John1
Very helpful member
- Joined
- Feb 25, 2006
- Messages
- 1,055
- Diagnosis
- 10/2000
- Country
- CA
- State
- NL
- City
- Newfoundland
Results of Patients Like Me trial for lithium
The results of a large patient-run lithium trial for lithium as an ALS therapy indicated no benefit after 6 months but did note some adverse affects. Personally, I took lithium from January to September last year and kept monthly records of my ALSFRS scores and observed no slowing of my progression from my pre-lithium period but did have adverse gastrointestinal side effects.
Here is a copy of the study's submission last month to the New England Journal of Medicine for inclusion as a "Letter".
To the Editor, In Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s Disease) motor neurons die, leaving the patient progressively unable to move, speak, eat, or breathe. A small clinical trial indicated that lithium combined with riluzole slowed ALS progression1. A number of patients began taking lithium to quantitatively track the progression of ALS. A focus of the effort was making data and graphs publically available in real time. We found that lithium does not slow ALS progression but does produce side effects in about 49% of patients, with severe side effects in 12%.
Patients took lithium under a doctor’s supervision and were asked to report their ALS Functional Rating Score - Revised, or ALSFRS-R2 monthly for six months. 60% of the patients opted to take lithium and riluzole, while 40% took only lithium. Of the 191 patients who started the study, 71 stopped taking lithium because of side effects or faster progression. Of the patients remaining on lithium 110 were still reporting data at 3 months and 50 at 6 months. 37 of the patients who stopped lithium reported scores at six months. 63 patients from the group who did not take lithium were used as controls. The study and control populations were very similar (mean ages differed by only 0.2 years, mean loss rate of ALSFRS-R by 7%). The study group had more men (82% vs 59%), but no difference in progression rates was seen as a function of gender in either group.At least 7 patients passed away and at least 3 went on ventilators. Patients who remained on lithium progressed at the same rate as controls (Figure 1). Patients who stopped lithium reported a faster progression, indicating that lithium may have worsened their disease prompting the discontinuation. No difference in progression was seen as a function of lithium blood level, initial ALSFRS-R score, or riluzole usage. Common side effects included weakness, increased urination, increased fasciculations, headache, fatigue, and nausea. 16 patients reported positive effects of relief from severe cramping, fasciculations, spasticity, and depression.
A study of this nature would be expected to have positive bias as sicker patients are less likely to continue reporting and there is no placebo control. Thus the finding of a negative result is compelling.
Karen Felzer, caregiver
Humberto Macedo, patient
References
1. Fornai F, Longone P, Cafaro L, et al., Lithium delays progression of amyotrophic lateral sclerosis. Proc. Natl. Acad. Sci 2008; 105:2052-2057.
2. Cedarbaum JM, Stambler N, Malta E., et al., The ALSFRS-R scale: A revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study group (Phase III). J. Neurol. Sci 1999; 169:13-21.
The results of a large patient-run lithium trial for lithium as an ALS therapy indicated no benefit after 6 months but did note some adverse affects. Personally, I took lithium from January to September last year and kept monthly records of my ALSFRS scores and observed no slowing of my progression from my pre-lithium period but did have adverse gastrointestinal side effects.
Here is a copy of the study's submission last month to the New England Journal of Medicine for inclusion as a "Letter".
To the Editor, In Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s Disease) motor neurons die, leaving the patient progressively unable to move, speak, eat, or breathe. A small clinical trial indicated that lithium combined with riluzole slowed ALS progression1. A number of patients began taking lithium to quantitatively track the progression of ALS. A focus of the effort was making data and graphs publically available in real time. We found that lithium does not slow ALS progression but does produce side effects in about 49% of patients, with severe side effects in 12%.
Patients took lithium under a doctor’s supervision and were asked to report their ALS Functional Rating Score - Revised, or ALSFRS-R2 monthly for six months. 60% of the patients opted to take lithium and riluzole, while 40% took only lithium. Of the 191 patients who started the study, 71 stopped taking lithium because of side effects or faster progression. Of the patients remaining on lithium 110 were still reporting data at 3 months and 50 at 6 months. 37 of the patients who stopped lithium reported scores at six months. 63 patients from the group who did not take lithium were used as controls. The study and control populations were very similar (mean ages differed by only 0.2 years, mean loss rate of ALSFRS-R by 7%). The study group had more men (82% vs 59%), but no difference in progression rates was seen as a function of gender in either group.At least 7 patients passed away and at least 3 went on ventilators. Patients who remained on lithium progressed at the same rate as controls (Figure 1). Patients who stopped lithium reported a faster progression, indicating that lithium may have worsened their disease prompting the discontinuation. No difference in progression was seen as a function of lithium blood level, initial ALSFRS-R score, or riluzole usage. Common side effects included weakness, increased urination, increased fasciculations, headache, fatigue, and nausea. 16 patients reported positive effects of relief from severe cramping, fasciculations, spasticity, and depression.
A study of this nature would be expected to have positive bias as sicker patients are less likely to continue reporting and there is no placebo control. Thus the finding of a negative result is compelling.
Karen Felzer, caregiver
Humberto Macedo, patient
References
1. Fornai F, Longone P, Cafaro L, et al., Lithium delays progression of amyotrophic lateral sclerosis. Proc. Natl. Acad. Sci 2008; 105:2052-2057.
2. Cedarbaum JM, Stambler N, Malta E., et al., The ALSFRS-R scale: A revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study group (Phase III). J. Neurol. Sci 1999; 169:13-21.
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