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Lorenzo’s Oil expected to remain well during the current follow-up period
even without therapy.
Directed by George Miller. Starring Nick Nolte, Susan Sarandon.
Universal studios. USA. 1992. Certificate 12. 135 mins.
The film cites fundamental differences between the
Lorenzo’s Oil is a film about a couple whose
priorities of the parents and of the medical establishment,
represented in this instance by Dr Nicolai (played by Peter
extraordinarily intelligent and promising 6-year-old son Ustinov who, except that he wears a bow tie, has copied my
became progressively disabled by adrenoleukodystrophy
appearance and speech with remarkable accuracy). The
(ALD), an X-linked disorder that affects 1 in 20 000 males. movie shows that we had proposed that Lorenzo took part in
After their initial shock, disbelief, and despair, the parents a controlled study of dietary very-long-chain fatty acid
were determined to learn complex medical and biochemical
restriction alone. That study, which had a sound rationale,
facts that enabled them to contribute substantially to the had been completed in 1982, two years before I met
understanding and possibly the therapy of this disease. The Lorenzo. Hesitancy to use erucic acid in ALD patients was
movie is deeply touching and demonstrates the power of based on the concern that the acid itself is of the
total commitment and the refusal to give up hope. As a work
of fiction, Lorenzo’s Oil is an excellent film. However, as a
very-long-chain type and that ALD patients could not
metabolise it; the capacity of normal Eskimos to tolerate
factual documentary it has three main flaws: it overstates the erucic acid might not assure its safety. Once this concern was
success that can be achieved with the oil, it invents conflicts
between the parents and the medical establishment, and it
relieved, approval by the institutional review board and the
Food and Drug Administration was obtained within 30
presents an inaccurate and malicious portrayal of a valued days. Because of the severity and relentless progression of
parents’ organisation. ALD, an open rather than a randomised study was
Lorenzo’s oil (a mixture of erucic acid and oleic acid) undertaken. All ALD patients who request to take part in
returns the raised plasma concentrations of saturated
the study can receive Lorenzo’s oil, although in the USA the
very-long-chain fatty acids, the principal biochemical cost of the oil presents a serious difficulty.
abnormality in ALD, to normal. The National Institutes of I find it hard to understand the reasons for the movie’s
Health and the Food and Drug Administration are funding inaccurate and mean-spirited representation of the parents’
a five-year study of the clinical efficacy of the oil, which is
now in its third year and has involved 257 patients with
organisation (the United Leukodystrophy Foundation).
The scene in which parents demand access to Lorenzo’s oil
ALD. I direct this study, and since its existence is widely over the objections of the organisation is not only
known, I am dismayed that the film’s producers did not preposterous, it simply did not occur. The topics discussed
acquaint themselves with the study’s findings. Our results, at the meetings are decided by the membership of the
and also those from Italy, indicate that the oil does not slow
the rapid progression of the childhood neurological form of
parents’ organisation. For scientific issues, the organisation
obtains the opinion of a 13-member international scientific
the illness. The motion picture attributes Lorenzo’s nine-
advisory committee that represents many points of view.
year survival after onset of symptoms to the oil, but 12 Contrary to the film’s presentation, the organisation has had
untreated boys who became ill at the same age have survived an important role in making the oil available to patients. The
to their late teens or early twenties. It is encouraging that
Lorenzo is reported to have acquired new skills
organisation has also supported research, including very
recent work that has led to the isolation of a putative gene.*
demonstrable through subtle testing procedures, but this This work acknowledges the support of the United
might be the result of his unique circumstances rather than Leukodystrophy Foundation.
because of the oil itself. The treatment of ALD is under intense investigation.
Lorenzo’s oil at best provides very limited benefit for the Bone marrow transplantation seems to be helpful under
milder adult form of ALD. Our studies, which involve more
than 100 patients, suggest that functional impairment
carefully selected circumstances and may prove to be the
most effective approach. The impending isolation of the
continues to progress slowly. A crucial but still unresolved ALD gene raises the long-term hope that gene therapy will
question is whether the oil can prevent or postpone the onset become possible. The rapid neurological progression
of neurological disability in boys who are neurologically associated with ALD seems to be due to a cytokine-
intact. We are following 61 such patients. 53 have remained mediated inflammatory response, and therapeutic trials to
well, but the follow-up time is still short. 1 patient who was counteract this are in progress. It is essential that the current
started on the oil at 3 years of age developed the severe form evaluations of the efficacy of Lorenzo’s oil be brought to
of the childhood illness at 5 years despite excellent control of
his plasma very-long-chain fatty acid concentration, and in 7
completion. At this time the jury is out.
boys the lesions shown by brain magnetic resonance Department of Neurogenetics,
Kennedy Krieger Institute,
imaging studies have increased in size. The oil is therefore 707 North Broadway,
not an absolute preventive treatment. Longer follow-up is Baltimore, Maryland 21205, USA HUGO W. MOSER
required to determine whether it has a significant effect.
Moreover, 50% of untreated patients escape the childhood *A mutation in a gene that codes for a peroxisomal transport protein has
form of the illness, so that half of the 61 patients would be recently been reported in 6 of 85 patients with ALD (see p 552).-ED. L.

The Oxford hyperostotic lesions in neolithic hunter-gatherers and the ills Textbook of Clinical Pharmacology. Demography. with its increased title. in any case. 25. and therapeutic processes is clearly and infectious disease. We may be guilty of shooting chronic disease by measuring serum ferritin concentrations ourselves in the foot over this issue if the end result is that (which are normal or raised in chronic disease). witholding iron deprives pathogens of the concisely presented. it was the "green confronted with the choice between a post-mortem sickness" of Shakespeare’s heroines. However. 1992. area of concern in professional circles and the public In a competitive market. incidence of disease. Textbooks play a vital part in this geographical space to examine peoples living in different strategy. are the basic principles of clinical or collected by studying the Afro-Americans or Kung pharmacology. Fortunately. striding retained for future use. Others. G. and Diseases somewhat boring evil and. In England. Pp 296.. little available in standard formularies that are more regularly doubt that medical students will continue to need a firm foundation of knowledge of both mechanisms of drug action updated and are free of charge. The With the publication in the UK of new General Medical definition of a commonly used drug is loose and includes Council guidelines on undergraduate medical education. 1992. the economics of drug use remains an important satisfies their need. The success of most textbooks rests with students and not and of when and how drugs should be used in clinical care. The main body of the text the body’s natural defences. DM128. But which clinical pharmacology is presented to the medical can one have too much of a good thing? The authors of this undergraduate will determine how much time they will be polemic believe that the answer is yes. not least the family . pharmacokinetic. Demography. BARNETT clinical pharmacology has been thought of by some clinicians in the same terms as a basic science-a necessary if Diet. Berlin: Aldine de Gruyter.. I remain sceptical about the iron required to prosper. domain. This Diet. goes a long way to achieving this difficult balancing act. which can be simply divided into: what do populations of today. Kent. Grahame-Smith. B. found on a papyrus dated 1500 BC. In this section the authors Oxford: Oxford University Press. Presentation is all important and the way in added to foodstuffs to combat iron deficiency anaemia. despite its unimaginative of our own sedentary. in terms of both content and readability. S. Similar information is an interesting position in this debate. the authors conclude that the drugs do to the body and what does the body do to drugs? hypoferric anaemia associated with settlement is more likely This part is well written and the introduction of the student to be due to increased pathogen load than to inadequate to the notions of pharmaceutical. Exactly how clinical pharmacology should best be taught Department of Pharmacology and Therapeutics. and Disease is a notable attempt to multi-author section is again well written with clear provide information through multidisciplinary networking. Stuart- attitude is now far less prevalent. though aware of the theories. I hope. Getting the balance right between environmental conditions and on vastly different diets. J. prepared to spend on the subject and how much of it will be They train their sights on a single target. known as Even pathology seems more exciting when the student is chlorosis until the nineteenth century. investigating porotic the important basic principles is not easy. The viewpoints are many but. subheadings and several helpful figures and tables to distract The modern haematologist. Leicester Royal Infirmary. The medical students see pharmacokinetics as being of little administration of iron in such cases may serve only to thwart direct relevance to their needs. THE LANCET 545 BOOKSHELF Oxford Textbook of Clinical Pharmacology and My only serious criticism of what is otherwise a valuable Drug Therapy textbook in this area is the inclusion of the section entitled Second Edition-D. overburdensome factual information and the presentation of They pace evolutionary time. committees and heads of medical school academic I question the need for inclusion of material in this format departments as to the definition of "core" versus "optional" which adds to the bulk and cost of the book without an subjects. Students must feel that a particular text In addition. Clinical pharmacology and therapeutics occupies equivalent increase in usefulness. The text is divided into four sections. it is of paramount importance to need to include 40 mathematical formulae in the differentiate true iron-deficiency anaemia from that of pharmacokinetics section. and probably irrespective of whether their data are palaeoanthropological most important. "Pharmacopoeia" of almost 200 pages (about one quarter of the total text excluding the index). There is. the Oxford textbook is well placed. K. with their teachers. Aronson. First. UK D. ISBN 0-192616757. In the past the teaching of Leicester LE2 7 LX. some that are rarely encountered by most clinicians and there will be considerable debate between curriculum others that are probably never now used by any. Iron seems to have demonstration and a lecture on the pharmacokinetics of entered therapeutic practice 350 years ago and today is freely phenytoin. students will learn therapeutics on the job after they qualify. Because. this Changing Perspectives in Anaemia. That aside. in the anaemia of chronic pharmacodynamic. the student. Edited by P. Yet it is still difficult to Macadam. encourage students to take a particular interest in clinical pharmacology when they are struggling to understand the The first description of what was probably anaemia is complexities of physical examination and clinical medicine. dietary iron intake. to medical students is less certain. should find much of interest. list ". those drugs which are most commonly used in clinical practice (a total of about 300 compounds)". ISBN 3-110138344. Pp 756. deals with disease-oriented clinical pharmacology. settled style of life.