Report on the future of RNAi-based therapeutics & diagnostics

The market for RNAi-based therapeutics is forecast to grow from 2013 onwards, as the first products enter the marketplace, to generate sales in excess of US$2.9 billion by 2020. The first siRNA based therapeutics will capitalize on the demand to treat viral infections and ocular conditions and in the longer term companies will be able to target niche areas of high unmet clinical need such as cancer, cardiovascular disease, metabolic disorders, inflammatory and neurological conditions.

More.....Report on the future of RNAi-based therapeutics & diagnostics

Positive results from phase 3 clinical trials of Linoclotide ....

Ironwood Pharmaceuticals, Inc. and Forest Laboratories, Inc. recently announced positive top‐line results from two Phase 3 clinical trials assessing the safety and efficacy of once‐daily dosing of the investigational drug linaclotide in patients with chronic constipation (CC). Analyses of the data indicate that in both multicenter, randomized, double‐blind, placebo‐controlled trials, statistical significance was achieved for the primary endpoint of 12‐week complete spontaneous bowel movement (CSBM) overall responder at the two doses studied in each trial (133 mcg/day: p‐values≤0.0012 and 266 mcg/day: p‐values<0.0001). In both trials, statistical significance (p<0.01) was achieved for all prespecified secondary endpoints, which included measures of bloating, abdominal discomfort, and average weekly CSBMs.

About Linaclotide :

Linaclotide (see the structure) is an orally delivered peptide that acts locally in the gut with no detectable systemic exposure at therapeutic doses and is intended for once‐daily administration. Linaclotide can be synthesized by solid-phase technology using Fmoc protections. Amino acids are coupled using DCC/HOBT and Fmoc groups are removed by means piperidne .Cysteinethiol groups are protected with trityl and cleavage of the peptide from the resin is done by TFA.

Mode of action :

Linoclotide acts agonist of guanylate cyclase type‐C (GC‐C), a receptor found on the lining of the intestine. Activation of GC‐C leads to increases in intracellular and extracellular cGMP. In preclinical models, extracellular cGMP inhibited afferent nerve firing and positively affected markers of abdominal pain, while intracellular cGMP led to activation of anion channels which stimulated anion and fluid secretion into the intestine, leading to accelerated intestinal transit. Linaclotide is a first‐in‐class compound in Phase 3 clinical development for the treatment of IBS‐C and CC.

Ref : http://www.ironwoodpharma.com/newsPDF/Linaclotide.Ph3.CC.results.11.02.09.pdf

A new blood test for breast cancer

A new blood test for breast cancer

FDA authorizes emergency use of IV antiviral "Peramivir"...

As for as my knowledge goes Peramvir, is an experimental antiviral drug, and being developed by BioCryst Pharmaceuticals for the treatment of influenza. Peramivir is a neuraminidase inhibitor, acting as a transition-state analogue inhibitor of influenza neuraminidase and thereby preventing new viruses from emerging from infected cells.

The development of peramivir is supported by the US Department of Health and Human Services as part of the US government's effort to prepare against the threat of an influenza pandemic.

The drug has had a long history. An oral formulation was abandoned by Johnson and Johnson due to poor bioavailability. BioCryst Pharmaceuticals is now developing a injectable version, in partnership with Green Cross Pharmaceuticals in South Korea and with Shionogi Pharmaceuticals in Japan. The drug is in Phase II studies

More interesting recent news (Oct. 23, 2009) about this drug is that, FDA has announced the issuing of an emergency use authorization (EUA) for the investigational antiviral drug peramivir intravenous (IV) in certain adult and pediatric patients with confirmed or suspected 2009 H1N1 influenza infection who are admitted to a hospital.

Specifically, IV peramivir is authorized only for hospitalized adult and pediatric patients for whom therapy with an IV drug is clinically appropriate based on one or more of the following reasons:

1. the patient is not responding to either oral or inhaled antiviral therapy, or
2. when drug delivery by a route other than an intravenous route -- e.g., enteral (absorbed by the intestines) or inhaled -- is not expected to be dependable or feasible;
3. for adults only, when the clinician judges IV therapy is appropriate due to other circumstances.....

more..

Steroid-induced osteoporosis can now be treated with Teriparatide

Steroid-induced osteoporosis can now be treated with Teriparatide

Geron plans to advance clinical program for spinal cord injury

In my earlier blog (Euphoria over Stem Cell Therapy.....) did mentioned that, there are many companies which falsely claimed that they have got technology (stem cell treatment) to treat spinal cord injury. But an established company like Geron, has plan to advance clinical development of its human embryonic stem cell (hESC)-based product, GRNOPC1, for the treatment of spinal cord injury. The plan is expected to enable Geron to re-initiate the Phase I clinical trial of GRNOPC1 in patients with complete thoracic spinal cord injury and to support future expansion of the trial to patients with cervical injuries.

I read earlier about the preclinical studies of GRNOPC1 and the company claimed that it is developing GRNOPC1 for spinal cord injury, but is also exploring application for other neurological diseases, including multiple sclerosis, stroke and Alzheimer disease really interesting to see the results.

As I had an opportunity to talk and work (contract research) with Geron [known for its first-in-class biopharmaceuticals (especially "antisense drugs" from oligonucleotides) for the treatment of cancer and chronic degenerative diseases] company, I know its credentials and am really excited to see some interesting out come from their advance clinical program. All the best....

Ref : http://www.geron.com/media/pressview.aspx?id=1195

Botox for Chronic Regional Pain Syndrome (CRPS). ...

We know that Botulinum toxin is a medication and a neurotoxic protein produced by the bacterium Clostridium botulinum, and is held to be the most toxic substance known to mankind with an LD50 of roughly 0.005-0.05 µg/kg. Despite its greatest known toxic effect, it is also used in very small doses to treat muscle spasms. The acceptance of BTX-A use for the treatment of spasticity and muscle pain disorders is growing, with approvals pending in many European countries and studies on headaches (including migraine), prostatic symptoms, asthma, obesity and many other possible indications are ongoing.

But some interesting findings of the same drug has been presented at the American Society of Plastic Surgeons (ASPS) Plastic Surgery 2009 conference, Oct. 23-27, in Seattle, i.e., botox can be used as a pain medication to fight Chronic Regional Pain Syndrome (CRPS).

The study found injecting Botox into the area affected by pain provides significant pain control. Eight patients suffering from severe pain received an average of nine injections , one every four weeks. All of patients reported a significant improvement (31.25 percent) in their daily pain control that was maintained for up to 17 months. Though the drug has been used to treat many problems like cervical dystonia (CD), strabismus, blepharospasm, primary axillary hyperhidrosis, glabellar lines (wrinkle fighter). this finding is more significant....

Ref : http://www.plasticsurgery.org/Documents/Media/ps2009/19-Botox-Chronic-Pain.pdf

PEDOT helps Artificial Limbs To Feel Heat, Cold, Touch !

We know that, Poly(3,4-ethylenedioxythiophene) or PEDOT (or sometimes PEDT) is a conducting polymer based on 3,4-ethylenedioxylthiophene or EDOT monomer. Advantages of this polymer are optical transparency in its conducting state, high stability and moderate band gap and low redox potential. A large disadvantage is poor solubility which is partly circumvented in the PEDOT:PSS composite, and the PEDOT-TMA material. (Note: Pedot is a transparent conductor. These conductors are used for LCDs and solar cells, among others.)

But more interesting property of this polymer has been established by Physicians at the American Society of Plastic Surgeons (ASPS). They have discovered that the polymer (3, 4-ethylenedioxythiophene or PEDOT) helps stimulate and grow new nerve fibers in severed nerves of amputees. Stimulating and growing nerve fibers are one of the first steps in providing amputees more neurologic control over their prosthetics. PEDOT functions similar to a wire. In the study, the PEDOT was placed in a tube, along with other biologic and synthetic materials, and grafted into the severed leg nerve of a rat. New nerve fibers grew and took over function for the dead or dysfunctional severed nerve springing targeted muscles to life.

Most interesting is the other study, plastic surgeons designed a cup containing cells and muscle that fits around the severed leg nerve of a rat. The PEDOT polymer was wrapped around all of the cells and muscle in the cup to provide an electrical charge. Tests were conducted 114 days after the procedure. The study found new muscle and blood vessels formed, nerve fibers sprouted, and muscle fibers started compensating for lost nerves. After tickling the rat's paw, doctors' were able to pick up electrical signals indicating sensation had returned. Hope in the days to come "real feeling" to artificial limbs.....

One can read/view this slide show..

Vegetables Can Protect Unborn Child Against Diabetes

Expecting mothers who eat vegetables every day seem to have children who are less likely to develop type 1 diabetes, a new study from the Sahlgrenska Academy has revealed......


More......Vegetables Can Protect Unborn Child Against Diabetes

Triple-combo Drug for Antiviral-resistant H1N1 ?

In laboratory testing, the triple combination of oseltamivir (Tamiflu), amantadine (Symmetrel) and ribavirin showed a significant capacity to stop flu-virus growth, says Mark Prichard....

More...http://www.sciencedaily.com/releases/2009/10/091027132426.htm

Epilepsy Drugs Could Treat Alzheimer's And Parkinson's

Epilepsy Drugs Could Treat Alzheimer's And Parkinson's

Scientists Discover Gene that 'Cancer-Proofs' Rodent's Cells

In my earlier blog, I did mention about how naked mole rats may help cure cancer. The same researchers have achieved something interesting i.e., they have discovered have discovered the gene that "cancer-proofs the naked mole". For details...

Junk DNA Mechanism That Prevents Two Species From Reproducing Discovered

Cornell researchers have discovered a genetic mechanism in fruit flies that prevents two closely related species from reproducing, a finding that offers clues to how species evolve.

More ...Junk DNA Mechanism That Prevents Two Species From Reproducing Discovered

New 'Schizophrenia Gene' Prompts Researchers To Test Potential Drug Target

The newfound gene, dubbed KIAA1212, serves as a bridge linking two schizophrenia genes: DISC1 and AKT. Suspecting KIAA1212 as one of many potential binding partners interacting with DISC1, whose name is an acronym for "Disrupted-in-Schizophrenia," the researchers genetically shut down the production of DISC1 proteins in newly born neurons in the hippocampus region of an adult mouse brain. The hippocampus contains a niche where native stem cells give rise to fully developed new neurons

Read : New 'Schizophrenia Gene' Prompts Researchers To Test Potential Drug Target

Now Cobroxin, available online......

In my earlier blog, I did mention about the launch of Cobroxin as OTC pain reliever. Now its available online. More...