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Fresh blood regulations for France
1408
region that
Noticeboard Fresh blood A
new
come
regulations for France
U, Welcher AA, Özcelik T, et al. Trembler mouse carries a point mutation in a myelin gene. Nature 1992; 356: 241-44. 2. Patel PI, Roa BB, Welcher AA, et al. The gene for the peripheral myelin protein 1. Suter
agency will govern French blood transfusion services-
l’Agence Franciase du Sang (AFS). Bernard Kouchner, the Minister of Health, knows that this reform has been long awaited. Since 1952,163 blood transfusion centres have run their own blood banks, with little central government control. The system nearly
collapsed when it emerged that many haemophiliacs had acquired transfusion-associated HIV infection (see Lancet 1991; 338: 809 and 1992; 339: 669). Last week, at the opening session of the 28th Congress of the French Federation for Blood Donors, held in Epinal (Vosges), Bernard Kouchner stated the lines along which the new agency will work. 51 % of the agency will be state-owned, 14% will be owned by the National Health Insurance Board and the rest will be divided between a range of government and private bodies (eg, INSERM and the Blood Donors Federation). Kouchner has appointed an "outsider" president of the new agency-Jacques Cincalbre, a transplant surgeon from Strasbourg. The AFS will decide the prices of blood products and volumes produced. It will also establish protocols for taking, stocking, and processing blood and blood products. The new director wants to create a body of inspectors who will enforce the new rules and has set up a committee for transfusion safety. This committee is independent of the AFS and will inform the Minister of Health directly of any deviation from the new procedures, or of any hazard from new viruses or blood-borne diseases. Finally, Kouchner wants to incorporate existing plasma fractionation centres into the new agency; the agency’s national fractionation laboratory will use plasma from unpaid donors. Boosting the supply of blood products from unpaid sources will reduce the import of products from paid donors.
Clumsy mice and Charcot-Marie-Tooth disease Charcot-Marie-Tooth disease (CMT), the commonest inherited peripheral neuropathy (affecting 1 in 2500 people), is genetically heterogeneous, arising from mutations at loci on several autosomes and the X chromosome. The most common form of the disease, type IA (CMT1A), characterised by distal muscle atrophy and weakness (often resulting in clawed hands and deformed feet, reduced nerve-conduction velocity, and hypertrophic neuropathy), is caused by a dominant mutation on the proximal short arm of chromosome 17. Three months ago the mouse model of CMT, a dominant mutation called trembler, on murine chromosome 11, was characterised as a defect in peripheral myelin protein-22 (PMP-22), which is normally expressed in Schwann cells and found in peripheral myelin.1 Trembler mice move clumsily and are subject to tremors and seizures, and their peripheral nerves show hypomyelination. Cloning and sequencing of PMP-22 complementary DNA from mutant mice revealed a point mutation
substituting an aspartic acid residue for a glycine. The PMP-22 locus lies in a region of mouse chromosome 11that seems to have an evolutionarily conserved counterpart in the region of human chromosome 17 containing the CMT 1 A gene (17pl2pll.2). This finding led Suter and colleagues to suggest that PMP-22 is a candidate gene for CMT and to predict that in man the PMP-22 locus would be found on the proximal short arm of chromosome 17.1 This week Sutar et al and three other groups 3-5 confirm that the PMP-22 gene does indeed map
is duplicated in CMT1A. Clumsy mice have not yet up with all the answers to CMT1A.
to
the CMT1A
region on 17p. Patel et al have cloned the human PMP-22 gene and note strong homology with the mouse gene.2 They found high levels of PMP-22 in femoral nerve and spinal cord but little in non-neural tissues. A puzzling feature of CMT1A is that linkage studies have shown duplication of some markers in the vicinity of the CMT1A locus, a finding that seems to favour a gene dosage effect, not a point mutation, as an explanation for the clinical manifestation of the human disease. Each of the groups reporting in this week’s Nature Genetics2-5 has produced direct evidence that PMP-22 maps to the
PMP-22 is a candidate for Charcot-Marie-Tooth disease type 1A. Nature Genetics
1992, 1: 159-65. 3. Valentijn LJ, Bolhuis PA, Zom I, et al. The peripheral myelin gene PMP-22/GAS-3 is duplicated m Charcot-Marie-Tooth disease type 1A. Nature Genetics 1992; 1: 166-70. 4. Timmerman V, Nelis E, Van Hul W, et al. The peripheral myelin protein gene PMP-22 is contained within the Charcot-Marie-Tooth disease type 1A duplication. Nature Genetics 1992; 1: 171-75. 5 Matsunami N, Smith B, Ballard L, et al. Peripheral myelin protein-22 gene maps in the duplication in chromosome 17p11.2 associated with Charcot-Marie-Tooth 1A. Nature Genetics 1992; 1: 176-79.
Take with
pinch of salt
A scrutiny of drug advertisements in ten peer-reviewed medical journals has come up with some unreassuring fmdings: 92% did not comply with at least one Food and Drug Administration rule relating to drug advertising; in 30% two or more reviewers disagreed with the implication that the product was the "drug of choice"; 28% would not have been recommended by the reviewers for publication; and 34% were judged by the reviewers to have needed major revisions before publication.! The authors of the report say that the FDA or the journals ought to develop a programme of surveillance of drug advertisements. In reply the
commissioner of the FDA2 describes the
extra
efforts that the
agency has put into regulating promotional activities. One of these is the project started last October to identify types of inappropriate clinical and statistical backing for claims made in advertisements. One example is the claim of comparability in safety and effectiveness with a competing product that is based on a clinical study designed to detect but does not show a statistical difference between the two products; the actual position may be that clinically important differences were not detected because of reasons such as inadequacy of sample size or variability in patient response. Another example is the claim of superiority in which the putative advantage is related to an end-point that was not the primary hypothesis tested by the study. The editors of the Annals of Internal Medicine argue that it is not feasible for each journal to independently review
advertisements in detail.3 They suggest a that a large multidisciplinary review body, of the type operating in Canada, might help; if one were to be set up in the USA, it should gather data on whether it gets better results than the existing system. They add that readers will always have to be critical of what they read. 1.
Wilkes, MS, Doblin BH, Shapiro MF. Pharmaceutical advertisements in leading medical journals: experts’ assessments Ann Intern Med 1992; 116: 912-19 2. Kessler DA. Addressing the problem of misleading advertising. Ann Intern Med 1992; 116: 950-51. 3. Fletcher RH, Fletcher SW. Pharmaceutical advertisements in medical journals. Ann Intern Med 1992; 116: 951-52.
Teachers’ prize
Monday saw teams from six medical schools presenting proposals for undergraduate oncology training programmes to an audience of deans of medical schools or their representatives. The occasion was not an ordinary medical education meeting. At stake was a ;E30 000 prize offered by the Cancer Research Campaign to the winning team to implement the proposals, to be paid only when the programme has been accepted by the school’s curriculum committee. All six finalists also received C2500 as a contribution to their programme. The CRC had seized the opportunity of the GMC’s review of undergraduate medical teaching to stimulate the development of oncology training programmes. Communication with patients is a skill the GMC would like developed. It would also like a shift from the force-feeding of students with facts towards problem-based learning and the encouragement of exploration of knowledge. All these elements hadlearly been taken into account by the competing teams which, to varying degrees, drew upon the help of modem teaching aids-computer and video programmes, simulated patients, plastic models. The judges, Prof David
region that
Noticeboard Fresh blood A
new
come
regulations for France
U, Welcher AA, Özcelik T, et al. Trembler mouse carries a point mutation in a myelin gene. Nature 1992; 356: 241-44. 2. Patel PI, Roa BB, Welcher AA, et al. The gene for the peripheral myelin protein 1. Suter
agency will govern French blood transfusion services-
l’Agence Franciase du Sang (AFS). Bernard Kouchner, the Minister of Health, knows that this reform has been long awaited. Since 1952,163 blood transfusion centres have run their own blood banks, with little central government control. The system nearly
collapsed when it emerged that many haemophiliacs had acquired transfusion-associated HIV infection (see Lancet 1991; 338: 809 and 1992; 339: 669). Last week, at the opening session of the 28th Congress of the French Federation for Blood Donors, held in Epinal (Vosges), Bernard Kouchner stated the lines along which the new agency will work. 51 % of the agency will be state-owned, 14% will be owned by the National Health Insurance Board and the rest will be divided between a range of government and private bodies (eg, INSERM and the Blood Donors Federation). Kouchner has appointed an "outsider" president of the new agency-Jacques Cincalbre, a transplant surgeon from Strasbourg. The AFS will decide the prices of blood products and volumes produced. It will also establish protocols for taking, stocking, and processing blood and blood products. The new director wants to create a body of inspectors who will enforce the new rules and has set up a committee for transfusion safety. This committee is independent of the AFS and will inform the Minister of Health directly of any deviation from the new procedures, or of any hazard from new viruses or blood-borne diseases. Finally, Kouchner wants to incorporate existing plasma fractionation centres into the new agency; the agency’s national fractionation laboratory will use plasma from unpaid donors. Boosting the supply of blood products from unpaid sources will reduce the import of products from paid donors.
Clumsy mice and Charcot-Marie-Tooth disease Charcot-Marie-Tooth disease (CMT), the commonest inherited peripheral neuropathy (affecting 1 in 2500 people), is genetically heterogeneous, arising from mutations at loci on several autosomes and the X chromosome. The most common form of the disease, type IA (CMT1A), characterised by distal muscle atrophy and weakness (often resulting in clawed hands and deformed feet, reduced nerve-conduction velocity, and hypertrophic neuropathy), is caused by a dominant mutation on the proximal short arm of chromosome 17. Three months ago the mouse model of CMT, a dominant mutation called trembler, on murine chromosome 11, was characterised as a defect in peripheral myelin protein-22 (PMP-22), which is normally expressed in Schwann cells and found in peripheral myelin.1 Trembler mice move clumsily and are subject to tremors and seizures, and their peripheral nerves show hypomyelination. Cloning and sequencing of PMP-22 complementary DNA from mutant mice revealed a point mutation
substituting an aspartic acid residue for a glycine. The PMP-22 locus lies in a region of mouse chromosome 11that seems to have an evolutionarily conserved counterpart in the region of human chromosome 17 containing the CMT 1 A gene (17pl2pll.2). This finding led Suter and colleagues to suggest that PMP-22 is a candidate gene for CMT and to predict that in man the PMP-22 locus would be found on the proximal short arm of chromosome 17.1 This week Sutar et al and three other groups 3-5 confirm that the PMP-22 gene does indeed map
is duplicated in CMT1A. Clumsy mice have not yet up with all the answers to CMT1A.
to
the CMT1A
region on 17p. Patel et al have cloned the human PMP-22 gene and note strong homology with the mouse gene.2 They found high levels of PMP-22 in femoral nerve and spinal cord but little in non-neural tissues. A puzzling feature of CMT1A is that linkage studies have shown duplication of some markers in the vicinity of the CMT1A locus, a finding that seems to favour a gene dosage effect, not a point mutation, as an explanation for the clinical manifestation of the human disease. Each of the groups reporting in this week’s Nature Genetics2-5 has produced direct evidence that PMP-22 maps to the
PMP-22 is a candidate for Charcot-Marie-Tooth disease type 1A. Nature Genetics
1992, 1: 159-65. 3. Valentijn LJ, Bolhuis PA, Zom I, et al. The peripheral myelin gene PMP-22/GAS-3 is duplicated m Charcot-Marie-Tooth disease type 1A. Nature Genetics 1992; 1: 166-70. 4. Timmerman V, Nelis E, Van Hul W, et al. The peripheral myelin protein gene PMP-22 is contained within the Charcot-Marie-Tooth disease type 1A duplication. Nature Genetics 1992; 1: 171-75. 5 Matsunami N, Smith B, Ballard L, et al. Peripheral myelin protein-22 gene maps in the duplication in chromosome 17p11.2 associated with Charcot-Marie-Tooth 1A. Nature Genetics 1992; 1: 176-79.
Take with
pinch of salt
A scrutiny of drug advertisements in ten peer-reviewed medical journals has come up with some unreassuring fmdings: 92% did not comply with at least one Food and Drug Administration rule relating to drug advertising; in 30% two or more reviewers disagreed with the implication that the product was the "drug of choice"; 28% would not have been recommended by the reviewers for publication; and 34% were judged by the reviewers to have needed major revisions before publication.! The authors of the report say that the FDA or the journals ought to develop a programme of surveillance of drug advertisements. In reply the
commissioner of the FDA2 describes the
extra
efforts that the
agency has put into regulating promotional activities. One of these is the project started last October to identify types of inappropriate clinical and statistical backing for claims made in advertisements. One example is the claim of comparability in safety and effectiveness with a competing product that is based on a clinical study designed to detect but does not show a statistical difference between the two products; the actual position may be that clinically important differences were not detected because of reasons such as inadequacy of sample size or variability in patient response. Another example is the claim of superiority in which the putative advantage is related to an end-point that was not the primary hypothesis tested by the study. The editors of the Annals of Internal Medicine argue that it is not feasible for each journal to independently review
advertisements in detail.3 They suggest a that a large multidisciplinary review body, of the type operating in Canada, might help; if one were to be set up in the USA, it should gather data on whether it gets better results than the existing system. They add that readers will always have to be critical of what they read. 1.
Wilkes, MS, Doblin BH, Shapiro MF. Pharmaceutical advertisements in leading medical journals: experts’ assessments Ann Intern Med 1992; 116: 912-19 2. Kessler DA. Addressing the problem of misleading advertising. Ann Intern Med 1992; 116: 950-51. 3. Fletcher RH, Fletcher SW. Pharmaceutical advertisements in medical journals. Ann Intern Med 1992; 116: 951-52.
Teachers’ prize
Monday saw teams from six medical schools presenting proposals for undergraduate oncology training programmes to an audience of deans of medical schools or their representatives. The occasion was not an ordinary medical education meeting. At stake was a ;E30 000 prize offered by the Cancer Research Campaign to the winning team to implement the proposals, to be paid only when the programme has been accepted by the school’s curriculum committee. All six finalists also received C2500 as a contribution to their programme. The CRC had seized the opportunity of the GMC’s review of undergraduate medical teaching to stimulate the development of oncology training programmes. Communication with patients is a skill the GMC would like developed. It would also like a shift from the force-feeding of students with facts towards problem-based learning and the encouragement of exploration of knowledge. All these elements hadlearly been taken into account by the competing teams which, to varying degrees, drew upon the help of modem teaching aids-computer and video programmes, simulated patients, plastic models. The judges, Prof David