FDA lifts hold of promising drug to treat Lou Gehrig's disease

The FDA has lifted a clinical hold after two years on a promising new drug for Lou Gehrig's disease. The manufacturer believes it could be a breakthrough drug for other diseases such as Alzheimer's, Parkinson's and Huntington's disease.

The U.S. Food and Drug Administration (FDA) has accepted biopharmaceutical company, CytRx Corporation's revised clinical trial protocol for arimoclomol, a new drug that has shown great promise in patients with Lou Gehrig's Disease (ALS). The move by regulators will allow researchers to test the drug in patients at levels up to four times the dose used in a Phase IIa trial. The protocol will allow a maximum dose during testing of 400 milligrams, three times daily, the company said in a press release. Arimoclomol has been awarded fast track designation and orphan drug status for the treatment of Lou Gehrig's disease, a disease in which certain nerve cells in the brain and spinal cord slowly die, making it hard to walk, speak, eat, swallow, and breathe, leading to injury, illness, and eventually death, says WebMD. ALS is a rare disease, affecting only 1 to 2 people out of 100,000 people worldwide. Orphan drug designation has been instrumental in providing incentives and fast tracking drugs to be used in the treatment of rare diseases. For instance, in the decade before 1983 only 10 drugs for rare diseases were brought to market but the since the Orphan Drug Act came into being there have been over 200 new treatments made available for patients with conditions that would have otherwise been ignored. CytRx President and CEO Steven A. Kriegsman said in a company press release, "We are delighted with the FDA's decision to approve a protocol that provides a safe and efficient avenue to ascend arimoclomol dosing to the target dosage level of 400 mg three times daily, which we believe will prove most effective in treating ALS patients. Arimoclomol's underlying molecular chaperone amplification method of action has shown promising clinical and preclinical study results in inhibiting the progression of ALS, a debilitating disease that currently lacks effective therapeutic treatment. We believe that this drug candidate has the potential to be a breakthrough drug with application in a number of other neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease and Huntington's disease, and as a treatment for stroke recovery and neuropathy. Recent independent findings also indicate that molecular chaperone technology represents a significant strategy in the future design of anti-aging pharmaceuticals." Kriegsman also says, "We have attracted significant interest from several pharmaceutical companies as potential partners to advance development of our molecular chaperone programs, and we view clearance to resume arimoclomol clinical testing as a key step in moving these negotiations forward." According to a Reuters report, Shares of CytRx were up as high as 16 per cent..