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Andy and JC get the lowdown from Robert Pacifici, CSO of CHDI Foundation, on the latest in Huntington's treatments from the recent 20th CHDI Therapeutics Conference in Palm Springs, California. Robert has spearheaded efforts at CHDI for the past 22 years, driving forward preclinical drug discovery programs and corralling efforts to develop bioassays, clinical biomarkers, patient-staging systems, clinical and regulatory consortia to galvanize industry efforts in this devastating and fatal neurodegenerative disease. Just four years after the field suffered several reverses for ASO therapies in late-stage clinical development, there is renewed optimism that a new set of disease-modifying therapies are within reach.

The Boulevardier

INGREDIENTS:

1 Oz bourbon (or rye)

1 Oz sweet vermouth

1 Oz Campari

Ice cubes

DIRECTIONS:

Stir over ice for 45 seconds, strain into a rocks glass over a large ice cube, and garnish with an orange peel.

Enjoy!

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Check out presentations given at the 20th CHDI Therapeutics Conference here

For a summary of the latest developments keep an eye out for a forthcoming news story at Nature Biotechnology

Paper's mentioned in the conversation:

Ai Yamamoto, José J Lucas & René Hen. Reversal of Neuropathology and Motor Dysfunction in a Conditional Model of Huntington's Disease. Cell 101, p57-66 (2000).

The Mixer music “Pour Me Another” courtesy of Smooth Moves!

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William Pao, CEO of Revelio Therapeutics, and former Chief Development Officer for Pfizer/Head of Roche pRED, sits down with JC and Andy to talk about his new book: 'Breakthrough: The quest for life-changing medicines'. By charting the ups and downs of eight different drugs and the challenges faced by those shepherding them through the complex drug approval process, the book provides a fascinating peek into the hidden world of pharmaceutical discovery and the tireless work of the dedicated R&D professionals who develop life-changing medicines.

INGREDIENTS:

6-8 mint leaves

Bar spoon of water

Ice cubes

1 Oz elderflower liqueur (e.g., St. Germain)

4 Oz Sparkling wine (e.g. Prosecco)

1-2 Oz Club soda.

DIRECTIONS:

Put 6-8 mint leaves and a bar spoon of water in a wine glass and muddle gently. Fill the glass with ice cubes and add the elderflower liqueur, sparkling wine and club soda. Mix gently. Slap a mint sprig to release the aromatic oils and add it as garnish. Enjoy!

The Mixer music “Pour Me Another” courtesy of Smooth Moves!

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Sebastian Giwa, co-founder of the Organ Preservation Alliance and the Biostasis Research Institute and serial entrepreneur, talks to JC and Andy about the promise of organ storage research in transplantation. Seb obtained a PhD in Economics at the Stockholm School of Economics and was named a Baker Scholar at Harvard and worked at Bridgewater Associates, Bain and Goldman Sachs. Since spending time at Singularity University/NASA's Global Solutions Program and working with the Obama White House, he has worked tirelessly to galvanize the new field of whole organ preservation seeking to massively expand the availability of donor organs for the hundreds of thousands of patients awaiting transplants.

Seb's ventures in preservation: Sylvatica Biotech (named for the wood frog that can survive freezing and developing supercooling technology to extend organ life), Ossium Health (bone marrow banking from deceased organ donors for patients with sickle cell anemia, leukemia, and lymphoma), and GaiaLife (human ovary vitrification for girls with cancer who have not yet reached puberty).

Seb's preferred digestivo: Flaming Sambuca ("Sambuca, ghiaccio e mosche"; roughly translated: “Sambuca, ice and flies.”)

You will need:
1 2-1/2 oz shot glass
3 coffee beans
1.5 fluid Oz (44 mL) of sambuca
1 Butane lighter

Directions:
Place the beans at the bottom of the glass. Pour in the Sambuca. Use the lighter (do not use matches containing sulfur or phosphorus; preferably long-reach lighter to avoid setting yourself alight!) to light the sambuca, then wait until the flame is low/blue and cover the shot glass with your hand to extinguish it.

Groups mentioned:
Bischof, J and colleagues. Physical vitrification and nanowarming at human organ scale to enable cryopreservation. BioRxiv. Physical vitrification and nanowarming at human organ scale to enable cryopreservation (November 11, 2024; non-peer-reviewed preprint).

Rubsinky, B and colleagues. Bioengineering 10, (2023)

Kizer, KW et al (eds). Realizing the Promise of Equity in the Organ Transplantation System. (National Academies Press; Washington DC; 2022).

"Five years ago, even top cryobiologists doubted that a human organ would ever be successfully frozen and thawed...My opinion has changed." Janet Elliott, Canada Research Chair in Thermodynamics at the University of Alberta. Scuddelari, M. Proc. Natl. Acad. Sci. USA 114, 13060-13062 (2017).

Toner, M., and colleagues. The promise of organ and tissue preservation to transform medicine. Nat. Biotechnol. 35, 530–542 (2017).

Uygun, K. and colleagues. Supercooling enables long-term transplantation survival following 4 days of liver preservation. Nat. Med. 20, 790–793 (2014)

The Mixer music “Pour Me Another” courtesy of Smooth Moves!