SPF Conference 2023 USA

Latest research presentations

For years, the annual SPF conference has provided access and insight into HSP and PLS research that is going on. The SPF itself has given almost US$12 million in total research grant funding over the years. The contribution of the SP Foundation is inestimable. 2023 is yet again another great conference, with the sessions available on the SPF YouTube channel. Congratulations and a big thank you to all involved.

Below is a summary of the major presentations at the conference, time stamped for convenient viewing. If you want to stay up with the latest research, then this is a ‘must watch’.

Friday Sessions

00:39:35 Diagnosis to Hope: LIfe as an adult caregiver

3 parents of children with HSP presenting (Chris, Katie and Bridget)

Empowering parents: Acceptance to Action to Appreciation

01:24:00 Education

01:37:00 Recreation and socialization

01:48:30 Childhood onset HSP: results of 2022 Survey

Ece Filiz (SPG3A parent) and Bridget Lassig (SPG4 parent) designed the survey.

03:46:00 “Rare Across America”

Deena Landfair (SPG7) – Education and Ambassador program.

06:16:50 Clinical trial readiness for childhood-onset HSP

Dr Darius Ebrahimi-Fakhari

06:20:55 What have we learned? 

06:26:40 Q&A

06:42:15 END

Saturday Sessions

01:09:30 Opportunities in HSP genomics and therapy (HSP-CERN)

Dr Stephan Züchner

01:34:20 HSP biology

Dr Craig Blackstone

01:57:30 Clinical trial readiness for HSP: lessons learned from translational research in rare diseases

Dr Darius Ebrahimi-Fakhari


05:38:50 Developing clinical trials for UMN diseases

Dr Hande Ozdinler

Drug candidate AKV9 (formerly NU-9)

Akava Therapeutics (Dr Silverman)

NU-9 treatment in animals


06:43:15 End

07:54:04 Panel Q&A

With Drs Piermarini, Liang, Mukesh, Ozdinler, Ebrahimi-Fakhari, Fink and Blackstone

09:30:30 End

Sunday Sessions

00:31:20 Gene therapy approach for SPG4-based HSP: a preclinical study

Dr. Emanuela Piermarini

00:35:00 Spastin isoforms; gain/loss of function; transgenic mouse model

00:42:00 Gene therapy approach

00:52:00 Alternative directions – Drug therapy CK2 inhibitors

00:55:00 Q&A

01:11:30 End

01:12:25 HDAC6: A promising therapeutic target for SPG4

Dr Liang (Oscar) Qiang

01:15 HDAC6 insights; Marion Murray Spinal Cord Research Center;

drug testing, embryonic stem cells, brain banks, in vitro, in vivo, AI

01:22 Mouse models, spinal cord

01:31 Human brain organoids for SPG4

01:36 Outcome measures for human organoids

01:38 What did we find? Reduction of stable microtubules.

01:41 HDAC6 activated by M1 isoform; what is HDAC6? Druggable target

01:51 Tubastatin A (Tub A) known HDAC inhibitor; partially reverses axonal defects and impaired gait in SPG4 mice; partially rescues early axonal degeneration in brain organoids; no adverse effects

01:52 High-throughput analyses to identify targets for SPG4 mechanisms Proteomics and Single-cell RNA sequencing to study M1 & M87 isoforms 

02:03:00 Learning from other research arenas – spinal cord injury

02:09:30 Q&A

02:15:15 End

02:38:05 Open Q&A session

Dr John Fink

03:46:15 End

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