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Biotech / Medical : Biotech Valuation -- Ignore unavailable to you. Want to Upgrade?

To: Ian@SI who wrote (22550)	1/18/2007 9:49:24 AM
From: Casaubon	Read Replies (1) \| Respond to of 52153

"But who's going to pay for the clinical trials ... it's $70 million to $100 million." listen man, if this stuff has a chance, then every cancer charity fund in the world would foot the bill for the clinical trials. Now, I'm not saying it won't work, but don't spit in the face of real research being done in the pharmaceutical industry.

To: Ian@SI who wrote (22550)	1/18/2007 1:13:32 PM
From: Biomaven	Read Replies (1) \| Respond to of 52153

Well I looked at the journal article briefly. Interesting idea, and clearly worth pursuing. But the authors definitely lose some points with the following statement: A very attractive property of DCA is its selectivity, evident by the lack of any systemic toxicity in this (Figure 8D) and other recent animal (McMurtry et al., 2004) and human studies (Stacpoole et al., 2006). Compare that with: Neurology. 2006 Feb 14;66(3):324-30. Dichloroacetate causes toxic neuropathy in MELAS: a randomized, controlled clinical trial. * Kaufmann P, <snip> Department of Neurology, Columbia University, New York 10032, USA. pk88@columbia.edu OBJECTIVE: To evaluate the efficacy of dichloroacetate (DCA) in the treatment of mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes (MELAS). BACKGROUND: High levels of ventricular lactate, the brain spectroscopic signature of MELAS, correlate with more severe neurologic impairment. The authors hypothesized that chronic cerebral lactic acidosis exacerbates neuronal injury in MELAS and therefore, investigated DCA, a potent lactate-lowering agent, as potential treatment for MELAS. METHODS: The authors conducted a double-blind, placebo-controlled, randomized, 3-year cross-over trial of DCA (25 mg/kg/day) in 30 patients (aged 10 to 60 years) with MELAS and the A3243G mutation. Primary outcome measure was a Global Assessment of Treatment Efficacy (GATE) score based on a health-related event inventory, and on neurologic, neuropsychological, and daily living functioning. Biologic outcome measures included venous, CSF, and 1H MRSI-estimated brain lactate. Blood tests and nerve conduction studies were performed to monitor safety. RESULTS: During the initial 24-month treatment period, 15 of 15 patients randomized to DCA were taken off study medication, compared to 4 of 15 patients randomized to placebo. Study medication was discontinued in 17 of 19 patients because of onset or worsening of peripheral neuropathy. The clinical trial was terminated early because of peripheral nerve toxicity. The mean GATE score was not significantly different between treatment arms. CONCLUSION: DCA at 25 mg/kg/day is associated with peripheral nerve toxicity resulting in a high rate of medication discontinuation and early study termination. Under these experimental conditions, the authors were unable to detect any beneficial effect. The findings show that DCA-associated neuropathy overshadows the assessment of any potential benefit in MELAS. Here's another quote: The chronic use of dichloroacetate (DCA) for diabetes mellitus or hyperlipoproteinemias has been compromised by neurologic and other forms of toxicity. Ironically, DCA is viewed as an environmental toxin and has been viewed with concern when found in wastewater at several Superfund sites. Peter