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#8 - Treatment Option Priorities [Kasey Altman Hackathon]

Thanks again for your interest in the Kasey Altman hackathon to find the best next treatment for this rare cancer patient. This is our weekly update (#8), especially for those who were unable to join us in person on Thursday (July 22).

In this session we focused on winnowing Kasey’s potential treatment options toward a short list of the two to five best, so that when we have our review board conversation in a few weeks, we will be discussing a shorter, more manageable list we can focus on for her best next treatment. On our working document of treatment options (here), there are currently between twenty-five and thirty options.

Experts Berkley Gryder, Elena Brin, and David Shalinsky shared their views on how they would think about the decision Kasey and her family will face, and shared the choice they would make.

  • Berkley Gryder: Focusing on Kasey’s PAX3-FOX01 fusion [The PAX3-FOXO1 fusion gene is a signature genetic change found only in alveolar rhabdomyosarcoma, and thought to be responsible at least in part for its malignant phenotype.] and MYCN amplification [The MYCN gene is a member of the Myc family of oncogenes. These genes play important roles in regulating cell growth and division (proliferation) and the self-destruction of cells (apoptosis).], and HDAC inhibitors [Histone deacetylase (HDAC) inhibitors are a relatively new class of anti-cancer agents that play important roles in epigenetic or non-epigenetic regulation, inducing death, apoptosis, and cell cycle arrest in cancer cells.] There are three drugs which inhibit HDAC on Kasey’s list, and I would choose Entinostat. 

  • Elena Brin: I would advise to consider a combination of at least two drugs, such as an HDAC inhibitor + another drug, e.g., a BRD4 inhibitor [Bromodomain-containing protein 4 (BRD4) is an epigenetic reader that recognizes histone proteins and acts as a transcriptional regulator to trigger tumor growth and the inflammatory response; inhibition of HDAC is thought to reduce PAX3-FOXO1 expression, while inhibition of Brd4 can downregulate Myc] would be ideal. Alternatively, a combination of HDAC inhibitors with common anticancer drugs for rhabdomyosarcoma (such as vincristine, actinomycin D, cyclophosphamide, etoposide and doxorubicin). Another combination to consider – an mTOR inhibitor [mTOR inhibitors are a class of drugs that inhibit the mechanistic target of rapamycin, that regulates cell growth, cell proliferation, cell motility, cell survival, protein synthesis, autophagy, and transcription.] and a tubulin inhibitor [Tubulin inhibitors are chemotherapy drugs that interfere directly with the tubulin system to inhibit cell division (mitosis)]. A third option would be a cancer antigen-specific delivery of a drug or cell therapy (e.g. anti-B7-H3-topoisomerase I inhibitor antibody drug conjugate or anti-B7-H3-CAR-T).

  • David Shalinsky: I would look at approved drugs (such as crizotinib is for ALK), mTOR, and DNA damage and repair.

We are updating and simplifying Kasey’s treatment options document with this input.

For more details, please reference the edited transcription of the meeting (below), and the video recording of the meeting below.

In Closing: Our Request

  • If you were in Kasey’s Review Board meeting, which treatment would you prioritize for her?


Kasey Altman, Delsee Altman, Lupe Montes, and Brad Power


Update #8 (5 Mins)

Q&A + Discussion (52 Mins)

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