Research Efforts
– Gene Therapy to TPP1 Deficiency

Blake’s Purpose is proudly supporting a research project “Gene Therapy to TPP1 Deficiency” with Dr. Beverly Davidson at the University of Iowa.

As all too many families know, current treatments for children afflicted with lysosomal storage disorders such as battens are merely supportive and do not alter the diseases progression. In recent years, researchers have identified the deficient enzyme in Batten’s Disease patients (TPP1) we now have the ability to work on a potential cure for this disease using Gene Therapy methods.

Updates on “Gene Therapy to TPP1 Deficiency” Trials:

• Davidson Laboratory has been able to reverse the deficits caused by this disease in mice.
• In the spring of 2010 her researchers progressed from working on mice with TPP1 deficiencies to dogs inflicted with the disease.
• Currently Dr. Davidson is currently working with the dog models on administering a “cocktail” of medicines using a couple of techniques; the first method administers treatment using brain surgery, while the second method uses vessels in the body to transport the medicines.
• Applications testing each method should be complete in the late spring of 2010, which means that we should know which method is having a greater impact on the dog models by fall.

In conjunction with the current study being funded by The Blake’s Purpose Foundation, Dr. Davison has been given funding to run a parallel study testing out these methods in non-human primates. It is felt that the laboratory can be more productive with these studies being done simultaneously, rather then being done in a series.

We are feeling very positive about Dr. Davidson’s findings thus far; however there is still so much work to be done. Upon a successful completion Davidson laboratories will be looking for more funding to produce a clinical grade drug to further this research, so the importance of fundraising doesn’t stop here, we still need your help!