AL Amlyoidosis is a rare disease which causes a buildup of harmful proteins (amyloids) in organs such as the kidneys and the heart. This accumulation causes organ damage as well as damage to various tissues in the body. The condition is treated by chemotherapy and stem cell transplants. Unfortunately, 80% of patients are not eligible to receive a transplant. Even for those who can, most patients sadly die from cardiac failure as neither of the approved therapies actually address the organ damage caused by the amyloids. The current rate survival is less than 18 months after diagnosis. There is very clearly an unmet need for this patient population.
Fortunately, thanks to collaboration between researchers, there is slow but steady progress on new therapeutic options. Caelum Biosciences and Alexion Pharmaceuticals have just announced their collaborative effort to develop a treatment which will actually reduce or eliminate amyloids in the body’s organs. This would improve organ function and ultimately stop the course of the disease. The potential treatment is called CAEL-101.
Researchers are very optimistic about the promise of CAEL-10 as a new treatment option.
This drug has already been studied in a Phase 1a/1b trial. It included 27 patients who were diagnosed with refractory AL amyloidosis which had relapsed. None of these patients had ever had improved organ function with standard treatments. This trial indicated that CAEL-10 was able to rapidly improve organ function. This improvement was especially prominent in the heart and kidneys. Additionally, the therapy was well tolerated in patients.
CAEL-10 has already received Orphan Drug Designation from the FDA. This designation extends to its use as both a therapy and a radio-imaging agent within AL amyloidosis.
Caelum and Alexion will work together to design the development program for the drug. Caelum will be responsible for actually conducting the development program through the Phase 2 clinical trial as well as manufacturing CAEL-10.
This collaboration is yet another indication of the importance of fusing minds and resources in order to best serve the patients who need it most. Hopefully, we will see this recent collaboration turn into a new treatment option for the AL Amyloidosis patient population soon.
You can read more about CAEL-101 and Caelum and Alexion’s work here.