We have six other trials in the planning process that could be supported in FY 1991. These include a treatment for head injury and a treatment for spinal cord injury. We would also support the National Acute Spinal Cord Injury Study III. Two other trials would determine the efficacy of a calcium channel blocker and an anti-convulsant to treat patients with partial seizures. We also would conduct a trial on the use of monoclonal antibodies to treat multiple sclerosis.

AUTISM RESEARCH

Question. In 1989, the movie "Rain Man" provided many in the public with their first exposure to the severe developmental disability know as autism. As that movie recedes from public attention, I am concerned that NIH not recede from appropriate efforts to combat this condition. Please describe for the record the Institute's FY 1991 plans for encouraging, funding, conducting, and collating research on the causes and treatment of autism. Please also outline the Institute's preliminary thoughts on autism research efforts for future years so that the Committee can appreciate how proposed FY 1991 expenditures for this purpose relate to longer term efforts to understand and combat this condition.

Answer. The Institute will continue in FY 1991 to promote its recent program announcement to encourage both basic neuroscience and clinical research concerning autism. It is quite clear that autism is a neurological defect in brain functioning. It is not a "behavioral disorder". A pediatric neurologist with a special interest in autism has been recruited to the NINDS staff to develop further the Institute's research program on autism. New research opportunities in autism and the other neurological behavior disorders were identified in the Decade of the Brain report to the House Committee on Appropriations. The Institute plans to pursue vigorously these opportunities such as the preliminary finding of underdeveloped areas of the cerebellum in some patients and the association of subtypes of autistic patients with the fragile X chromosome.

QUESTIONS SUBMITTED BY SENATOR ARLEN SPECTER

GAUCHER'S DISEASE

Question. Dr. Goldstein, I have been informed by the Gaucher's Disease Foundation that scientists at the Institute have made significant progress in the treatment for Type I Gaucher's Disease in recent months. Would you describe this progress for the Committee?

Answer. Children with Goucher's disease lack an important enzyme that is responsible for metabolizing fatty material in the body. Without the enzyme, fatty substances build up in tissues and organs and lead to an enlarged spleen, liver, severe anemia, mental retardation, bone pain and fractures. In some cases, more severe neurological complications may occur. Type I Gaucher's disease afflicts approximately 20,000 people in the United States.

A team of NINDS intramural scientists, led by Dr. Roscoe 0. Brady, have had great success with an enzyme replacement therapy for these children. The enzyme, isolated from normal human placental tissue, was administered to 13 Gaucher patients. These patients have had remarkable clinical benefit. Their anemia has been corrected, the size of the enlarged spleens and livers has decreased, and magnetic resonance imaging indicates that there is skeletal improvement. In addition, many have been able to return to normal daily activities, and they all emphatically state that the quality of their lives is greatly improved. Seventeen (17) Gaucher's patients have acted as controls in this clinical trial. They have not yet received the enzyme. Attempts are now being made to determine the minimal required doses, and to find a means of prolonging and activating the actions of the enzyme.

In related work, the same group of investigators is attempting to develop other approaches to enzyme replacement therapy. Investigators have inserted the human gene for the enzyme into mouse bone marrow cells in the laboratory, which have then produced the human glucocere-brosidase enzyme. Additional work must be done before determining the utility of this technique in humans but, if successful, clinicians would have a method to treat Gaucher's disease that would be permanent.

Question. Does the NINDS have sufficient resources to continue to build upon these research findings in FY 1990? What are the implications of not proceeding with further clinical trials at this time?

Answer. We do not have adequate funds in FY 1990 or FY 1991 to build upon these research findings without impacting other clinical research projects. Although the studies with enzyme replacement therapy so far are very positive and encouraging, many questions about the treatment remain unanswered. For instance, we need to know the optimal doses of enzyme and the long-term efficacy of the treatment. The intramural studies in Gaucher's disease are continuing, at a reduced level commensurate with the available resources, but certainly an increased level of effort is justified by the amazing results obtained already.

Question. Has the drug been approved for use by the FDA?

Answer. The FDA has released a treatment IND for the enzyme, so that people can now buy the enzyme with individual FDA approval. There is a plan to submit a new drug application (NDA) request by mid-March. If the review is expedited--which is expected--approval could be received by mid-summer. Even with FDA approval, patients must deal with the issue of cost of the drug, which is expensive.

CLINICAL TRIALS

Question. Dr. Goldstein, how many clinical trials have been approved in FY 1990? How many of these have received funding?

Answer. Eight new clinical trials have been approved for funding in FY 1990, although additional applications may be received prior to the end of the fiscal year. Of the eight trials approved, one will be funded.

Question. How many will you be able to fund under the FY 1991 budget request? Which ones will not receive funding?

Answer. No new clinical trials will be funded under the FY 1991 budget request; only ongoing clinical trials will be funded. There are six proposed new trials in the planning process that could be funded. These include a clinical trial for the treatment of acute head injury and a trial for the treatment for acute spinal cord injury. We would also support the National Acute Spinal Cord Injury Study III. Two other trials would study the efficacy of a calcium channel blocker and an anticonvulsant to treat patients with partial seizures. We also would conduct a trial on the use of monoclonal antibodies to treat multiple sclerosis.

NATIONAL INSTITUTE ON DEAFNESS AND OTHER COMMUNICATION DISORDERS

STATEMENT OF DR. JAMES B. SNOW, JR., DIRECTOR

ACCOMPANIED BY DR. JAY MOSKOWITZ, ASSOCIATE DIRECTOR FOR SCIENCE POLICY AND LEGISLATION, NATIONAL INSTITUTES OF HEALTH

SUMMARY STATEMENT

Senator HARKIN. Dr. Snow, Institute of Deafness and Other Communication Disorders, we are delighted to have you with us as the first permanent Director of the National Institute on Deafness and Other Communication Disorders.

You set another precedent in being the first senior scientist recruited to serve at NIH from the private sector in many years, and we are delighted that you have agreed to lead this very important endeavor.

As you know, I have a strong interest in your Institute and want to follow what you are doing very carefully and want to be as supportive as possible.

I also want to give my public thanks again to Dr. Moskowitz next to you for helping to establish the Deafness Institute and to get it going for the last year, and I know you are delighted to be back in your other position again and have Dr. Snow on board, but you have done a great job in getting it going and I really appreciate it very much.

Dr. Snow, your request for next year is $122.85 million or a 4.48percent increase. Again, welcome and please proceed.

Dr. SNOW. Thank you very much, Mr. Chairman. In the 11⁄2 years since the Institute was created by the Congress great strides have been made. We are particularly indebted to Dr. Jay Moskowitz for his very able stewardship that he provided the new institute during its initial development. Our goal is to maximize the gains that can be realized from research on human communication.

The NIDCD is pleased to join with other institutes at the NIH in celebrating the "Decade of the Brain." Neurobiology is an important component of our research portfolio, both extramurally and intramurally. An example of the focus of the NIDCD on neurobiology is neural regeneration. Regeneration of nervous tissue after injury and illness occurs in very few places in the human body. Interestingly, nasal neuroepithelium is one tissue that does have the ability to regenerate. Research in this area has profound implications for the repair and regeneration of the nervous system.

Scientists supported by the NIDCD have utilized the tools of molecular biology and molecular genetics to uncover important information on how human communication systems work at a funda