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US tightens oversight of gene-therapy trials
POLICY AND PEOPLE
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n March 9, Spain’s Federation of Relatives of Alzheimer’s Disease Patients (FRADP) and the national police force signed an agreement which may decrease the distress caused when a patient with Alzheimer’s disease becomes lost. Next September, a pilot project, which will aim to accurately locate patients with Alzheimer’s disease who wander from home and get lost in the streets, will be started in Barcelona. Small electronic devices will be attached to patients’ clothes, such that they cannot be removed by the patient. They will incorporate microprocessors and small batteries. The device will send Global Positioning System satelliteguided signals to a police control centre; patrol cars at city zones near the signal will then be alerted to pick up lost patients. These electronic localisers, which will emit signals similar to those from mobile phones, will be activated only if the patient strays from a previously defined radius. Also, if a patient reported lost by relatives has not been found after a given time, the police control centre may itself activate the device. The margin of error is estimated to be 10 m. Early next month, all Barcelona police will receive training courses in how to act in the presence of a patient with Alzheimer’s disease at the time of a first contact, and how to return them home without causing fear. Police will also carry notes to enable them to remember the symptomatology and behaviour of individual patients. Paloma Ramos, president of FRADP, said that the agreement has developed from concerns that relatives are often unable to control family members with Alzheimer’s disease, especially in the first stages of disease “when the patient has mobility and no physical impairment, but becomes frequently disoriented and loses his or her memory, thereby meaning that the patient may be unable to remember his or her name or address”. Although only a small number of patients will participate initially, Ramos hopes that if the Barcelona project is successful, it will be extended nationwide.
Xavier Bosch
THE LANCET • Vol 355 • March 18, 2000
US FDA rejects defence of gene-therapy trial it approved the trial in which n a sternly worded letter, the US Gelsinger was enrolled, and that FDA rejected arguments made by other data, from experiments on the University of Pennsylvania in three primates, had not been defence of its gene-therapy proreported because in gramme in which Jesse two cases it involved a Gelsinger, an 18-yeardifferent gene-therapy old Arizona man, died and in the remaining last autumn. After an case because it investigation, the FDA involved a mild illness, shut down the trial and not death, at a dose 17 seven others being times higher than the done at the university’s highest dose in the prestigious Institute trial. for Human Gene But in a March 3 Therapy, alleging letter sent to James numerous protocol Wilson, director of violations including the university’s geneenrolment of ineligible Wilson—defending U Penn therapy institute, FDA patients, failure to officials contend, among other comobtain fully informed consent, and plaints, that the institute had indeed neglecting to report important safety violated protocols, failed to provide data. animal data that suggested “a signifiThe university responded with a cant risk for human subjects”, and 34-page letter, which, while admitfailed to establish standard operating ting some shortcomings in its proceprocedures necessary to conduct a dures and data reporting, argued that clinical study. The FDA officials gave all patients participating in the trial Wilson 15 days to tell the agency had properly enrolled and had prowhat steps the institute planned to vided informed consent. With regard take to remedy these problems. In the to the allegations about failure to meantime, the institute’s trials will report important safety data, the uniremain on hold, the FDA said. versity maintained that some of the data in question had been in the hands of the FDA for months before Michael McCarthy
I
Robert H Clink
Spain plans electronic tagging for patients with Alzheimer’s disease
US tightens oversight of gene-therapy trials he US FDA and National Institutes of Health have launched two initiatives aimed at improving protection for patients participating in gene-therapy trials and at restoring public confidence in gene-therapy research. Since the death of 18-year-old Jesse Gelsinger, who died on Sept 17, 1999, while participating in a phase I trial of gene therapy for ornithine transcarbamylase deficiency, investigations into US gene-therapy programmes have found numerous protocol violations. More than a dozen trials have now been halted either voluntarily by the researchers or as a result of orders from the FDA. The first initiative, the Gene Therapy Clinical Trial Monitoring Plan, will toughen current regulations governing the monitoring of clinical gene-therapy trials. Clinical-trial monitoring programmes are supposed to provide assurance that patients’ rights and wellbeing are being protected, that the trial is being done according to protocol and good
T
clinical practice, and that data reporting to government-oversight bodies is accurate and complete. Recent investigations, the FDA said, found that the clinical monitoring of several gene-therapy trials had been “less than adequate”. Under the new rules, researchers will have to submit their monitoring plans routinely for FDA review. In addition, the agency will carry out “surveillance” to make sure the plans are being followed and are adequate. The second initiative, called the Gene Transfer Safety Symposia, will consist of a series of public meetings that will bring together researchers from different studies to discuss safety and other issues relevant to the trials. These symposia will be held about four times a year, the agencies said. In addition, the agencies will provide support to other institutions and professional organisations to hold safety conferences focusing on gene therapy. Michael McCarthy
997
O
n March 9, Spain’s Federation of Relatives of Alzheimer’s Disease Patients (FRADP) and the national police force signed an agreement which may decrease the distress caused when a patient with Alzheimer’s disease becomes lost. Next September, a pilot project, which will aim to accurately locate patients with Alzheimer’s disease who wander from home and get lost in the streets, will be started in Barcelona. Small electronic devices will be attached to patients’ clothes, such that they cannot be removed by the patient. They will incorporate microprocessors and small batteries. The device will send Global Positioning System satelliteguided signals to a police control centre; patrol cars at city zones near the signal will then be alerted to pick up lost patients. These electronic localisers, which will emit signals similar to those from mobile phones, will be activated only if the patient strays from a previously defined radius. Also, if a patient reported lost by relatives has not been found after a given time, the police control centre may itself activate the device. The margin of error is estimated to be 10 m. Early next month, all Barcelona police will receive training courses in how to act in the presence of a patient with Alzheimer’s disease at the time of a first contact, and how to return them home without causing fear. Police will also carry notes to enable them to remember the symptomatology and behaviour of individual patients. Paloma Ramos, president of FRADP, said that the agreement has developed from concerns that relatives are often unable to control family members with Alzheimer’s disease, especially in the first stages of disease “when the patient has mobility and no physical impairment, but becomes frequently disoriented and loses his or her memory, thereby meaning that the patient may be unable to remember his or her name or address”. Although only a small number of patients will participate initially, Ramos hopes that if the Barcelona project is successful, it will be extended nationwide.
Xavier Bosch
THE LANCET • Vol 355 • March 18, 2000
US FDA rejects defence of gene-therapy trial it approved the trial in which n a sternly worded letter, the US Gelsinger was enrolled, and that FDA rejected arguments made by other data, from experiments on the University of Pennsylvania in three primates, had not been defence of its gene-therapy proreported because in gramme in which Jesse two cases it involved a Gelsinger, an 18-yeardifferent gene-therapy old Arizona man, died and in the remaining last autumn. After an case because it investigation, the FDA involved a mild illness, shut down the trial and not death, at a dose 17 seven others being times higher than the done at the university’s highest dose in the prestigious Institute trial. for Human Gene But in a March 3 Therapy, alleging letter sent to James numerous protocol Wilson, director of violations including the university’s geneenrolment of ineligible Wilson—defending U Penn therapy institute, FDA patients, failure to officials contend, among other comobtain fully informed consent, and plaints, that the institute had indeed neglecting to report important safety violated protocols, failed to provide data. animal data that suggested “a signifiThe university responded with a cant risk for human subjects”, and 34-page letter, which, while admitfailed to establish standard operating ting some shortcomings in its proceprocedures necessary to conduct a dures and data reporting, argued that clinical study. The FDA officials gave all patients participating in the trial Wilson 15 days to tell the agency had properly enrolled and had prowhat steps the institute planned to vided informed consent. With regard take to remedy these problems. In the to the allegations about failure to meantime, the institute’s trials will report important safety data, the uniremain on hold, the FDA said. versity maintained that some of the data in question had been in the hands of the FDA for months before Michael McCarthy
I
Robert H Clink
Spain plans electronic tagging for patients with Alzheimer’s disease
US tightens oversight of gene-therapy trials he US FDA and National Institutes of Health have launched two initiatives aimed at improving protection for patients participating in gene-therapy trials and at restoring public confidence in gene-therapy research. Since the death of 18-year-old Jesse Gelsinger, who died on Sept 17, 1999, while participating in a phase I trial of gene therapy for ornithine transcarbamylase deficiency, investigations into US gene-therapy programmes have found numerous protocol violations. More than a dozen trials have now been halted either voluntarily by the researchers or as a result of orders from the FDA. The first initiative, the Gene Therapy Clinical Trial Monitoring Plan, will toughen current regulations governing the monitoring of clinical gene-therapy trials. Clinical-trial monitoring programmes are supposed to provide assurance that patients’ rights and wellbeing are being protected, that the trial is being done according to protocol and good
T
clinical practice, and that data reporting to government-oversight bodies is accurate and complete. Recent investigations, the FDA said, found that the clinical monitoring of several gene-therapy trials had been “less than adequate”. Under the new rules, researchers will have to submit their monitoring plans routinely for FDA review. In addition, the agency will carry out “surveillance” to make sure the plans are being followed and are adequate. The second initiative, called the Gene Transfer Safety Symposia, will consist of a series of public meetings that will bring together researchers from different studies to discuss safety and other issues relevant to the trials. These symposia will be held about four times a year, the agencies said. In addition, the agencies will provide support to other institutions and professional organisations to hold safety conferences focusing on gene therapy. Michael McCarthy
997