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PCUT's pilot project is the non profit manufacture of a promising biological product for the treatment of Amyotrophic Lateral Sclerosis (ALS).
Also known as Lou Gehrig’s Disease, ALS is a uniformly fatal neurodegenerative syndrome affecting more than 30,000 Americans and their families. The cause and mechanism of the illness are poorly understood, and in the 140 years since ALS’s discovery, no approved therapies have been effective at slowing the neuromuscular deterioration of the sufferers.
The product we pursue has, however, demonstrated a dramatically favorable effect on patients who have had the chance to use it. Its safe use in humans has been well established in clinical trials in over 120 people.
Mecasermine Rinfabate (rhIGF-1/IGFBP3) was FDA approved for growth hormone insensitivity disorders while trademarked as Iplex in 2006. In peer reviewed clinical trials on over 120 patients, the drug made good on its promise to deliver IGF-1’s restorative qualities to the body in a safer and more stable form than that of earlier approved free IGF-1 therapies. In fact, no serious adverse effects (SAE's) have been attributed to the drug’s use in the reports of any of the trials. Its potential for ALS is that the IGF-1 has long shown neurotrophic (restorative and protective) effects in motor neurons, and the IGF-1/IGFBP3 complex is a stable and effective delivery mechanism in the human body. Furthermore, the ongoing U.S. Investigational New Drug (IND) program has shown favorable results in patients For a disease about which so little is known and for which all approved therapies are ineffective at slowing progression, the safety and efficacy data we have for Mecasermine Rinfabate clearly merit further human trial.
Due to economic reasons, the drug’s producer de-prioritized the project and dismantled its production capacity in early 2009, rendering the drug unavailable to all but a few patients in existing Expanded Access Programs. The existing supply is expected to end by the end of 2010.
Partnership for Compassionate Use Therapies (PCUT) is now working to resume the production so that the drug is available, as rapidly as possible, to the community of ALS patients who choose to try it in their fights to slow their disease progression.
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