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Omega, Public Health National Honor Society, University of California; Doctor
ACUTE SPINAL CORD INJURY
Senator HARKIN. Thank you very much, Dr. Goldstein.
I understand there are nerve cells that are damaged by trauma and repair themselves if you get to them with 8 hours, you say.
Dr. GOLDSTEIN. Yes, sir; in the spinal cord.
What do you do? What kind of a treatment is it? Let's say that someone has broken their neck or broken their back or something like that. And the spinal cord, obviously, has not been severed. It has been bruised.
Dr. GOLDSTEIN. That's right, sir.
Senator HARKIN. What do you do? What is the treatment? What do they do?
Dr. GOLDSTEIN. There is a drug that is commonly available on shelves in emergency rooms. It is not a new drug. It has been there for years. That drug in one large amount, an overwhelming amount—and that was the amazing thing about it is given to the patient intravenously immediately and then for 24 hours. That drug prevents the damaged nerve cell from dying. It gives it the opportunity to be protected from the deadly process killing it and then begin to repair itself.
The treatment, by the way, costs about $100, and it prevents a lifetime of severe disability, often being confined to a wheelchair. It is as simple as that.
The drug is so innocuous that if the physician thinks the patient may have a spinal cord injury and is not sure, it is well worth giving the drug. It costs about $100 and it does not have a deleterious side effect. It can have a tremendous positive effect.
Let me volunteer the next question, and that is will it work in the brain, a head injury. We don't believe it will.
CHRONIC DEGENERATIVE BRAIN DISEASES Senator HARKIN. How about Parkinson's disease? Dr. GOLDSTEIN. No, sir.
Senator HARKIN. I mean early intervention. Not that drug, but could there be early intervention for things like Parkinson's or Alzheimer's, things like this?
Dr. GOLDSTEIN. I think I understand your question.
The scientific issue is shared with my colleague in the Aging Institute--and we work closely together on it. The focus of NINDS attention on Alzheimer's disease and Parkinson's diseases and other degenerative diseases of the brain, which we have singled out for priority attention, is not the patient who has advanced Parkinson's 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 committee 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.
Dr. RAUB. OK
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.
What is it about early treatment that is so
Angwer. Within the first hours following severe head or spinal cord trauma, a cascade of biochemical events can occur to make the injury worse (secondary injury). As demonstrated by an NINDSsupported 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?
Angwer. 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.
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