Genzyme Corporation has put its money where its heart is, hoping to leverage support from like-minded corporations and individuals toward the creation of the NDRI National Rare Disease Biomaterials Resource (NRDBR). The proposed plan will expand on NDRI's successful rare disease pilot initiative to secure more human tissue resources for investigators in the field.
"We at Genzyme Corporation have been involved with and interested in treating patients with genetic and rare diseases since our inception," says John McPherson, Ph.D., Senior Vice President, Cell and Protein Research and Development. "Our initial contribution of $50,000 will help NDRI get the ball rolling to secure additional funding needed to ensure the rare disease initiative's continued progress." Genzyme has pledged to contribute $50,000 a year over the next five years to NDRI as agreed upon milestones are met to increase the supply of donated human tissues and donor DNA and cell lines for investigators studying rare diseases.
"Genzyme and NDRI have worked together for many years," he adds. "They have supplied human tissue for our tissue engineering initiatives around cartilage repair and cardiac cell therapy, and most recently in our development of a device to help patients with acute renal failure." Genzyme has also worked with NDRI in the context of both stem cell and islet cell research programs. "We have had a longstanding relationship with NDRI that has been very productive and very helpful."
For a number of years, Genzyme has been involved in research and product development for rare genetic diseases known as lysosomal storage disorders (LSDs), launching the first enzyme replacement therapy for Type 1 Gaucher disease in 1991. Since then, the company has developed a recombinant version of the enzyme as a second generation treatment for Gaucher disease and introduced other treatments for Fabry disease, Mucopolysaccharidosis I (MPS I) and Pompe disease. "Additionally," he says, "we have invested over the years in gene therapy platforms for cystic fibrosis and are now also working on a treatment for patients with polycystic kidney disease."
As NDRI's efforts continue to broaden access to tissue resources for rare disease research, Dr. McPherson predicts, "Investigators from both academic institutions and industry will gain a better understanding of the nature of these diseases and the specific pathophysiologies that are contributing to the mortality and morbidity associated with them. We are confident NDRI will put our seed money to good use."