C1 ALTERNATIVE MEDICINE‐ HYPE OR HOPE?
Barrett S
Board Chairman, Quackwatch, Inc., NCAHF Vice President and Director of Internet Operations, P.O. Box 1747, Allentown, PA 18105, USA
E‐mail address for correspondence: [email protected]
‘Alternative medicine’ has become the politically correct term for questionable practices formerly labeled quack and fraudulent. During the past 15 years, many reports, even in medical journals, have contained no critical evaluation and have featured the views of proponents and their satisfied clients.
To avoid confusion, ‘alternative’ methods should be classified as genuine, experimental, or questionable. Genuine alternatives are comparable methods that have met science‐based criteria for safety and effectiveness. Experimental alternatives are unproven but have a plausible rationale and are undergoing responsible investigation. The most noteworthy is use of a 10%‐fat diet for treating coronary heart disease. Questionable alternatives are groundless and lack a scientifically plausible rationale. The archetype is homeopathy, which claims that ‘remedies’ so dilute that they contain no active ingredient can exert powerful therapeutic effects. Blurring these distinctions enables promoters of quackery to argue that because some practices labeled ‘alternative’ have merit, the rest deserve equal consideration and respect. Enough is known, however, to conclude that most questionable ‘alternatives’ are worthless.
The ‘alternative movement’ is part of a general societal trend toward rejection of science as a method of determining truths. In line with this philosophy, ‘alternative’ proponents assert that scientific medicine (which they mislabel as allopathic, conventional, or traditional medicine) is but one of a vast array of health care options. Instead of subjecting their work to scientific standards, they would like to change the rules by which they are judged and regulated. ‘Alternative’ promoters often gain public sympathy by portraying themselves as a beleaguered minority fighting a self‐serving, monolithic ‘Establishment’.
Under the rules of science, people who make the claims bear the burden of proof. It is their responsibility to conduct suitable studies and report them in sufficient detail to permit evaluation and confirmation by others. Instead of subjecting their work to scientific standards, promoters of questionable ‘alternatives’ would like to change the rules by which they are judged. ‘Alternative’ promoters may pay lip service to these standards. However, they regard personal experience, subjective judgement, and emotional satisfaction as preferable to objectivity and hard evidence. Instead of conducting scientific studies, they use anecdotes and testimonials to promote their practices and political maneuvering to keep regulatory agencies at bay.
C2 PHENOTYPIC VARIATION IN MND/ALS
Leigh PN
King's MND Care and Research Centre, PO 41, Department of Clinical Neuroscience, Institute of Psychiatry, King's College London, UK
‘The fundamental law should be engrained that the starting point of all treatment is in a knowledge of the natural history of a disease’. William Osler, 1907.
Perhaps identifying a ‘cure’ for MND should be no more challenging than finding effective treatments for asthma, heart disease, cancer, epilepsy, or multiple sclerosis. All are heterogeneous disorders and in each case management relies largely on therapeutic agents that act on generic disease mechanisms and take little account of the complexities of individual variation. Our hope for MND is that common molecular mechanisms and therefore common therapeutic targets will link all forms of MND. Our fear is that new therapies will be effective only in sub‐groups of people with rare sub‐types of MND. The relevance of phenotypic variation in relation to the care of people with MND/ALS is evident in relation to symptom control, but it is not yet clear what heterogeneity means for disease modifying therapies – for example, riluzole. We do not know if there are responders and non‐responders. The challenge is to find methods for understanding the molecular basis of clinical heterogeneity and to integrate such methods into clinical trials and epidemiology.
First we must understand the nature of clinical and genetic heterogeneity in MND. A disease entity is well exemplified by those forms of familial MND for which gene mutations have been identified such as familial MND associated with SOD1 or VAPB mutations, or linked to loci on chromosomes 9, 16, 18, or 20. Other MND genes remain to be localized to a chromosomal locus. All these examples could be said to be different diseases, although all share some or all of the typical clinical features of MND. While some SOD1 gene mutations are associated with a stereotyped phenotype, others are not. Genetic background appears to influence phenotype (e.g. in D90A homozygous familial MND). Specific gene therapy for people with SOD1 gene mutations is being developed. The same approach may be needed for the other gene mutations, at huge effort and cost and for the benefit of relatively few individuals at risk.
There is also striking phenotypic variation in sporadic MND/ALS. The differences in prognosis between bulbar onset and limb onset disease are well known, but it is important to recognize other MND syndromes, such as the ‘flail arm’ and ‘flail leg’ syndromes, and lower motor neuron forms of MND (PMA‐ progressive muscular atrophy). The presence of cognitive change in a significant proportion of people with MND is now apparent. This was hardly discussed back in 1990. These phenotypic variations must be recognized and their biological basis better understood if physicians are to tailor advice on disease progression, prognosis, drug therapy, and care to the needs of the individual.
How can this be achieved? Undoubtedly we are missing opportunities to understand the biological basis of these important variations within the rubric of MND/ALS by our failure to pool understanding and resources nationally and internationally. Large‐scale clinical trials and prospective epidemiological and natural history studies can furnish prospectively acquired clinical information linked with DNA and pathology resources that will be necessary to attack these issues. Hundreds of MND patients and controls – most likely thousands – are needed if this approach is to succeed.
We should now rapidly advance beyond the era of anecdote and small studies to building international collaborations that will allow us, in time, to understand the molecular basis of MND in its various forms – familial, sporadic, bulbar onset and limb onset, flail arm and flail leg, with and without cognitive impairment and so on. At the same time we should develop new methods to understand individual and sub‐group responses to disease‐modifying treatments. The arrival of a generic therapy that halts disease progression in all forms of MND may of course obviate all this effort. Let us hope so. But perhaps, like AE Houseman, we should: ‘Do as a wise man would, and train for ill, and not for good’.