StemCell Therapy

SAN FRANCISCOOncology is clearly a major medical and societal issue: a major killer that, while predominately affecting the older population, can strike the young through a mixture of environmental factors or a genetic lottery. Its no wonder we struggle to even call it by its name, preferring just the "big C."

Biopharma has acted accordingly over the years and spent billions (and made many more billions) developing new oncology therapies, with the media and political focus falling on cancer drugs far more acutely than any other area, whether that be over pricing (the current average cost of a new cancer drug in the U.S. is around $100,000), effectiveness/safety or rejection from healthcare gatekeepers, such as the National Institute for Health and Care Excellence in England.

But cancer is only one disease area: Heart disease is the biggest killer in the U.S., yet there are very few new and innovative CV drugs out there, with influenza complications, such as pneumonia, Alzheimers disease, stroke and diabetes complications all leading causes of death in the U.S. There has been a war on cancer, but not a war on stroke.

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There is also the growing threat of antimicrobial resistance, where decades-old antibiotics are ceasing to work against common forms of bacteria as they evolve; this, coupled with the fact that most life science companies arent working on a next generation of antibiotics (R&D costs are high with little or no ROI), means we could very well be facing a new surge in deaths in the future from once preventable diseases and infections.

At the J.P. Morgan Healthcare Conference in San Francisco this week, we at FierceBiotech wondered what the industry was doing about this and asked a range of life science C-suite execs: What therapeutic areas beyond cancer are most important to the industry now? both in terms of unmet need as well as where there is some real innovation.

Jim Robinson, chief operating officer at Paragon Biosciences, said: I spent 10 years in oncology, so I understand that point. Still, the biggest issue we face today that has to be figured out is Alzheimers. Looking at the aftermath, its scaryits going to be trillions of dollars in 20 short years in terms of the expense treating patients with Alzheimers. In 20 short years, I might be one of those patients!

I think its been a vast wasteland of failure. Im hoping something comes about before Im 70 to allow us to treat it. Whether the industry is willing to shift more resources to pursue treatments or not remains to be seen, especially after the latest failures. I dont know if the industry will shift to Alzheimers or more CNS treatments associated with cognition, but Im hoping.

Oncology good news is when the industry shifted and thousands of drugs moved through development. We see a significant transformation in certain cancers. If the incentive or the approach from a regulatory pathway that shifts incentives to research in Alzheimers, we will find some answers.

The biotechs CEO, Jeff Aronin, who is also CEO of Paragon Capital Partners, echoed this need for answers. I have a focus there with one of our companies, but in general, I would answer a little broader, he explained. Ive been involved in CNS drug development for a very long time and remember the 90s, which they called the decade of the brain, but we really didnt make a lot of advances in neuroscience and psychiatry that we thought we would have.

I think over the next few years is where we are really going to see many medicines approved. We have learned so much more and advancing in many different areas. In neuroscience and psychiatry, I would add theyre also an area of tremendous cost to the healthcare system and we still dont have a lot of great solutions, whether its Alzheimers or any of the neuropsychiatry areas were working in.

BioNTechs Sean Marrett also saw Alzheimers and other neuroscience areas, such as Parkinsons disease, as still major and unmet issues, as well as multiple sclerosis, which has seen great strides but still needs work.

Alzheimers is certainly a major issue, but also one that is more entrenched in the west: We live longer and are therefore more susceptible to diseases associated with aging.

Lyndra Therapeutics CEO Patricia Hurter asked us whether we meant our question in relation to the developed world or the developing world? We asked her two cents on both.

Women in Africa either get pregnant or get HIV, she said starkly. Their economic prospects are horrendous. It means their childrens economic prospects are horrendous. Were working with Gilead on HIV prophylaxis and on an oral birth control. Eventually, when the drugs are potent enough, we could do a once-a-month pill of each. To have them in one capsulethat would be fabulous. That would be transformative for developing countries.

For America, I think so many things like hypertension and diabetes are a chronic epidemic and people are having bad health outcomes [that could be avoided] if they took well proven drugs in an adherent way. Its an adherence issue. Theyre not feeling sick so they dont take the medicine. But in fact, it is still progressing [it, referring to stuff like hypertension, NASH that progressively gets worse without you feeling sick until its advanced].

It leads to unnecessary hospitalizations. If we could combine modern methods of distribution that a poly-pill combination that a person neededonce a week they would take one thing to keep them from progressing.

Karuna Therapeutics CEO Steve Paul also pointed to suicide rates that keep going up, whereas in certain types of cancer and cardiovascular disease theyve started tracking down.

And, finally, Westlake Village Biopartners Managing Partner Sean Harper said: Because there are so many areas of unmet need, its hard to say just one therapeutic area or one modality. Its exciting: the cellular engineering thats going to result in the ability to do regenerative medicine type efforts is I think going to be the next really amazing sort of thing.

With the fact that you can manipulate human cells now the way you can, and make multiple difficult edits to engineer things out of them and so on, that is just a new frontier. There are a lot of settings where you can just imagine what that can do. Its not 100 years away. Its now. Its happening already, people are doing it. I think that, to me, is the most exciting area.

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JPM: Biotech, VC execs on where the industry should look beyond cancer - FierceBiotech

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