National Association of Parents of Autistic People
Modern medicine advances through working hypotheses which are normally built on a sound rational basis and must subsequently be validated. Hypotheses are never formulated and then validated by researchers of quality, as it is essential to work in teams and benchmark one’s work vis-a-vis that of other teams, in a world-wide context. Anyone engaged in research must now know English, which allows researchers to benchmark their work vis-a-vis the rest of the world, which mainly publishes in the English language.
Science journals perform an essential function, in that they disseminate all results, even when they are negative, among those who pursue a specific field of research. On every prestigious journal is an editorial team who select articles, as only those complying with agreed guidelines of methodological appropriateness will be accepted.
All serious researchers must possess an exceptional degree of integrity and mettle, which will allow them to face a peer review before presenting their research to the general public. Each theory must by submitted to an international dialogue among peers, which takes places in journals, conferences and, more recently, on the Internet.
Unfortunately, alongside this type of research, which must be encouraged, supported and promoted, another sort is raising its head. The latter sort is pursued by researchers who do not abide by the above-mentioned rules. These researchers formulate a theory, fall in love with it, and immediately implement it by way of proposals for a therapy, then they find enthusiastic followers and propagate new pathogenic theories or therapies as if they were not merely theoretical, or still in the trial phase, but proven causes and therapies of documented efficacy.
Medical scientists, when they wish to document or validate the efficacy of a new therapy, adopt a methodology based on the study of two comparable groups: one group is treated with the therapy under scrutiny, whilst the other group is administered a non-therapy, i.e. a so-called ‘placebo’, which is an inactive therapy indistinguishable from the active drug. Neither the patient nor her/his family nor the doctor who is treating her/him must know whether the patient is taking the drug or a placebo (a ‘Double-blind Trial’). After a pre-determined period of time, stock is taken of the patient’s improvement, if any, still without knowing the type of treatment used. Only after the appropriate assessments are the envelopes opened, which contain the names of the drug takers and the placebo takers. Statistical calculations are then performed, and a verdict is issued on the balance of probabilities to establish whether the differences in improvements between the main group and the placebo or control group are statistically significant. A possibility still remains (five per cent or one percent) that these differences may have occurred by chance. Further studies may or may not confirm the early results.
The above method is not the fruit of methodological sadism by scientists intent on torturing patients while they wait for their treatment. It is dictated by an awareness of the history of medicine, which shows us that therapies have been applied for centuries in the conviction that they would be beneficial, then turned out to be useless or even harmful. Examples of this are bloodletting to treat fainting fits or pneumonia or live-cell therapies administered twenty years ago or so to autistic patients, now in their thirties.
Unless we conduct controlled trials, we run the risk of treating patients with ‘placebos’, i.e. bogus therapies which act only on the suggestionable minds of patients and their relatives. Many ‘therapies’, publicised by health professionals to their clients bypassing peer reviews have not been validated and rely for their effectiveness on the testimony of individuals, a meaningless exercise which will not demonstrate whether we are dealing with an effective drug or a placebo.
Left in a vacuum in regard to knowledge of effective therapies for Autism, many parents enthuse and exclaim triumphantly on the Internet’s mailing lists, or urge other parents to undertake costly journeys to America in a quest for outlandish tests and therapies, yet refuse to take part in trials intended to verify the effectiveness of a new therapy. The reason for this is that an honest professional will openly state that s/he is conducting trials, whereas a dishonest one may charge the earth for therapies which have never been validated as being efficacious.
The same enthusiastic parents will profess contempt for ‘Italy’ and admiration for ‘America’, as if there existed geographical boundaries in Science. The real boundaries are those between scientists who abide by the ethics of experimentation and those who do not, selling illusions. The latter exist in all nations, and America is no exception.
We noticed the birth of hypotheses and proposals for Autism therapy which seemed interesting to us, so we hoped they would be validated and confirmed.
We witnessed instead the opposite situation: no-one was able to validate those theories, yet at some point in time someone began propagating them as if they had been validated and their effectiveness were beyond doubt.
Immediately, a cohort of parents began distrusting Italian science and extolling American science, not realising that real scientists have dialogue with one another the world over, and true knowledge knows no boundaries, just as collective delusion knows no boundaries (see e.g. the ‘iceberg mother’ theory, it too being of American origin).
Reichelt’s hypothesis on urinary peptids and on the efficacy of a gluten/casein free diet was formulated some fifteen years ago but has not yet been universally validated. It would be appropriate to start off with subjects whose parents report an improvement in connection with the diet, and then methodically verify whether there exists a causal connection with the diet or not. To achieve this, few resources are needed.
Intestinal dysmicrobism and the ensuing treatment with probiotics, antifungals and vancomicin is also a hypothesis which has not yet been validated.
Likewise, with chelation of heavy metals and homotoxicology.
The only evidence provided by the promoters of these new therapies to document their efficacy is the positive feedback from some parents. No-one ever tells us how many took the treatment, nor what percentage found it beneficial. In anticipation of the international Congress in Barcelona in Autumn 2002, which was attended by many doctors who advocate treatment with unvalidated therapies, we read and heard an argument which proves extremely attractive to parents, in particular to parents of small children: ‘You cannot afford to wait, as children are less and less likely to improve with the passage of time. We know that some children have registered some improvements, according to their parents, after undergoing this or that therapy: have a go and, with a bit of luck, your child will improve too’. This attitude has proved extremely attractive to parents. The juxtaposition of all such therapies, some of which are often in conflict with one another, at one large international meeting, was a brilliant publicity stunt: none of the promoters of one specific theory had to measure themselves against other promoters because, in accordance with the above-mentioned principle, children will be led through a number of therapies for which, one after the other, their parents will have to pay, until they realise that they have wasted their time and resources. Some of them may even be glad to have paid up for the sake of preserving the chimera of their children’s recovery.
What is puzzling is that some neo-behaviorists, i.e. psycho-educationalists who had meticulously provided evidence of the efficacy of their therapies compared with other treatments, then agreed to rub shoulders with other practitioners who offer cures and improvements without providing any evidence for their theoretical efficacy.
Controlled trials, with a placebo/control group are not mentioned. This information is conveyed through non-accredited bulletins or the Internet, and none of the publications are found in reputable, peer-reviewed journals.
We most certainly do not despise the poor desperate customers, in this case the parents, who find themselves in a situation of extreme vulnerability and weakness.
We believe health ‘professionals’ bear a heavy responsibility whenever they publicise the success of therapies whose efficacy has never been validated.
Obviously, if there were substantial progress in the knowledge and therapy of Autism, all bogus therapies would vanish. Progress, however, does not fall from heaven. It is the outcome of serious, patient and well-funded research. If the resources which are invested in bogus therapies were to be devoted to reputable research, we would be taking gigantic strides.
Yet industry shows little interest, possibly fearing that a negative outcome might have a negative effect on its reputation: journalists are always keen to make headlines of those who carry out experiments on disabled children and dole out pills to them. In the public sector, only a few professional researchers are interested in pursuing this field, which has not been proved to be very satisfactory; on the other hand many parents, instead of encouraging proper research, on the one hand allow themselves to be ‘spellbound by sirens’ promising the impossible, on the other hand will not collaborate with genuine researchers who will call a spade a spade, informing parents on the distinction between validated therapy and research studies.
It seems paradoxical, yet it is true: over time we have had to fight, and are still fighting, against psychotherapists who remain focussed on with psychodynamic, systemic, do-it-yourself or ethological paradigms, and who persist in making the same false assumptions as those of Kanner, Bettelheim, Tustin and the Tinbergens. We demolished their credibility vis-a-vis parents, who now don’t trust ‘experts’ and believe they can cope on their own. Some of these parents have actually initiated therapies by themselves, first for their own children and then for other people’s children, thus becoming specialised ‘practitioners’. Unfortunately, very few of these have agreed to submit their theories to clinical trials and to a review of outcomes by independent referees. Thus, do-it-yourself practices continue to be used, resulting in those collective delusions with which we are well acquainted. On the other hand, we all know that that private medicine may hinder us from carrying out genuine tests of efficacy: it may be that those who get paid will not easily admit that their treatment is useless; private medicine could operate selectively, in the sense that those who are satisfied with their treatment (or wrongly believe they have improved) stay loyal to their specialist, whereas those who believe to have been badly treated will refuse to continue paying their specialist and will go elsewhere, thus preventing their own specialist from assessing poor outcomes.
Last year, we put pressure on six Regions (Abruzzo, Calabria, Umbria, Marche, Emilia Romagna and Veneto) to persuade them to launch a research project, funded by the Ministry for Health, for the assessment of psycho-educational therapies, in conjunction or not with pharmacological therapies. This research project had commenced in 2001 in Veneto and Emilia Romagna, using regional funds, and has already yielded early outcomes: i.e. data on the number of autistic subjects and relevant services. A request for funding has been approved, thanks also to other FISH associations (FISH = Italian Federation for Conquering Disability), and commenced officially in Winter 2002. Coordination is assured by Regione Abruzzo, whose President, Avv.to Tagliente, we sincerely thank for his support. An important role in all this was played by Germana Sorge, Chair of ANGSA Abruzzo. The scientific director of research is Professor Lucio Cottini, who also heads the excellent Regione Marche project (see Antonella Foglia’s report in the present Bulletin). This year, a research project has been mooted, which extends the previous one, embracing the Regions of Sicily, Puglia, Piedmont and Tuscany. We hope the project will be endorsed by the Ministry. A nation-wide monitoring centre could then be set up.
Led by Stella Maris, the Science Research Institutes, IRCCS, have requested an appropriate research grant from the Ministry, with the involvement of Professor Flavio Keller, who is still awaiting the promised research funding from FIRB (Research Grants Office) in the Ministry for Research. For us it is already a victory that the conditions for research grant include a whole page on Autism. This compares favourably with our efforts six years ago to secure the inclusion of two lines on Autism as an organic illness in a similar competitive grant scheme. Better news comes in relation to progress in research on Reeline, the outcome of which will soon be published by the prestigious journal Science, also over Professor Keller’s name. At the same time, genetic research is progressing.
One example of fruitful research has been achieved by Professor Costantino Salerno, who had discovered the relationship of adenylosuccinate lyase to Cryptogenic Autism (i.e. due to unknown causes) in a child, F.T. Salerno replicated a test involving methylxanthine on the urine of autistic and non-autistic subjects after they had ingested a chocolate bar, with the cooperation of ANGSA, AUT AUT, a teacher and others. The first researcher had detected substantial anomalies in half of the autistic subjects, as they seemed to retain that stimulating substance, present in cocoa, for longer periods than the other subjects. Professor Salerno obtained different outcomes and noticed that there is no significant difference between autistic and non-autistic patients, and will publish these results in an international journal. Researchers normally do not like invalidating the results of colleagues’ research, and it is often difficult to publish research outcomes which merely contradict the hypotheses of others. However, this is the way scientific research advances, in that (barring invalidations of invalidations) we ought to conclude that Autism deficits are not due to the metabolising of chocolate. Research continues along other paths...
We do all we can to assist researchers who assist us, while expecting nothing in return.
At least, let us show them our gratitude.
Daniela Mariani Cerati