14 
be as neutral as possible; vhen the data are submitted, the working group 
could decide if adequate information had been supplied. 
The working group agreed to add the phrase "and/or other animals" to Section 
I-B-2-c-(l)-(e) . Dr. Walters suggested the sentence be modified bo read: 
"Has a protocol similar to the one proposed for a clinical trial been carried 
out in non-human primates and/or other animals? What were the. results?" 
Fir. Temin asked whether the word "malignancy" in Section I-B-2-c-(l)-(c) 
was the correct term. Dr. Mahoney pointed cut that the working group might 
also wish to knew if benign tumors develop. Dr. Motulsky suggested the working 
group substitute the words "development of neoplasia" for the word "malignancy" 
since neoplasia refers to both malignancies and benign tumors. The working 
group agreed to this proposed change. 
Dr. Walters called the attention of the working group to Section I-B-2-c-(2) 
of the document. He said language asking how long the animals have been studied 
after treatment had been added to this section. The working group agreed it 
was reasonable to add this language to the document. 
Dr. Grotstein thought the phrase "particularly germ line cells" should be added 
to the first sentence of .Section I-B-2-c-(2). That sentence would then read 
as follows: 
"If a non- retroviral delivery system is used: Vhat animal studies have been 
done to determine if there cure pathological or other undesirable consequences 
of the protocol ( including insertion of DNA into cells other than those 
treated, particularly germ line cells)? 
The working group agreed to this proposed modification. 
Dr. Walters then called the attention of the working group bo Secbion I-B-3, 
Clinical procedures, including pabient moni boring , of the document. He noted 
that language requesting information on other similar studies had been included 
in this section of the document. The working group agreed bo this preposed 
modification. 
Dr. Walters said three items had been transferred from Section I-B-3 of the 
previous version of the document (Attachment II) to create a new section entitled, 
Reporting Requirements . The working group agreed to this modification. 
Dr. Walters said language dealing with autopsy had been included in Section 
I-B-3-f and IV-B of the document. Dr. Tamin thought Section I-B-3-f should 
specifically require an autopsy. The initial hurren gene therapy protocols win 
be scientific experiments, and an autopsy should be performed in order to gain 
all possible information. Should the patient die, one would wish to knew why 
the patient died. One would also wish bo know how the therapy affected the 
patient. Dr. Tamin said investigators cannot lose track of human gene therapy 
subjects. 
[ 99 ] 
