The Lab in Charge of Curing Glanzmann’s Thrombasthenia

The last weekend of April 2022 was quite the weekend, to say the least. Board officers visited Dr. David Wilcox in his lab (the Kelly Weil Laboratory) at the Medical College of Wisconsin. The lab has been working for decades to make many advances in gene therapy including a specific type of gene therapy that would not only be able to cure Glanzmann’s Thrombasthenia but would have little to no risk to the patient.

The process would involve first taking bone marrow cells from the patient. Unlike a traditional bone marrow transplant, this would be a sort of “micro” transplant where only a small amount is needed.

Patients would stay in nearby housing during the process where family members could also stay/visit.

While there, doctors would begin the process of gene therapy. Doctors will correct specific cells that will instruct the body to produce the correct type after they are transplanted. Since the cell that has gone through the therapy is taken from the patient themself, there is little to no chance that the patient’s body will reject it (graft vs. host disease or GVHD).

After the patient receives the new cells they are closely monitored to make sure all goes well. During this time the patient will be in an ICU-style room to receive immediate care if needed. 

Of course, we all want to know when clinical trials will begin? We are in the final stretch! We need to spread awareness of GT and gene therapy. Funding is of huge importance so Dr. Wilcox can continue to push forward to get approval for clinical trials.