous system.
Question. Does this suggest that early intervention may be successful for Alzheimer's and Parkinson's disease patients?
Answer. The results from the methylprednisolone trial are not directly applicable to chronic degenerative disorders such as Alzheimer's disease and Parkinson's disease. However, we have evidence from another NINDS-supported clinical trial that treatment with deprenyl in early Parkinson's disease may enable patients to live and work normally for a longer period of time before requiring more aggressive therapies. This trial is ongoing to evaluate the long term effects of deprenyl and the value of another agent, tocopherol, in combination with deprenyl. The results so far are encouraging; we may be able to move beyond symptomatic treatment into ways to actually intervene and arrest or reverse the underlying process of many neurological diseases.
Question. Your institute and the Aging Institute are the two big players in Alzheimer's research. How do you distinguish the Alzheimer's research sponsored by the Neurological Institute from that sponsored by the Aging Institute?
Answer. As part of its mission, NINDS focuses on understanding the fundamental etiology and underlying pathogenesis of Alzheimer's disease and related disorders. The NINDS is responsible for pioneering research efforts in understanding how the brain is
changing during the earliest stages of Alzheimer's disease and relating these findings to ongoing genetic, metabolic, and cellular studies.
The National Institute on Aging research efforts emphasize diagnosis, developing treatment and management strategies, and educating the lay and professional audience with regard to the disease.
Question. I understand that a relatively new discovery in the field of Multiple Sclerosis (MS) is that the disease results in the failure of the blood/brain barrier.
Please discuss these findings. What implications does it have for additional MS research?
Answer. Magnetic resonance imaging (MRI) studies of multiple sclerosis (MS) patients provide evidence that the blood/brain barrier is disturbed in Ms. During the scans, a contrast agent used to enhance the images was able to cross the blood/brain barrier and highlight lesions in the brain. At this point, we don't know if the breakdown in the barrier is a primary effect or even a causative factor in MS or whether it is secondary to some other process. The same studies demonstrate that the disease can be progressing even if, outwardly, the patient's symptoms appear stable. These findings have yital implications for MS research. Scientists now have a tool, MRI, to monitor both the disease process and the effects of treatments.
Question. While we have 40 percent fewer stroke cases than we did 10 years ago, we still have 500,000 cases a year and more than 2 million people are living with the disabilities caused by stroke.
Please tell the Committee about the progress made in the field of stroke research.
Answer. Over the past year, NINDS-supported clinical trials have demonstrated the efficacy of two strategies to prevent stroke. The first trial showed that treatment with either aspirin or warfarin for patients with a specific heart condition (atrial fibrillation) greatly reduced the risk of stroke. Most recently, we have found that a surgical procedure on the large arteries in the neck, carotid endarterectomy, can prevent stroke in many patients who have severe blockage of the carotid artery and have already shown warning signs of stroke. We are continuing to study this procedure in patients with less severe blockage and patients who are asymptomatic.
Stroke research shows that, although the major brain damage occurs within the first few hours of stroke, injury to certain cell groups continues to progress for as long as 2 days. Several causes have been found, including the entry of toxic levels of calcium and other molecules into the nerve cell and an abnormal release of neurotransmitters. We are pursuing clinical studies of several promising therapeutic agents. A major research priority for the