Apart from the possibility that Shire will reactivate the process of obtaining EMEA approval of iPlex as a therapy for Short stature, two additional possibilities also seem promising -
1. SBMA / SMA -
"Findings show IGF1 compounds hold therapeutic potential
The researchers treated SBMA mice with a compound called mecasermin rinfabate (brand name Iplex), which is a combination of IGF1 and IGF1 binding protein 3. (The binding protein helps IGF1 last longer in the body.)
Male mice were randomly assigned to receive abdominal injections of the IGF1-based compound at a daily dose of 15 milligrams per kilogram of body weight, or an inactive substance. To mimic the typical clinical setting (in which a diagnosis of SBMA occurs after symptoms appear) the researchers began injections when the mice were 10 weeks old, after disease onset, and continued through 20 weeks.
The researchers evaluated body weight, motor function and survival in the mice. They found that, when compared to the placebo-treated mice, those treated with IGF1 had:
... less body weight loss;
... significantly improved grip strength; and
... longer survival time (by an average of approximately three weeks).
In addition, the researchers found that the IGF1-treated mice had markedly reduced muscle shrinkage (atrophy) and degeneration.
The study results provide a basis for further study of IGF1-based compounds as potential therapies for people with SBMA."
"SBMA may be a better candidate for IGF1-based therapies than ALS or MMD1, because in addition to the nonspecific benefits it confers on muscle and nerve survival, it changes the behavior of the abnormal AR protein that underlies SBMA."
2. Duchenne's - NCT01207908
"Update on IGF-1 Study at Cincinnati Children's Hospital"
"Interim analysis includes that gain in height was greater in the IGF-1 group compared to controls, but there will be no analysis of the neuromuscular outcomes until the study has been completed."