INTERIM REPORT OF IPLEX USAGE
FOR TEN ALS PATIENTS
The original terms established for those patients admitted into the Trial for Experimental Usage of Iplex (Mecasermin Rinfabate, rhlGF-1/rhlGFBP-3)required an evaluation to be prepared and submitted at the end of the first year of usage. This interim report has been prepared for the following reasons:
Because the average life span of an ALS patient is between 2 to 4 years post diagnosis, the ALS patient population has been persistent in their inquiries about the status of IPLEX, whether it is feasible or even advisable to be used.
The anxiety and stress level of the ALS patient population is extremely high, leading to an even greater worsening of symptoms. Providing information sooner rather than later appears to be the more humanitarian and compassionate approach to take.
Patients currently taking Iplex have asked that an evaluation be done sooner rather than later. Those few admitted into this clinical trial feel an obligation to the rest of the ALS community to be as forthcoming with information as is possible.
Therefore, the following interim report of results is being provided.
During the 30-week period of Iplex usage that comprised the first half (26 weeks and follow-up four-week period) of the one year authorized investigational new drug trial, there was a remarkable parity between the apparent responsiveness of the individual patients compared to the aggregated group of patients at large. Symptoms monitored for their absence, presence, or their degree of severity included the following: Hyperreflexia; Tongue Movement; Swallow; Hand Strength; Shoulder Strength; Fatigue; Clonus; Nausea; Atrophy; Breathing; Lability; Lower Arm Strength; Fasciculations; Dizziness; Cramps/Pain; Weakness; Balance; Speech; Upper Arm Strength; Tremor/Palsy; Rigidity; Libido. In the consolidated 30 week period, individual symptoms showed a maximum decline of 9% (Balance) followed by 6%(Tremor/Palsy). The maximum improvement of an individual symptom was 39% (Fasciculations) followed by 28% (Fatigue). The critical function (Swallowing) improved by 25% and Breathing improved by 1%. Compared to the normal relentless worsening in virtually all symptoms over any continuous 30-week period of time, the use of Iplex appears to have stabilized, if not improved, the condition of its users. Those using Iplex demonstrated an average improvement of symptoms of 9.3% (all symptoms combined) after the 30-week period. There were no adverse events or serious adverse events during this 30-week period.
The structure of the investigational new drug trial did not allow for the use of a double blind placebo controlled study. Further, the use of an historical control was assumed to be of less veracity than the carefully scrutinized observations of the patients themselves, supported by caregiver observation and the supervising investigator.
Associated issues of limited supply of Iplex by the manufacturer Insmed and extremely high cost associated with the use of Iplex other than when it is provided without charge to the patient as part of this investigational new drug trial remain. Therefore, continuing supply and patient cost continue to be obstacles that must be resolved prior to Iplex becoming a viable resolution for the ALS community.
We urge that FDA greater oversight and or other potential manufacturing alternatives be quickly and purposefully explored in order to maximize the benefits of Iplex usage by middle- and late-stage ALS patients.
Read the Report in its Entirety
What makes IPLEX the drug of choice?
IPLEX is free IGF-1 combined with the binding protein IGF-3, one of six naturally occurring binding proteins that have been shown to cross the blood brain barrier.
IPLEX reduces muscle atrophy.
The positive effects of IPLEX last longer and maintain stabilization.
IPLEX overcomes the obstacles presented in free IGF-1 (Increlex and Myotrophin). Dosage, half life, negative side effects of nasal swelling and hypoglycemia are virtually non-existent with IPLEX. The addition of the natural binding protein, BP-3, allows for significantly higher dosing, passes the blood brain barrier, and has a markedly increased half life. IPLEX is delivered by once daily injection, rather than twice daily. IPLEX is safe and well tolerated, even in infants for short growth stature for which it was originally developed.