TrialSite News is, above all, dedicated to transparency and access to clinical trials with an emphasis on clinical research sites and investigators—those who are on the frontlines of clinical research. We bring to light the events and highlights of the clinical research—from translational research to clinical trials—that we believe patients and health professionals should know about. From life-saving therapies to unacceptable behaviors centering on greed and corruption, we cover it all. One such case of highly questionable practices came to our attention in the case of Errant Gene Therapeutics. We first covered them in our article, which has thousands of views, “A Familyman’s Battle Against the Forefront of Capitalist Medicine: The Case of Errant Gene Therapeutics.” We kept our audiences informed thereafter with “Follow-Up on Errant Gene Therapeutics: Their Major Victory in New York Court,” “Summaries of Errant Gene Therapeutics vs. Sloan Kettering Court Exhibits,” and finally, “Their Justice-Driven Road to Prosecution: Errant Gene Therapeutics vs. Third Rock Ventures.” 

Since then, TrialSite News has spoken with Patrick Girondi, founder and CEO of EGT, and thought it important to update our readers on where he’s at now. Currently, Patrick is in Tanzania, Africa, visiting Professor Lucio Luzzatto and Dr. Julie Makani at Muhimbili National Hospital in Dar es Salaam, one of the largest hospitals in Tanzania with the highest population of Sickle Cell Disease (SCD) patients. (Tanzania has about 11,000 SCD births annually.)

Luzzatto was for several years the President of the Ethics Committee of the American Society of Gene Therapy; he set up and chaired for several years the Department of Human Genetics at Memorial Sloan Kettering, and he has been a pioneer in gene therapy. Professor Luzzatto has been truly instrumental in bringing awareness to the world. Last year, together with Julie Makani, Professor of Haematology at Muhimbili, he organized an International Haematology workshop where top researchers like Michel Sadelain and Dr. Marina Cavazzana attended. We invite you to also read his article, “Outrageous prices of orphan drugs: a call for collaboration.”

Julie Makani is a professor in the Department of Haematologyand Blood Transfusion at the Muhimbili University of Health and Allied Sciences(MUHAS). Also a visiting fellow and consultant to the Nuffield Department of Medicine, University of Oxford, she is based in Dar es Salaam, Tanzania. In 2011, she received the Royal Society Pfizer Award for her work with sickle cell disease.

Girondi’s Take on This

Our drugs in the U.S. may become so overpriced that patients will need to go to other countries for their treatments, going against America’s interests. Girondi, for example, is patient-centric in his role as CEO. He has never taken a salary, and his initial intention with starting EGT has always been golden. 

He states in an email to TSN directly that “Since my son was diagnosed in 92, there has been this team that included Dr. Susan Perrine from Boston University and Patricia Giardina from Cornell Weill, Nica Cappellini from U of Milan, collaborations with St Jude, NotreDame, Memorial Sloan Kettering, Cornell Weill, NIH, U of Illinois, Amsterdam Medical Center, Tor Vergata University in Rome and more. This was all done the old fashioned American way.” He continues, “I, the CEO, invested not divested. My president of 10 years, Sam Salman, never took a paycheck. We were able to produce the world’s first commercial batch of gene therapy vector in 2010 on a shoestring budget of 15,000,000.”

TrialSite News has commented on the need for both scientific altruism and for commerce—both are needed, but if  for example the quest for money far outweighs patient interests then over time everyone will pay.

The Race is On

With the current court case being appealed over and over again, the race is on between EGT/SKI and Bluebird to come up with enough data to get FDA Approval to take this miraculous gene therapy to market. Girondi hopes that by collaborating with experts in the field, including Professor Luzzatto, John Tisdale, Christopher Ballas and more, he can come up with a game plan on how to get his company’s product to market in a way that is sustainable for them and makes the drug accessible to patients. But the odds are against those with the financial wherewithal in the business of drug development.   

According to Girondi, “Over decades, I have seen and managed pull and tug with researchers, juggling resources and priorities. The management team must lead by example. Research needs resources. The Magic in keeping costs down is in allocation of precious resources. Investors, researchers and patients are betrayed when management takes excessive compensation. Whether this comes through salary, stock options or bonus, it all affects the cost of the final product. The key is accessibility to the people who need the treatment. This is one of management’s top priorities. Great examples in my opinion are Emil Kakkis CEO of Ultragenyx, Chris Garabedian, X CEO of Sarepta and Jeffrey Marrazzo, CEO of Spark, all people who have led by example.”

Scientific altruism reflects an important movement that must gain steam in not only America but also worldwide. Commerce is important—the market represents an important underlying social and economic-driven systemic construct to allocate capital, labor and resources—for what we hope is ultimately a force of efficient and effective contribution to ongoing advancements in critical areas, such as medicine. Dynamic groups, such as EGT, should be embraced—its energies infused into a compelling drug development ecosystem reflecting altruistic and capitalistic forces. 

Call to Action: TrialSite News will keep tabs on this story to keep readers informed. “Knowledge is power,” and it’s that power that will start an important change in the industry.

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