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Post by David Feder on Sept 23, 2006 17:12:29 GMT -5
Experimental cancer drugs counter muscle deterioration seen in muscular dystrophy
Researchers test previously 3 drugs: trichostatin A, phenybutirate and valproic acid. They choice trichostatin A because the drug was more effective.
Valproic acid is the only drug available to use now, because the drug is used in a type of epilepsy in children. The side effect is hepatic toxicity. If valproic acid inhibit desacetylase efficiently this is the most close drug of this group to clinical trial in DMD in few time
The cost of Trichostatin for experimental use is high: the cost of 1 mg is US$ 105. If the dose is the same of mice - diary cost more than US$ 4,000.
Vorinostat is another cancer drugs with the same action (deacetylase inhibitor). Vorinostat was tested in lymphoma and FDA will approved the drug to use in cancer in October/2006.
David
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Post by David Feder on Sept 23, 2006 17:13:59 GMT -5
If the dose is the same of mice - diary cost for human use is more than US$ 4,000.
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Post by bhopeful on Sept 23, 2006 19:41:15 GMT -5
Dr. Feder:
Do you know if trichostatin A has been used in humans and/or has it been approved for use by the FDA or any other regulatory agency ?
How serious is the side effect hepatic toxicity when using Valproic acid?
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Post by David Feder on Sept 23, 2006 20:21:54 GMT -5
I am sure that trichostatin don't was approved by FDA. I don't see articles in human trial until this moment.
Valproic acid is a well know drug used in a type of epilepsy in children (Absence). The side effect in liver is rare but it's possible to be very serious. There are report of serious hepatic failure and death after the medication but there are blood test to diagnosis this condition and the drug can be interrupted. You can see the advertise in the instruction of Depakote:
BOX WARNING:
HEPATOTOXICITY:
HEPATIC FAILURE RESULTING IN FATALITIES HAS OCCURRED IN PATIENTS RECEIVING VALPROIC ACID
AND ITS DERIVATIVES. EXPERIENCE HAS INDICATED THAT CHILDREN UNDER THE AGE OF TWO YEARS
ARE AT A CONSIDERABLY INCREASED RISK OF DEVELOPING FATAL HEPATOTOXICITY, ESPECIALLY
THOSE ON MULTIPLE ANTICONVULSANTS, THOSE WITH CONGENITAL METABOLIC DISORDERS, THOSE
WITH SEVERE SEIZURE DISORDERS ACCOMPANIED BY MENTAL RETARDATION, AND THOSE WITH
ORGANIC BRAIN DISEASE. WHEN DEPAKOTE IS USED IN THIS PATIENT GROUP, IT SHOULD BE USED WITH
EXTREME CAUTION AND AS A SOLE AGENT. THE BENEFITS OF THERAPY SHOULD BE WEIGHED AGAINST
THE RISKS. ABOVE THIS AGE GROUP, EXPERIENCE IN EPILEPSY HAS INDICATED THAT THE INCIDENCE OF
FATAL HEPATOTOXICITY DECREASES CONSIDERABLY IN PROGRESSIVELY OLDER PATIENT GROUPS.
THESE INCIDENTS USUALLY HAVE OCCURRED DURING THE FIRST SIX MONTHS OF TREATMENT.
SERIOUS OR FATAL HEPATOTOXICITY MAY BE PRECEDED BY NON-SPECIFIC SYMPTOMS SUCH AS
MALAISE, WEAKNESS, LETHARGY, FACIAL EDEMA, ANOREXIA, AND VOMITING. IN PATIENTS WITH
EPILEPSY, A LOSS OF SEIZURE CONTROL MAY ALSO OCCUR. PATIENTS SHOULD BE MONITORED CLOSELY
FOR APPEARANCE OF THESE SYMPTOMS. LIVER FUNCTION TESTS SHOULD BE PERFORMED PRIOR TO
THERAPY AND AT FREQUENT INTERVALS THEREAFTER, ESPECIALLY DURING THE FIRST SIX MONTHS.
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Post by David Feder on Sept 23, 2006 20:35:39 GMT -5
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Post by bhopeful on Sept 24, 2006 19:28:22 GMT -5
Dr. Feder: If Valproic Acid is currently being prescribed for use by children for other disorders, does this mean if researchers wanted to do clinical trials for DMD, phase 1 trials could be by-passed?
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Post by David Feder on Sept 24, 2006 20:18:43 GMT -5
It's right. It's possible to start clinical trial in phase 2 (DMD patients). We can see results in few time. And the drugs is inexpensive and is very knowledge from doctors.
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Post by bhopeful on Sept 24, 2006 21:13:13 GMT -5
Dr. Feder: What do we as parents and grandparents of boys with DMD have to do to push this drug to trial or will the drug company see an opportunity and move it forward to trial themselves.
I hope organizations like Parent Project MD and the MDA will push for this drug and others like it to go to trials as soon as possible. Hopefully they will put predjudices and differences aside and do what is right for all those boys affected by DMD.
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Post by ljsdad on Sept 25, 2006 0:55:37 GMT -5
i know this is great but the research did not say valpronic acid ,it said tricostatin A.
Surly out there some where some paitent is taking this valpronic acid for something else.
it would be interesting to hear from them.
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Post by David Feder on Sept 25, 2006 6:07:49 GMT -5
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Post by sonrise on Sept 25, 2006 6:56:41 GMT -5
David,
How and why were these experimental cancer drugs effective?
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Post by David Feder on Sept 25, 2006 8:23:49 GMT -5
I'm not sure but I think that this drugs turn on or turn off some genes. I don't know if this drug can correct some genes. The results of this article is very solid in mdx mice. They use in mice with more age than other research and the pathological aspect is the same of normal mice. I don't know if the result will be the same in human disease.
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Post by David Feder on Sept 25, 2006 8:31:30 GMT -5
Hi all: I have the full article an I can send only by e-mail because this article is protected by copyright. Please ask for the article in my e-mail (feder2005@gmail.com or david-feder@uol.com.br
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Post by David Feder on Oct 5, 2006 11:08:47 GMT -5
Drug may help treat muscular dystrophy www.upi.com/ConsumerHealthDaily/view.php?StoryID=20061004-123314-1102rLosartan act similar to ACE inhibitor. DMD people can present cardiac dysfunction and need treatment with ACE inhibitor and beta-blockers. Some doctors recommend the early use of this medications to prevent this dysfunction. Losartan can give the same effect of ACE inhibitor. In other words if the doctor change the recommendatin and instead of ACE inhibitor they recommend losartan it’s possible to prevent cardiac dysfunction and increase the muscle force and/or muscle regeneration. |
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Post by Michelle Scaglione on Oct 5, 2006 16:24:13 GMT -5
David
Are you going to put your son on Losartan?
Michelle
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