ALS Patients May Find Hope in New Drug Treatment

By: Neomi Heroux

The FDA has announced that trial uses of Insmed’s drug Iplex will be allowed for certain patients with Lou Gehrig’s disease or ALS, amyotrophic lateral sclerosis. ALS is a progressive neurodegenerative disease that affects the brain and spinal cord, reducing the ability for the patient to control voluntary muscles and always leads to death. There are no proven treatment options.

Iplex had only been approved for treatment of a growth hormone deficiency, but is not being currently marketed because of a court order relating to patent infringement according to the FDA website. Many doctors have petitioned the FDA for permission to use the drug for specific patients with ALS, but the agency had, until now, denied the requests. Among the reasons cited for denial was the failure of other therapies that had been produced as potential treatments for ALS. The controlled clinical trials in those instances had shown no benefit and indicated that the treatments may have proven more detrimental to the patients than placebos.

Upon learning that the drug had a very limited supply and that clinical trials for ALS in Italy had shown no marked adverse effects the FDA decided to allow limited use of the drug in the treatment of the disease. Clinical trials will test the drug’s risks, harmful side effects or benefits. Individual patients who have applied for use under an Investigational New Drug application and whose requests were received by March 6 will be included in the trials. The remaining supplies of the drug will be used for ALS patients who did not meet the March 6 deadline but can apply to be selected for the clinical trials through a “lottery”.

The FDA website announced that, “The FDA is mindful of the need to strike a balance between access to unproven therapies for patients with limited treatment options, and the ethics of subjecting those patients to drugs with unacceptable risks or unconfirmed benefits. Today we have chosen a means to provide access to Iplex to as many patients as possible, consistent with all of the considerations discussed earlier. “

Any ALS sufferer who is part of the clinical trials will not only be a link to improved treatment for their own disease, but could benefit all who suffer. If the trials indicate that Iplex is beneficial to those who suffer from ALS it may not only provide treatment but could lead to more and better drugs for treatment and all those who suffer from ALS could really “win the lottery.”

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