We need to raise $50k to study Syngap Mini-brains

Executive Summary:

• There is a new opportunity for SRF & the Syngap Community to support a team of researchers from the Keck School of Medicine of USC on their work of unlocking and understanding the Syngap mutation.
• Dr. Coba has been connected to SRF since 2017 and since then he has invested time and resources in studying Syngap. Recently he has collaborated with Dr. Giorgia Quadrato to develop organoids or mini brains to generate knowledge about Syngap1.  
• We are hoping to support this new revolutionary area of research with a grant of $50,000 for the purchase of equipment that will enable this research team to show proof of principle in a new area of research in this new field.  If we hit our target, it  will be matched by additional commitments from outside our community and support and assist Drs. Coba and Quadrato to stay engaged in Syngap Research.

Great news!

SRF has identified an opportunity to expand quality science in Syngap and to encourage more strong academic scientists to invest their time in creating knowledge that may accelerate a therapy for our children.  This article is an ask of all the friends of Syngap to help SRF raise the funds needed to make this happen.

After learning more about the work of Drs. Coba and Quadrato, we reviewed their proposal with our Scientific Advisory Board (SAB) who is supportive of this project.  We are pleased to share this with the parent community, and request your support for this initiative.  

100% of donations received by SRF go to research, as the founders cover all overheads.  Please read on and learn about why we are excited about this, and then consider donating and sharing this project with your networks!  

Who is Dr. Marcelo Coba & how did he become interested in Syngap?

Marcelo Coba, PhD, is an associate professor of psychiatry and behavioral science at the University of Southern California’s (USC) Keck School of Medicine. His research focuses on systems biology, synaptic signaling complexes, protein phosphorylation, and psychiatric disorders, along with other SynGAP1 relevant topics.

Dr. Coba’s impressive educational background includes a Doctor of Pharmacy (Pharm.D.) and postdoctoral work in Neuroscience. His education sets him apart and aligns well with SynGAP patients’ needs. He already holds an RO1 grant, which is the pinnacle of science funding in the US. His team has been studying Syngap for some years, and is an exciting addition to the strong list of researchers in the Syngap community.

Hans Schlecht, MD--an infectious disease doctor, a fellow SynGAP parent, and the Science and Medicine Lead on the SRF team--is the reason SRF is connected with Dr. Coba.  Upon receiving his son’s diagnosis, Hans was eager to see what science was currently being conducted to learn more about SynGAP1 mutations, with the hope of finding a treatment. In 2017, he searched SynGAP1 as a keyword in the NIH Reporter. His search yielded the names of the researchers that mention the protein or mutation in their work. Through his networking, Dr. Schlecht scheduled a phone call with Dr. Coba. He was immediately impressed by Dr. Coba’s willingness to engage and to be a partner with a common goal.

Reflecting on Dr. Coba, Hans said “A doctor is assessed on the three As: Affable, Available & Able. Coba is all of these.  He is easy to talk to, he replies to calls and emails and he’s a capable scientist.”

Early on, Dr. Coba offered to meet Hans when he was out for a meeting with the Broad.  Hans drove out and they spent a few hours together.  Since then Dr. Coba has met with other SRF leaders at Global Genes in 2018 and he attended our 1st Roundtable at AES last year.

Mike Graglia, Managing Director of SRF, said about Dr. Coba that he “is simply fascinated by the science and SynGap. He has spent hours letting us pick his brain and ask questions about his papers. Maybe it is his training as a pharmacist, but he is really easy to work with and talk to. I’m thrilled to think that SRF is supporting his work.”

What’s the Science?

Drs. Giorgia Quadrato and Marcelo Coba are using patient-derived human brain organoids generated from stem cells. Organoids are complex, 3D structures that exhibit human-specific qualities much more accurately than the alternative research strategy, mouse models. They can be thought of as miniature brains in test tubes that react in ways very similar to actual human brains, thereby allowing Dr. Coba and Dr. Quadrato’s teams to conduct “clinical trials” of potential treatments in petri dishes quickly and safely.

How Do We Make This Happen?

By supporting the Syngap Research Fund’s commitment of $50,000 grant. You will be supporting half of the purchase cost to specialized equipment, which will significantly accelerate their research into developmental disabilities. The balance of the cost will be paid by the Keck School of Medicine

Together we can help Drs. Coba and Quadrato increase scientific knowledge about developmental disabilities caused by the SNYGAP1 mutation and ultimately develop life-changing treatments.

Please help SRF advance Syngap Research for Rare Disease Week

Please donate to help SRF support USC in securing and maintaining this cutting-edge technology. Our hope is to foster this work so scientists and researchers like Drs. Coba and Quadrato are encouraged to mentor other minds and thus have a multiplicative effect on science!

Donate to our Go Fund Me or if you prefer to send a check, please use the donate button above for details and put “Organoid” in the memo line.

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