TERMINAL ALS PATIENTS DENIED EMERGENCY ACCESS FOR COMPASSIONATE USE OF IPLEX

February 23, 2009

As stated in the FDA's own Patient Access to New Therapeutic Agents for Pediatric Cancer: Report to Congress 15 (Dec. 2003) “Because the FDA's standards for terminally ill patients who lack alternative treatment options are accommodating, most –- indeed, nearly all –- of the single-patient IND requests submitted to FDA are approved.”

In violation of their own standard for compassionate care to the terminally ill, the FDA has denied access for ALS patients to Iplex, the only bio-chemical pharmaceutical known to reduce symptoms and improve the quality of life for this terminally ill population. Iplex is not a cure. It is the best opportunity that's been developed and one that must be available to ALS patients as soon as possible.

Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease) strikes without warning -- anyone, anywhere, anytime. ALS begins with irregular limb weakness, body-wide tremors and speech difficulty. Eventually, frequently within months, patients lose the ability to move, breathe, eat, drink and speak. ALS sentences its victims to what is called "life in a glass coffin" and death within 2 to 4 years. More than 7000 new cases of ALS are diagnosed annually in the US.

Iplex was available from November 2006 to February 2007. The few ALS patients who used Iplex described the experience as a seismic shift in their bodies, offering the ability to eat, speak, breathe, walk more easily and at least temporarily reverse other symptoms of ALS. Iplex was then withdrawn from the market due to a corporate patent dispute between Genentech, Tercica and Insmed. After intense lobbying by the ALS community, the corporations reached an agreement on November 8, 2008 that Iplex would be available to ALS patients worldwide, subject to regulatory approval.

The FDA then determined that Iplex (which, before being withdrawn, had been available through single, off-label prescriptions issued by licensed physicians) must now be subject to an extensive IND (Investigational New Drug) request from each patient's physician. Patients were verbally assured by FDA these requests would be accepted, the FDA administrators even stating their knowledge that “Iplex was totally safe.”

ALS patients were shocked and devastated when, on January 16, 2009, the FDA rejected the first IND requests they received from ALS patients and have since stated that all future requests will be rejected.

The FDA's reasons are spurious, based on “unsubstantiated reports” that the drug may be dangerous and perhaps fatal. There are no such reports worldwide. Iplex has been tested and found safe for infants and young children. The second reason offered is that “bloggers would want Iplex for their own use, thereby diminishing or negating the potential for clinical trials.” This is also a non-issue and false. No clinical trials are scheduled and even if there were, most ALS patients would be ineligible because their condition is too advanced. Iplex may be the only oasis in the desert of ALS and should not, cannot, be denied.

The FDA must be urged to reverse their decision to reject Iplex, an already FDA-approved pharmaceutical for other conditions, abide by their previously-stated policy of facilitating alternative treatment options for the terminally ill and allow ALS patients access to Iplex through off-label prescriptions.

Please contact your legislators and the FDA stating your support for Iplex availability to the ALS community.